How to Make a Treatment Decision

Myeloma Patient Cafe®

Myeloma Patient Cafe® – How to Make a Treatment Decision from Patient Empowerment Network on Vimeo.

Five myeloma patients from around the country sat down to discuss their disease and how to make the best treatment decision for them.

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Transcript:

Cindy:

Hello everyone and welcome to the Patient Empowerment Network’s Myeloma Patient Café. My name is Cindy Chmielewski, and I’ll be hosting the program today. I was diagnosed with multiple myeloma way back in 2008. The Myeloma Patient Café is an opportunity for myeloma patients to get together and discuss their disease and also to share tips and information about myeloma.

Today’s topic is going to be how to make a treatment decision. But before we get into that discussion, I think it’ll be nice if we all go around and take a few minutes to introduce ourselves. We could say our name, where we’re from, and just a short history of our treatment journey. I’ll go first.

As you know, my name is Cindy. And I’m from Lawrenceville, New Jersey, and I was diagnosed with multiple myeloma back in 2008. When I was diagnosed, my myeloma was really tricky to begin with. My induction therapy stopped working for me just after a few cycles, and a stem cell transplant failed to put my myeloma into remission. So, I was very, very scared back then, and I retired from teaching.

But fortunately, a combination therapy was able to put my myeloma in what’s called a very good partial remission. But it took almost three years to get that maximum response. And right now, I’m staying in that very good partial remission with maintenance therapy.

Mike:

I was diagnosed in 2013 after having MGUS in 2009 and smoldering myeloma in 2010. My initial treatment was part of a clinical trial, and I had Revlimid, Velcade, and dex for three cycles, then a stem cell transplant, and then two more cycles of RVD and then maintenance therapy on Revlimid until 2018. I was really fortunate. I went into stringent complete remission and MRD negative. But then in July 2018, I began to relapse. So, I’ve started another treatment with Elotuzumab, Revlimid, and dex, and that’s been working pretty well recently.

Cindy:

Good. Thanks for that introduction. And Alan, would you like to share a little bit about your story?

Alan:

Sure, I was diagnosed in 2016. I was very far along, and the myeloma was very advanced. And when I was first diagnosed, I was put in the hospital and nearly died in the hospital because of some pain meds they gave me. It just shut down my digestive system. But anyway, everything happened really fast because it was so advanced. I really didn’t have much time to make decisions. We found it because my L5 vertebra had collapsed. I found out I had fractures in every vertebra, clusters of fractures in my ribs, 147 lesions on my bones, and 60 percent of the cells in my marrow were cancerous.

Like I said, it was so advanced I really didn’t have much time to make plans, but I went through two rounds of VDT-PACE. I went to a myeloma clinic that specialized in multiple myeloma. I did two rounds of VDT-PACE, then tandem stem cell transplants, then 16 rounds of Darzalex or daratumumab. Then they told me I was in remission and then went on maintenance treatments. I did Ninlaro and dex and – oh, Revlimid. That’s what it was. I did that for about seven or eight months. And my blood counts were just staying so low that they finally took me off that. My white count would never get over about 1.5 to 2. So, then I went back on the Darzalex, and that’s what I’m on now. I’m in stringent remission.

Cindy:

Good to hear that. You had some journey there. And Connie, can you tell us a little about your story?

Connie:

I was diagnosed January 3rd of 2010 after I fractured my sternum in a race. A gal was pacing me, and I thought I had a sports injury and just had too much fun sprinting. But it took about nine months before they finally realized that I’d fractured my sternum that day, and then I got diagnosed with the multiple myeloma.

Cindy:

And Josine, how about you?

Josine:

Hi. I was diagnosed actually five years ago this past month, Columbus Day weekend 2014, with 32 compressed fractures in my spine and my ribs from – I thought it was from lifting a box of legal documents at work in Manhattan, but it wasn’t. It was the myeloma pushing out because I had – 95 percent of the plasma cells were cancerous. Fast forward three months later I had a stem cell transplant, never went into remission. I’m on a triplet now of Revlimid, Ninlaro, and dex, and it brought my M-spike down to 0.2, which when I started out, I was at 8.0. So, life is good.

Cindy:

So, now let’s talk about how we make treatment decisions. And Mike, I’m gonna go back to you. When you were in smoldering myeloma, you had that opportunity to either watch and wait or to go on a clinical trial. What made you decide clinical trial? What was part of that decision-making process?

Mike:

When I was in smoldering myeloma, I wasn’t really offered a clinical trial. It was when things switched from smoldering to active myeloma. And what made me decide to do the clinical trial is a couple of things. One, my background is that I am a scientist, an engineer. So, I’ve been interested in the science behind treating multiple myeloma and other cancers and wanting to do my little part to try to help advance the science through participating in a clinical trial. So, that was one factor.

The second factor is that I felt like I’d get really good treatment period, but I’d get the very best treatment if I was in a clinical trial. And it turns out that I was monitored much more frequently while I was in the clinical trial than I would’ve been otherwise. And that ended up over time giving me, I think, some peace of mind. So, I’m really glad that I did participate in the clinical trial.

Cindy:

Good. Anyone else participate in a clinical trial or had that option brought up to them? No?

Alan:

I did not.

Cindy:

No. Did your doctors ever ask if you were interested? Or was that never brought to your attention?

Alan:

It was never brought to my attention.

Cindy:

Josine, did the doctor ever ask you?

Josine:

No, no. I would definitely be up for it, but it never came up in conversation.

Cindy:

How about you Connie?

Connie:

My numbers are very, very gradually going up like myeloma likes to do. And so, we have – looking ahead to a relapse at some point, my specialist brought up clinical trials and that we could discuss them and look them over and decide at that point.

Cindy:

Good. And while I’m talking to you, I think I read in your biography that you chose not to go ahead and have a stem cell transplant. Is that correct?

Connie:

That’s correct.

Cindy:

And can you talk about – a little bit why – what went through your head to make that decision?

Connie:

I think at that particular time the result of quality of life or overall length of life were very similar in terms of whether you had a stem cell transplant or in my case with standard risk myeloma. And I might at that time could’ve – everything that we had talked about and looked at with the research – maybe buy six more months of overall life – of survival. And I have to admit I was a little bit skeptical about – I guess I’d say killing off your immune system and how that might come back or not come back. And with standard risk, I decided to go up to Mayo Clinic and have them collect my stem cells as an insurance policy of sorts and then see how my myeloma progressed.

Cindy:

And at that time, were you in a complete remission when you were making the choice of whether or not to have a stem cell transplant?

Connie:

No, I wasn’t. I know the standard of care, and that was explained to me – was to have a stem cell transplant. But when I looked at the numbers with my specialist and my local oncologist, Revlimid was seeing some very good results. So, it really looked very similar to me. I thought with a deeper response up front we might get an overall better response, but I decided to – at my age – just to see how it would progress.

Cindy:

And that’s why this is such a crazy disease because everyone has a different presentation and yeah. And, I think, Alan, you were a little bit different. You had double transplants? Was that tandem transplants?

Alan:

That’s correct, about 60 days apart.

Cindy:

Can you talk about how you decided and why you decided to have such an aggressive line of therapy?

Alan:

Well, I think part of it comes to my personality type. I’m a business owner, and I’ve been fairly successful in life by surrounding myself with people that know way more than I do and letting them do their jobs. As I said in the beginning, things happened so fast with us we didn’t have much time to make decisions. And we were sent to a myeloma center. They specialize in multiple myeloma, and I feel like it’s probably the best in the world. But, of course, I guess everybody feels that way about where they’re treated, but my doctor was one of the top people in the world that specialized in multiple myeloma. He’s a clinical research scientist. And he told me how bad it was, and he said we’ve gotta treat it aggressively. But if we do, we feel like we can get you in remission.

And, you know, I really – when I first met him, he introduced himself by his first name, and I liked him, and I trusted him. And I had done a little bit of research on him, and I had just decided to do what he said. And it really made it – in many ways, it was easier for me because my situation was so bad that – and I know there are people that have had way worse than I have. But it was urgent that we make decisions. I didn’t really have a lot of time to think about it. So, I just surrounded myself with really good people and did what they said.

Probably the only decision I’ve really had to make is between my first transplant and my second. And like I said, they were only about 60 days apart. I did really well recovering from my first transplant. They released me to go home 14 days after my transplant, which they said was fairly unusual. The day I got home, I got C. diff And I was brutally ill, and then I got the flu. And then I got C. diff, and then I got the flu again. So, basically out of a two-month break, I was sick for a month.

Cindy:

Oh my gosh.

Alan:

And when I went back, he told me – he said, “Look, I know you’ve had a hard time. So, we can go one of three ways. Your test results were very good, so we can either let you go home for a couple weeks to recover.” Of course, he knew I had to run a business too while I was going through all that. And he said, “Or, we can do a reduced dose of melphalan, or we can just give you the full dose.” And I’m an idiot, and I said just give me the full dose. So, that also says a lot about my personality card.

But anyway – so, I don’t have any regrets. It’s taken me a long time to get back on feet, but I live a pretty normal life. About four or five weeks after I got home from my second stem cell transplant, I made arrangements to continue a family tradition of going fishing with my dad and my sons in South Louisiana. And I looked like a ghost honestly, but I’ve forced myself to do a lot of things I didn’t feel like doing so that I could recover.

Cindy:

Josine, can you talk about your initial treatment and if you were part of that decision-making process?

Josine:

Sure.

Cindy:

I know that initial treatment shocks us, and many of us are really, really sick. So, we’re really not part of it, but talk a little bit about your experience.

Josine:

Yeah. I was totally out of it at that point when I was first diagnosed because I was on morphine for 10 days, and I lost 10 days of my life. So, I never had anyone say that I had this cancer or discuss any treatments me. So, I was at their mercy. They gave me bendamustine and CyBorD – no. Yeah, CyBorD and bendamustine in the hospital. And I had, like I said, no recollection of any of it at all. My husband was there to make all those decisions at that time.

Cindy:

So, now, unfortunately, we have myeloma. And myeloma is one of those diseases of relapse and remission right now, although I’m very hopeful that we’ll be curing some people very soon. No one’s saying that they have a cure for myeloma. So, there’s a possibility that we will relapse in the future. Hopefully, the really, really, really distant future, but there’s that possibility. So, when we’re thinking about treatments, what do you think are some of the things that you consider when making that treatment option?

Mike:

In my case, Cindy, last year – last July, my oncologist gave me eight different options. And we went through the list, the pros and cons of each of those eight options, and finally together decided on the Elotuzumab, Revlimid, and dex. And it was very important to me to sort of understand what his thinking was and why he liked this option versus that option and so forth. Part of it had to do with how effective we thought things would be given my myeloma and my history with myeloma. Part of it had to do with side effects that we wanted to avoid. I have peripheral neuropathy left over from Velcade. So, that sort of ruled out Velcade. But anyway, it’s great that we had so many different options to be able to choose from. I’m fortunate enough to be in that position now. And it made me feel good to be able to go through those options one by one with my myeloma specialist.

Cindy:

Some of the things you considered is what your specialist was thinking, why he picked a treatment; but you also considered some side effects from previous treatments when selecting your new treatment, which makes a lot of sense. If you already had neuropathy, trying a treatment that is known to cause neuropathy might not be the best choice.

Mike:

Exactly.

Cindy:

Yes. Anybody else – things that they considered in the past when making a treatment decision or think they would be considering in the future for future treatment decisions?

Alan:

I had to make the decision about going on maintenance. And I have a lot of friends with multiple myeloma, and some of them chose not to do maintenance. I guess, once again, it goes to my personality type. The first thing I asked my specialist was, “If you were me, what would you do?” Nobody knows better than he does. And why would you do that. And he told me, and then the other side of it is – going back to my personality, I’m more likely to do the most aggressive thing to go ahead and get it over with. And that’s kind of why I decided I should finish my maintenance. They originally scheduled three years of maintenance for me. And I should finish that in January or February of next year.

Cindy:

So, it was trusting your specialist but asking why he chose that treatment.

Alan:

That’s correct.

Cindy:

But your personality is – go for the gusto there.

Alan:

Yes. And the other side of it is that right now I’m an 18-year-old in the body of a 70-year-old. I’m actually 52, but I figure I can handle the more aggressive treatments right now than maybe I could 10 years from now. I’ve got kids. I’ve got a grandson. And I figure if it means me doing the most aggressive things so I can be with them longer, I don’t mind doing that.

Cindy:

And Connie, I think one of your considerations was quality of life. I heard you talk about quality of life when you made that decision about not having your stem cell transplant right up front, harvesting your cells, keeping them in the fridge just in case you need it. Can you talk a little bit more about that?

Connie:

Yeah. I looked at having the stem cells available, so I could have a stem cell transplant if I needed one. And I wanted to continue to compete with the race walking. So, that was a small part of it. Also, I with my oncologist – this last appointment he mentioned that if my numbers continue to just go very slowly up that maybe the next appointment that we would discuss some options. He did mention that he liked – maybe for my particular situation – adding daratumumab. And we would be looking probably at a two- or three-agent combination. I’m, unfortunately, not able to tolerate the dex. So, I’m not sure how important a factor that is for trying to enter a clinical trial. I’ve noticed most of them do use dex because of the synergy there. So, that’s a bit of a concern for me as well.

Cindy:

I know you are not on your induction therapy now. You had some treatment decisions to make. Can you talk a little bit about what you thought about when going through those?

Josine:

Oh, sure. When the famous 100-day visit to the hospital – after you have your stem cell transplant; my specialist asked me if I wanted to go on consolidation or right onto maintenance. And I guess I was kind of like Alan. I’m like – I wanna do this consolidation first because I wanna do as much as I can to get to where I have to be and then start the maintenance. The only thing is I was on Velcade for those eight weeks, and there was no change at all in any of my numbers. So, then I was on Rev only for a year and a half.

And after that, the light chains went up, and I had new lesions. And my specialist had suggested adding Ninlaro and dex. And I had known that – well, he had told me. I didn’t know it then – that Ninlaro and Velcade were in the same class. And I was questioning him. I said, “Why would I go on that if the Velcade did nothing for me. And he said, “Well, working as a triplet it’ll work better.” And it really did. So, I’m grateful for that, but I was very confused at the time.

Cindy:

You mentioned the words consolidation and maintenance. Can you explain what the difference between consolidation and maintenance therapy is?

Josine:

Sure, consolidation is something that you do right before a maintenance program. It’s just eight weeks. They’re gonna try something to bring the numbers down even lower because, obviously, the stem cell transplant wasn’t as magical as they thought it would be for me at that time. So, I opted to do that just to give it a little boost to see if something else would work. And then the maintenance – I know Alan said he’ll be on it for three years. I think I’m gonna be on it indefinitely. I didn’t get an end date on mine.

Cindy:

And usually consolidation is more of a full dose of whatever treatment that you’re choosing to use as consolidation, whereas maintenance is usually a reduced dose or a reduced scheduling.

Mike:

One thing my myeloma specialist has said to me recently is that the line between consolidation and maintenance is kind of blurring now, and more patients are on sort of maybe in between consolidation and maintenance where you’re on treatment with more than one agent for an extended period of time. He’s told me I’m gonna be on something forever and ever for the rest of my life. So, it’s sort of hard to say whether it’s consolidation or whether it’s maintenance. It all just sort of blurs at this point.

Cindy:

Yeah, it’s hard to make that distinction. When does the consolidation end and the maintenance begin? At what dose level?

Alan:

That also shows how different we all are, and I’m in some different Facebook groups and support groups and things. And I see people asking what are the – how does the treatment progress, and what are the side effects of this drug and that drug. But we’re all different. And that’s the thing that is so important, even though there are some general guidelines – even in our treatments. I was classified as low risk. They got me into remission pretty quickly. So, things could change; but as of right now, there isn’t an end date to my maintenance treatments. I know people that didn’t do any maintenance. And then I also know people that probably won’t ever stop. And we were all treated at the same place.

Cindy:

It is very different from person to person, from treatment to treatment and even within yourself. Sometimes, you respond very quickly to one treatment and very slowly to another. So, that’s one of the benefits of being seen by a myeloma specialist, someone who only treats myeloma. Is everyone here being seen by a myeloma specialist?

Mike:

Yes.

Alan:

Yes.

Cindy:

Actually, I see two myeloma specialists – one and one for a second opinion, but I really do get my treatment locally. So, I’m very fortunate that all three of my doctors communicate with each other and work well with each other. So, let’s think about – if we had to make a treatment decision in the future and you were given two options, what kind of information would you like to know about each of those options before you make that decision?

I knew early on – one of the considerations I thought of after my stem cell transplant didn’t work was, I was still working at the time, and I was a teacher. And being a teacher, it was hard to take off from school to go to an infusion center to get my treatment. Or if I had to go several times a week, that just was not something possible. And I was trying to continue to teach.

So, one of things that I was considering back then was how the treatment was given. And one of the treatments that I chose was an oral treatment because that allowed me to continue to be employed. Eventually, I did retire. And that wasn’t as much of a concern, but back then, when I was still working and knew that it would be a conflict, that was something that came into my decision-making process. Anybody else?

Alan:

I would have to say for me the side effects probably would be the least important. I think I can probably endure a lot at this point in my life if it’s just temporary. I do have three businesses, and that would come into play as far as how treatments would go. My primary business as a financial advisor – I can pretty much do that from anywhere. In fact, even when I was going through my stem cell transplants, I always had my laptop with me. But since then, in the last year or year and a half or so, I’ve started two more businesses, and that would definitely come into play.

Cindy:

Does that – you were saying –

Alan:

And the other thing is I’d wanna know what the track record is, you know? Do we have a long-term track record?

Cindy:

So, you’re saying side effects for you would be least. But track record – are you talking more about the efficacy of the drug, how well it works compared to other drugs? What do you mean by track record?

Alan:

I would wanna know – I would be more willing to trust something that had a long-term track record of success than something new that we really just don’t know that much about. And that conversation actually came up with my doctor because there are a lot of new drugs out on the market right now. And he did tell me. He said, “Some of myeloma specialists are kind of getting away from the older drugs that we know work and going to these newer drugs.” He said, “I like to combine the two.” And that’s basically what he did. For my consolidation round, instead of doing a lower-dosed VDT-PACE of Velcade, dex, and thalidomide like they had historically done – for my consolidation round, they put me on the daratumumab. And I did 16 weeks of that, one treatment a week for eight weeks and every other week for eight weeks, and then I went on my maintenance.

Cindy:

Any other things that you would wanna consider or information you would want to know about a treatment before you make that decision?

Mike:

I think we’re getting close to the point where it’s gonna be important to understand a lot about the molecular basis of your particular form of multiple myeloma in order to be able to personalize the treatment. So, what particular mutations are driving your or my myeloma at this particular point? Because we know that changes over time, and what drugs are most effective against those mutations? I don’t know if we’re exactly at this point yet, but I think we’re getting close to that. So, when I relapse again, that’s something that I’m gonna be talking with my doctor about – exactly what mutations have I got and what are the best drugs against those mutations?

Alan:

I do agree with that, and I know the Myeloma Institute where I was treated they do genetic studies on every patient. I’m sure they do that in other facilities also. And I definitely agree that that’s where they’re trying to go. And hopefully, they’ll be there soon.

Cindy:

Finding a treatment that’s aimed at one of the mutations you have – the goal of precision medicine. That’s pretty exciting. Any other things that you might want to consider? How do find out about new treatments? There’s so many new treatments first that are FDA-approved and available. But there are also a lot of treatments and clinical trials. I know when I was newly diagnosed, I had no idea what was available to treat multiple myeloma. I didn’t even know if I had a choice of treatments. I just blindly followed my doctor’s orders. My doctor told me what he thought was best, and I said yes.

But now I know there are so many treatments. How do you find out information about them so that you can have that engaged discussion with your doctor?

Josine:

Selinexor – the newest one that was approved – one of the gentlemen in our support group has been on it for eight months on a trial. So, we watched him go from literally look like he’s dying to dancing the jig. It’s awesome. So, knowing people who are on that particular drug or whatever and then inquiring about it because I know Krissy is starting with that as well. That’s how we learn. And we just learn everything from the IMF. I learn on online and from our support group. Deena is an amazing support group leader.

Cindy:

Great. So, you learn information through your in-person support group –

Josine:

Yes.

Cindy:

– and through talking to someone else who’s been on that treatment. Other ways we could gather information about treatment options? Alan, do they talk about treatment options in some of your online support groups?

Alan:

They do. I see a lot of information about that. I’m an administrator on a Facebook group for a particular drug. So, I see a lot of people making comments about different treatment options that they’re doing and their success.

I probably don’t put as much research into this as a lot of people do. I’ve battled the fight of not becoming my disease is what I call it. You can’t overwhelm yourself with information. I’m a big picture guy anyway. I’m not an engineer type-like. My wife is. Her dad was a retired engineer. So, she wants all the little details. I just want the big picture. When I go in, they do my test. I just wanna know good or bad. That’s all I wanna know. I would have to say that talking to people that have the disease and their experiences probably has a bigger impact on me than anything. Because I believe that sometimes studies can be skewed, and I like personal knowledge.

Cindy:

Anybody else? Anyone actually go to the studies and read the studies or abstracts of the studies or ask their doctors about studies?

Mike:

I do.

Cindy:

I thought you would sneak up. You’re in my support group. I know you talk about those studies, so do I. Go ahead. Talk a little bit more Mike.

Mike:

I am the detail guy on that. So, I do read the studies. I’m on the institutional review board for the cancer center that I’m treated at. So, I get to see some of the trials even before they start. I’m fascinated by the disease and the science. If I take off my patient hat and put on my scientist hat, multiple myeloma is a really, really interesting disease. It’s a complex disease. It’s a complicated disease. And there’s a lot that we can learn about cancer in general by using multiple myeloma as a model cancer.

So, it’s fascinating to me to talk with my doctor about the research; and fortunately, he puts up with my dumb questions for the most part. So, to me, I just enjoy kind of understanding as much as I can about it. It gives me a sense of power. And maybe that’s an illusion, but it still helps. The more that I know, the more comfortable I feel about things. So, I do a lot of reading about it and keeping up with webcasts and so forth that are put on various foundations. And there are lots and lots of opportunities to learn. There’s a lot more to learn than I have time for, but it’s an interesting disease.

Alan:

Mike, you and I are exact opposites, and we’d make a great team. You know that, right?

Cindy:

Right.

Mike:

Yeah, even though we’re wearing similar shirts.

Cindy:

I’m glad we have such a varied panel today. It’s good having many different perspectives. Any other ways we educate ourselves about treatment options that are coming up?

Alan:

I wanna add also that I try to participate in events like this. I do quite a bit of public speaking. I’ve been asked to be a PACE ambassador for one of the pharmaceutical companies. So, I travel around, and I get multiple myeloma specialists all over the country. So, I’ve learned a lot through those conversations. And I think it’s important for us to do things like this to give back. We all know how scary it is when we’re first diagnosed. And if we can do something like this or help somebody that’s newly diagnosed, I mean we’ve done a great thing.

Cindy:

Right. I agree with you 100 percent. Being a retired teacher, it’s in me to help educate others because I really truly believe that knowledge is power. And there has to be just a variety of ways, whether it’s through teleconferences or online support groups or in-person support groups or mentoring, there’s just so many ways that you give back and help someone.

Alan:

To me, the first of the unknown is worse than the actual treatments.

Cindy:

Exactly. And being able to talk to someone who’s been in your shoes is the absolute best.

Alan:

Do you mind if I share something with you real quick?

Cindy:

Go ahead.

Alan:

I mentioned that I went fishing four or five weeks after my second stem cell transplant. While I was there, I got a phone call from one of my clients, and one of her good friends had just been diagnosed with multiple myeloma. And he was gonna be treated where I was. And I was able to – she wanted me to talk to him. So, I called him. I shared my story. I told him how bad I was. And he said, “Well, I’m nothing like that. They caught mine early.” But I said, “Well, you understand my situation was serious.” I said, “I’d like to tell you where I am now. He said, “Okay.” I said I’m in South Louisiana fishing with my dad and my sons.” And he said, “You’re kidding!” I said, “No, I caught a 30-pound fish last night. I’ll send you a picture in a minute.” And just to hear the change in his voice, the tone of his voice, to give him that encouragement that everything was gonna be okay –

Cindy:

Right.

Alan:

– was an amazing feeling. About a year and a half later, I was fishing again in South Louisiana. And I only go a few times a year at most. And I got a phone call, similar situation – a single dad with a 13-year-old daughter. And I got to share my story with them. I got her – I got them on speakerphone so they could both hear me. My daughter was 14 when I was diagnosed. I will never forget that – being able to talk to them and encourage them because we all know how important your attitude plays a part in our recovery.

Cindy:

Exactly. Thank you so much for sharing that very personal story. And I’m sure we all have a similar story of a way that we spoke to someone and probably made a difference at that point in their journey. So, we’re coming to the end of our program. What are some things that you know now that you wished you’d knew then about making treatment decisions? Anything that you know now that you wished you knew in the past?

Connie:

I think one of the things I’ve learned is not to self-diagnose. I think when – it took nine months to get my myeloma diagnosis. I had broken a rib previously. And so, after that race, I thought I had just broken another rib. And it got better and went away. And a couple of months later when I turned over in bed, it felt like a knife going through me and took my breath away. And then another two months went by. I thought – well, I just re-broke it. Another two months went by and same thing, turned on my side in bed at night, and it went another knife through me.

So, I just even competed in some other races that summer. And I didn’t quite feel like I could go as fast as I wanted to or I might really do some serious damage. And that’s when I knew I needed to see a doctor. And I did, and I got bronchitis. And they first treated me and checked out everything for heart and didn’t find anything. And then, when I got the bronchitis, I went back to the doctor. And that’s when they decided to send me to a specialist, and he just touched my sternum and realized it was deformed and said he didn’t even wanna touch it until he got some images. And that’s when he – after the images that he got, he referred me to the West Michigan Cancer Center for further diagnosis.

Cindy:

Josine, any final words of wisdom?

Josine:

Well, like Connie was saying about self-diagnosis. I thought I hurt my back at work. If I had only heard the words multiple myeloma in life growing up, which I never heard of it until diagnosis, I think it would’ve been less painful a journey to say the least. Well, you know, it’s all part of everybody’s journey. And we’re here today, and every day’s a gift.

Alan:

As far as things I wish I would’ve known; I wish I would’ve known how hard the battle would be after the major treatments. It took me a long time to bounce back. My immune system just wasn’t very good. I kept pushing myself probably harder than I should have, but it goes back to the same thing – is do things that you don’t feel like doing. Push yourself to do the things – there are days when I don’t feel like getting up out of bed, even today. For the last week and a half, I’ve been fighting a cold. I actually – Thursday before last, me and my oldest son took our four-wheel drives to an off-road event and camped out for the weekend. Of course, I got sick the day we got there. But we had a great time and made some great memories, and the price was well worth it.

And that’s kind of the attitude that I have. I know that there’s a price to be paid at times for the things that I do, but every day is just a blessing. Every day is an opportunity to have a positive impact on somebody else’s life, and every day is a day to make memories with my family and my friends and the people I love. And put your focus there.

Cindy:

Wonderful words.

Alan:

The way that I put it in my talks is – we will find in life whatever we look for. If we look for reasons to be sad and upset and depressed, we will find those. If we look for reasons to smile and be happy, we will find those also.

Cindy:

That’s true. Very good. I could listen to your words of advice all day long, but we don’t have all day. So, how about Mike. Do you have any final words of wisdom? Advice?

Mike:

I guess the thing that comes to my mind that I know now that I didn’t know at the beginning – there’s so much. I didn’t know anything. But I realize now that I’m not alone. I felt very alone at first, but I’m not alone, and I’m not alone in lots of different senses. One sense is that there are other patients and other folks who are going through multiple myeloma just like I am. And so, a forum like this is really important to be participating in. I believe participating in in-person support groups is important, online support groups.

So, just realizing that you’re not alone is a key thing. And another way that I’m not alone is I have a wonderful team of doctors and nurses and healthcare professionals working to make me as well as I can be. And then I also am very blessed to have great friends and family. So, just knowing that I’m not alone is a key, key thing.

Cindy:

I like that. I’m not alone. Very good. Well, we’ve come to the end of our time. I think we learned a lot of great information between each other, and I’m hoping that it’s gonna be very beneficial to the myeloma community. So, to our audience, thank you for joining us for this Patient Empowerment Network programming, Myeloma Patient Café. I am

Cindy Chmielewski. And remember that this fifth -grade teacher says, “Knowledge is power and is your best medicine of all.” Thank you very much for joining us.


Please remember the opinions expressed on Patient Empowerment Network (PEN) are not necessarily the views of our sponsors, contributors, partners or PEN. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Maintenance Therapy and Continuous Therapy in Myeloma: What’s the Difference?

Maintenance Therapy and Continuous Therapy in Myeloma: What’s the Difference? from Patient Empowerment Network on Vimeo.

Nurse Practitioner, Beth Faiman from the Cleveland Clinic, explains in maintenance therapy versus continuous therapy in multiple myeloma, which can sometimes be confusing.

Beth Faiman is a nurse practitioner in the department of hematologic oncology at Cleveland Clinic. More about this expert here.

See More From The Pro-Active Myeloma Patient Toolkit

Related Resources

What Does Remission Mean in Myeloma?

Relapsed and Refractory Multiple Myeloma: What’s the Difference?

Find Your Voice Myeloma Resource Guide

Transcript:

Beth Faiman:

I’d like to define the difference between maintenance therapy and continuous therapy. When patients have a stem cell transplant, they have a pre-therapy, the transplant consolidation is the second step, and then they have a maintenance to maintain that remission. For some people that don’t have a transplant, you can just stay on continuous doses of a therapy that’s very well tolerated. So, maintenance and continuous can sometimes be confused, but it’s — maintenance is lesser doses of something that got you into remission and continuous is just kind of staying on that same dose of tolerated medication.

How Side Effects Can Be Managed in Myeloma

How Side Effects Can Be Managed in Myeloma from Patient Empowerment Network on Vimeo.

Beth Faiman, a nurse practitioner specializing in multiple myeloma, discusses side effects in myeloma and shares what can be done to prevent or reduce these issues in patients.

Beth Faiman is a nurse practitioner in the department of hematologic oncology at Cleveland Clinic. More about this expert here.

See More From The Pro-Active Myeloma Patient Toolkit

Related Resources

Office Visit Planner – Myeloma

Discussing Treatment with Your Doctor: Key Questions to Ask

Diagnosed with Myeloma? An Advocate’s Key Advice

Transcript:

Beth Faiman:

In multiple myeloma, there are numerous side effects, but the most common side effects of treatment are oftentimes the lowering of blood count. So, for example, depending on which type of therapy you’re on, maybe it’s lenalidomide or carfilzomib or some others, you can get some lowering of blood count.

So, those blood counts need to be regularly monitored. Another side effect might be peripheral neuropathy. Now, that’s more common in drugs such as bortezomib or thalidomide.

And so, it’s important to look for that symptom and report if you have any numbness or tingling in your fingers or feet, or dizziness, or anything odd to your healthcare team. Because by adjusting the medication doses, then those patients can actually stay in treatment longer with better control.

Other things with the monoclonal antibodies, some of the newer drugs that are currently available will produce an increased chance of infusion reactions. Now, that’s only at the very beginning of the infusion. So, once patients have received that therapy,  they can feel comfortable to keep taking that with lesser chance of side effects.

And then, finally, many drugs with myeloma have an increased risk of blood clots. So, patients should stay active, keep well-hydrated, and know that they’re at an increased risk. Most providers will recommend a baby aspirin for all patients taking these drugs like lenalidomide, thalidomide, pomalidomide, and carfilzomib. And that’ll lessen their chance of blood clots.

The last thing I’d like to add in is an increased risk of infections. Myeloma is a cancer of the bone marrow plasma cells that are responsible to protect you from getting sick, and unfortunately, they don’t work. Many therapies will further weaken the immune system. So, getting a seasonal influenza vaccine, a pneumonia vaccine every five years, and making sure they take shingles prevention is a very effective way of keeping yourself healthy.

Key Considerations When Choosing Myeloma Treatment: What’s Available?

Key Considerations When Choosing Myeloma Treatment: What’s Available? from Patient Empowerment Network on Vimeo.

Beth Faiman, a nurse practitioner specializing in multiple myeloma at the Cleveland Clinic, shares tips for making treatment decisions and discusses the evolution of myeloma therapy in recent years.

Beth Faiman is a nurse practitioner in the department of hematologic oncology at Cleveland Clinic. More about this expert here.

See More From The Pro-Active Myeloma Patient Toolkit

Related Resources

The Benefits of Seeking a Second Opinion in Myeloma

Find Your Voice Myeloma Resource Guide

Overwhelmed By a Myeloma Diagnosis? The Key Steps to Take

Transcript:

Beth Faiman:

There are so many treatment options, and that’s one of the reasons why it’s so important for patients to at least seek an opinion once or twice with a myeloma specialist because treatment changes so rapidly. We have over 20 medications that are approved for the management of myeloma and so the patients need to figure out what’s important for them.

Oftentimes you think, father knows best or doctor knows best. And I hear from time to time that you’re the doctor, you should know what is best for me. But I say, “I understand what might be the best treatment for you in terms of response rate, but we have to balance quality and quantity of life. What are the things that you’re willing and your family’s willing to accept for treatment?”

Do you want to undergo a stem cell transplant which maybe takes you out of commission for a couple of months? Or take an oral therapy every day or an IV therapy intermittently? So, there are oftentimes more than one decision, and this is what we like to practice at my institution. It’s called shared decision making where you have a partnership between the patient and their caregiver, and the healthcare team and we work together to mutually decide what’s best for that patient.

So, sometimes just really trying to get that cure or eliminate the myeloma cell clone as best as possible might not be the right answer now, especially if you’re a single mom or a single dad or caring for a loved one. But maybe that might be a future goal. So, having that conversation is so important. And patients should feel empowered to be able to have that conversation with their healthcare team because if they don’t, then maybe they need to see a different doctor or specialist so they can feel comfortable with them.

I am so excited about all the new classes of drugs that are so — that are currently available. When I started managing myeloma in 1994 or 1995 there was only stem cell transplant and maybe melphalan or Cytoxan, and those drugs were not very effective in controlling the disease. I’m now able to mix and match treatments and give patients different opportunities to meet these milestones. You know, patients were so worried about not being here in two or three years, and now it’s 20 years later. So, forming those relationships and keeping them living healthy longer is so important.

We now have drugs available that can have the possibility of achieving what’s called minimal residual disease or MRD, where we’re eliminating in the bone marrow, the myeloma clone

That was unheard of five years ago even. So now we have the BiTE therapies and CAR T-Cell therapies, and some of the newer drug classes that will hopefully have a functional cure.

People ask me what a cure in myeloma is, and hopefully, we’ll have a real cure. But, living out your normal life span compared to people that don’t have myeloma, and really enjoying life as you do it. So, I always tell patients don’t forget about health maintenance and checking cholesterols, looking for secondary cancers, keep a primary care provider on hand because as a team, we can all work together, to have you live your best life as possible.

Evolving Approaches to Myeloma Treatment: Staying Up-to-Date

Evolving Approaches to Myeloma Treatment: How to Stay Up-to-Date from Patient Empowerment Network on Vimeo.

Multiple myeloma research is fast-moving and showing promise. Dr. Peter Forsberg, a myeloma specialist, provides an overview of the changing treatment landscape and shares resources for keeping up with the latest news.

Dr. Peter Forsberg is assistant professor of medicine at the University of Colorado School of Medicine and is a specialist in multiple myeloma. More about Dr. Forsberg here.

See More From The Pro-Active Myeloma Patient Toolkit

Related Resources

Find Your Voice Myeloma
Resource Guide

Staying Abreast of the Latest News in Myeloma: Tips from an Advocate

What is Personalized Medicine?

Transcript:

Dr. Peter Forsberg:

I think research in multiple myeloma remains a really active area. It’s been a major evolution over the past 20 years. Myeloma’s been one of the real success stories of modern oncology in terms of how much research has translated into improved options for patients.

But, many new things continue to evolve. It can be challenging to feel like you’re abreast of what’s going on. I think there are great resources for patients. Organizations like the International Myeloma Foundation, the Multiple Myeloma Research Foundation, or Leukemia and Lymphoma Society are good places for patients to start.

I also think that social media can be useful although those types of things can be a bit of a double-edged sword. I certainly find lots of things out via Twitter, and I think there’s a pretty active myeloma community in some of those areas, but you have to be a little bit careful about where you point your attention when you’re interacting with the internet. I think there can be lots of places where you might get less up to date or less thorough information and that can sometimes be concerning or challenging for patients So, I do think it is great that we have tools, but it is important to be thoughtful about how you approach them and trying to find good, reliable resources in that regard.

 I think there’s a lot of really exciting things on the horizon. That’s gonna include using tools that we have in better ways. I think we’re gonna be expanding our approaches to how we treat newly diagnosed patients. It looks like we’ll be starting to use four-drug regimens in patients with newly-diagnosed myeloma in the near future, hopefully with ever-improving results. We’re gonna be more cutting edge in terms of how we test and measure disease, using things like minimal-residual disease testing in different and expanded ways.

And then there’s a number of immunotherapeutic treatments especially that are looking very promising in relapsed myeloma.

That includes CAR T-Cell therapies, bispecific monoclonal antibodies, and antibody drug conjugates, all of them look like really promising approaches and really new things that hopefully in the not-distant future are gonna expand our toolbox for how we’re able to help maintain and improve life for patients with multiple myeloma.

Know What Your Doctors Know: Multiple Myeloma

This video was originally published by the National Comprehensive Cancer Network on April 15, 2019, here.

Experts discussed updates in the treatment of multiple myeloma during live webinars hosted by the National Comprehensive Cancer Network (NCCN) on April 15 and 16, 2019.

Get The Best Myeloma Care NOW: A Physician’s View

Get The Best Myeloma Care NOW: A Physician’s View from Patient Empowerment Network on Vimeo.

Advocating for yourself is critical when diagnosed with multiple myeloma. Dr. Peter Forsberg details the value of collaborating with your healthcare team on treatment decisions.

Dr. Peter Forsberg is assistant professor of medicine at the University of Colorado School of Medicine and is a specialist in multiple myeloma. More about Dr. Forsberg here.

See More From The Pro-Active Myeloma Patient Toolkit

Related Resources

Overwhelmed By a Myeloma Diagnosis? The Key Steps to Take

Should You Consider a Second Opinion? Advice from a Myeloma Advocate 

Find Your Voice Myeloma Resource Guide

Transcript:

Patient education and self-advocacy I think are critical in multiple myeloma. Myeloma is a complicated disease. Getting your head around it can be challenging. Beyond that we have more and more treatments. Treatments are fairly complex. Our goals can be pretty different patient to patient. So really, patient education can be a key to understanding that and removing layers of complexity from something that can be a little challenging to get into.

I think self-advocacy is also really important in that, sometimes you can feel swept up into a wave of what the next treatments are gonna be, what the next steps are. So, making sure you’re taking time to voice your opinions or concerns for yourself, to make sure that you’re not leaving stones unturned in terms of what your best options are, what the best next steps are, what treatments or testing might be available.

I think myeloma, maybe more so than even some other diseases because it’s such a unique type of cancer, one where patients are often dealing with it for many years… Making sure that there’s a good level of education that evolves over time can help make sure that the patients get the best out of their treatments; to make sure that they’re able to have the most fulfilling experience dealing with their cancer and with their cancer team, and making sure that they’re advocating to get all options available to them in the mix potentially.

I think patients are often very thoughtful about knowing that providers are busy and that clinic can be kind of fast-paced, but I want to make sure that they know that the last thing that they’re ever doing is bothering me or other members of my team when they ask questions. I think one of the keys to making sure that everybody is comfortable with the steps we’re taking with their myeloma is to recognize that it’s a team. And the patients and myself and other members of my team, you know I think that the goal is for all of us to be on the same page and to understand what we’re working towards.

So, I think that my philosophy about how best to take care of patients tis to try to make it as collaborative as possible. To make sure people understand what we’re doing and why. And to be all on the same page I think you have to feel comfortable to take a moment to say, “Why are we doing this?” or to voice concerns about what’s going on or what the next steps might be.

ASH 2018 – Latest News and Research in Multiple Myeloma

ASH 2018 – Latest News and Research in Multiple Myeloma from Patient Empowerment Network on Vimeo.

Dr. Amrita Krishnan, Director of the Judy and Bernard Briskin Center for Multiple Myeloma Research, City of Hope, shares the latest news and research that was shared at the ASH 2018 conference in the field of Multiple Myeloma.


Transcript:

Esther Schorr:

Hello to everybody.  This is Esther Schorr with Patient Power, and I’m here today at the 2018 ASH conference, the American Society of Hematology.  And I’m surrounded by, oh, 20-, 25,000 amazing researchers and clinicians who are studying hematological malignancies.  And I have about me today Dr. Amrita Krishnan.  Is that correct?

Dr. Krishnan:
That’s correct.

Esther Schorr:
And she is the Director of the Multiple Myeloma Program at the City of Hope in Los Angeles.  Thank you for being here.  So what I wanted to talk to you about today is what’s going on for myeloma patients.  What are the headlines from ASH this year?

Dr. Krishnan:
Good morning, Esther.  Thank you for the opportunity to talk.  I don’t even know where to begin.  There’s—every myeloma session has been packed, standing room only, which tells you obviously, number one, the advances we’re making and the enthusiasm regarding them.

I’d say the three biggest news really is obviously CAR-T cells in relapsed disease, and we started out just hearing about one CAR-T construct, the BB121.  Now we obviously are hearing many other companies presenting their results and other CAR-T constructs, which I think is very good for us because we can understand better both the technology as well as side effects and efficacy and understanding among different T-cell constructs.

The other big thing, I would say, antibody drug conjugates by specific antibodies.  And then the last but not least let’s not forget in terms of stem cell transplantation there was a big session this morning looking at new drugs in the maintenance setting, so specifically oral proteasome inhibitors.

Esther Schorr:
Oh, boy.  Okay.  So now I’m going to drill down a little bit from a lay person’s standpoint about what you just said.

Dr. Krishnan:
Okay.

Esther Schorr:
It’s a lot of alphabet soup.  So I know that you’ve been doing some work with the drug daratumumab (Darzalex).  It’s a mouthful, and I know that that’s a monoclonal antibody.  And can you talk a little bit about what the relevance is about that?  Because I think our audience has probably heard of it but doesn’t know what’s happening in that area.

Dr. Krishnan:
Sure, happy to.  So daratumumab targets CD38, which is a protein on the myeloma cell.  So it’s very specific in terms of attacking the myeloma cell.  Now, that protein is expressed in other things like red blood cells, but really it’s very highly expressed on plasma cells, sort of the myeloma cell per se.  So daratumumab was already approved for relapsed myeloma, both multiply relapsed as well as patients who have a first relapse in myeloma in combination with some of the other drugs we think about such as bortezomib (Velcade) or lenalidomide (Revlimid).

This meeting this year, though, the big excitement is in regards to using daratumumab in newly diagnosed myeloma.  So we already know it’s a very effective drug in the relapsed setting.  We’re familiar with the toxicity profile, and overall it’s quite well tolerated, and so now the question becomes if it’s such a good drug should we move it earlier in the course of therapy to get the maximum benefit.

Esther Schorr:
So it could be a first-line therapy.

Dr. Krishnan:
Exactly.  So it’s already approved in the first-line setting in combination with Velcade, melphalan (Alkeran) and prednisone (Deltasone), so VMP, but that’s not a regimen we use in the United States.  So there’s going to be an abstract presented Tuesday, so I can’t even tell you yet because it’s a late breaker, but we only know a little hint of it which is using daratumumab plus lenalidomide and dexamethasone (Decadron) in newly diagnosed patients.  It’s called the MAIA study, and that’s the one that we’re all waiting to hear to see is that going to establish a new standard of care in the newly diagnosed front-line setting.

Esther Schorr:
So that helps me to understand that, thank you.  So then are there other studies that you’re involved in that would be interesting for patients to know about?

Dr. Krishnan:
So there’s another study that actually was presented yesterday.  It’s called the GRIFFIN study.  That uses daratumumab in combination with sort of I would say a quote/unquote standard regimen in the United States, RVD or lenalidomide, Velcade and dexamethasone.  And what it asks again the same question.  If you add to our standard backbone another potent agent, does it even further improve the responses?  So what they presented on Saturday was very early data on 16 patients, so we need to wait more, but it just shows you the excitement around that.  And that data they presented really was around the safety and suggesting that it’s a well-tolerated combination with a very high response rate, 100 percent response rate.

Esther Schorr:
Well, that would be my question then just as a care partner myself is when you’re talking about doing those kinds of combinations of two, three, four drugs are you all looking at the combined toxicity of those things and the side effects?

Dr. Krishnan:
Oh, yes, absolutely.  So the MAIA study, for example, very specifically looked at the three drugs of daratumumab plus len-dex comparing it to the two drugs, lenalidomide and dexamethasone, so–and the same thing with the GRIFFIN study.  That also was randomized so half the people got daratumumab in combination, the other half just got the standard RVD.

And there was, to be fair, a lightly higher increase in side effects when you added the daratumumab, a bit more infections and a bit more blood toxicity, so lower white counts.  So it is something to sort of, you know, take as a note of caution too, when you add more drugs that you do certainly expect that you are going to get more toxicity.  And obviously it becomes does the benefit outweigh the potential risk.

Esther Schorr:
As usual.  So I guess the other question I have is where does stem cell transplantation fit in all of this, or does it?

Dr. Krishnan:
So obviously I have a somewhat biased opinion.  I come from City of Hope, which is the largest transplant center in California, and two things I could say in terms of myeloma.  So we do over 8,000 transplants a year in the United States for myeloma, so it suggests that it’s a standards of care backbone of therapy.

As a transplanter I would say transplant still has the longest track record in terms of remission length and even if you compare it to standard RVD chemotherapy you get a longer remission when you throw transplant into that mix.  And I think what will be of interest to us is further improving upon that by either different maintenance strategies or induction strategies, so new treatment before the transplant as well to further improve the outcomes of the transplant.

And then the other thing I should mention, this is not a study that is open yet but it’s a study that we actually had some meetings about through the BMTCTCN, so a cooperative group of transplant networks, trying to ask the question.  So this is a group—you know, I used to chair the myeloma committee, and I’m still on the committee—we try and look ahead.  Right?  So we say, what can we do as a strength of network of transplant centers that patients really need?  What is the question they want to ask?  And one of the unmet needs is high risk-myeloma.  So whatever we do right now, and there’s been data presented at this meeting too, we need to do better.

For those patients who have advanced stage of myeloma, high-risk cytogenetic abnormalities, the therapy we have right now is still not optimal.  And one of the things that we’re going to do that we’re very excited about is we’re going to open a study that we’re literally going to go home and start writing in January, using CAR-T cells after an autologous transplant for patients with high-risk myeloma.

Esther Schorr:
So that gives—that’s hope for patients that have not had any real viable treatments till now or durable ones.

Dr. Krishnan:
I think that durable is what we would say.  So we’re all very excited about that.  It’s going to harness our strengths as transplanters, our strengths in cellular therapy and CAR-T and moving it up front.

Esther Schorr:
Good.  Well, thank you, Dr. Krishnan for all the work that you and your associates are doing.  I know that it’s, especially for myeloma patients and their families, it’s so important.  So thank you.

This is Esther Schorr from San Diego at the ASH conference.  Remember, knowledge can be the best medicine of all.

On the Horizon for Multiple Myeloma

ASH 2018 Conference Coverage

Dr. Elisabet Manasanch, Assistant Professor Department of Lymphoma/Myeloma at The University of Texas MD Anderson Cancer Center, shares what’s the latest and on the horizon for Multiple Myeloma.

What Is the Value of Diversity in Clinical Trials?

Clinical Trial Mythbusters

Clinical Trial MythBusters: What Is the Value of Diversity in Clinical Trials? from Patient Empowerment Network on Vimeo.

Downloadable Program Guide

In this MythBusters program, Senior Vice President and Chief Medical Officer (CMO) of ASCO, Dr. Robert Schilsky, and 20+ year CML survivor, Mel Mann along with Cecelia Mann, will unpack some of the issues that have led to the lack of diversity in clinical trials and initiatives in place that are changing all of this.


Transcript:

Andrew Schorr:
And greetings from near San Diego, Carlsbad, California. I’m Andrew Schorr from Patient Power. Welcome to this Patient Empowerment Network program, the next in our series Clinical Trial Mythbusters, and this program is so important, discussing what is the value of diversity in clinical trials. And, believe me, you’ll hear it is so critical. We have to do better, and we’re going to discuss that over the next hour.

I want to thank the companies that have provided educational support through grants to the Patient Empowerment Network. They have no editorial control, but their support is welcome. And that is AbbVie Incorporated, Astellas, Celgene and Novartis. All right.

We’ve got a lot to discuss today, and we welcome your questions along the way. I want to first introduce someone who, like me, has greatly benefited from a clinical trial and believes that they are alive today because of their participation. And so joining us from Atlanta is Mel Mann along with his wife and care partner Cecelia Mann. Mel, welcome to the Patient Empowerment Network program.

Mel Mann:
Thank you very much.

Andrew Schorr:
And we’re going to hear more of Mel’s story in just a minute. I want to introduce a very prominent medical expert who joins us. He is the senior vice president and chief medical officer at really the largest cancer organization, the American Society of Clinical Oncology, and that is Dr. Richard Schilsky. Dr. Schilsky, welcome to our program.

Dr. Schilsky:
Thank you, Andrew. Happy to join you.

Andrew Schorr:
Okay. And are you in the Washington, DC, Virginia area?

Dr. Schilsky:
That’s where our organization is based, in Alexandria, Virginia, yes.

Andrew Schorr:
Thank you for being with us. I should mention that Dr. Schilsky has had a lot of experience related to trials. He was the head of hematology/oncology at the University of Chicago, which of course Chicago is a very diverse city, and the University of Chicago does a lot of research. And he also helped run a big research group that doctors from around the world are part of, and he did that for many years. So we’re going hear more from Dr. Schilsky in just a minute. But, first, Mel. So Mel, in the late ’90s you were dying, right, of chronic myeloid leukemia, correct?

Mel Mann:
Yes, I was.

Andrew Schorr:
Losing weight and being told that there wasn’t much to do, right?

Mel Mann:
Correct, yes.

Andrew Schorr:
Maybe a transplant. But you were in Atlanta and you went from doctor to doctor, but somehow you got to MD Anderson, a major research center in Houston. What did they offer you there?

Mel Mann:
When I first went out to MD Anderson they said they were going to put me on a clinical trial after clinical trial. And the first thing they did was increase my dose of interferon, and that was the medication, the standard therapy at the time, and then they tried different combinations of drugs, and eventually I started on different clinical trials.

Andrew Schorr:
Okay. And, Cecelia, you were there in Atlanta and he was scooting over to Houston, it’s not exactly around the corner. Why were you supportive of that?

Cecelia Mann:
I was supportive of that because that was the last chance that he had to a cure and for survival. So from the very beginning, whatever type of treatment he needed when he was flying around, whether he was going looking for bone marrow transplants, doing bone marrow drives, and therefore I was supportive of. We had a five‑year‑old daughter at the time, and so anything that Mel needed I was there to support him.

Andrew Schorr:
So, Mel, this is a happy story because here we are in 2018 as we do this program and you are with us when many people with CML at the time were not with us that long. Hopefully, a transplant could be curative, but a lot of people passed away. You were lucky enough to come back as they were rolling through different trials and there was a new one that opened up for a drug called Gleevec, a pill.

Mel Mann:
Yes.

Andrew Schorr:
What happened?

Mel Mann:
Okay. So in the summer of ’98 the Phase 1 Gleevec study opened up, and I went out to MD Anderson, and I was patient number two, and I started taking it at a low dose, and it was effective for me. And eventually they increased the dose and it started changing my leukemia around to eventually I reached what they call a major molecular response. And that was 20 years ago. This summer I went over 20 years.

Andrew Schorr:
Wow. Well, Dr. Schilsky, is that an example of a patient getting, if you will, tomorrow’s medicine today, what we hope for?

Dr. Schilsky:
Absolutely. And, first of all, it’s such a wonderful story, Mel. It’s great to hear you tell it, and it’s exactly why we do research, exactly why we do clinical trials, to try to discover the new therapies that people need that will give them the kind of long‑term survival and quality of life that you’ve been experiencing. It’s just‑‑it’s wonderful.

Andrew Schorr:
So, Dr. Schilsky, let’s get into the problem. So, generally, there are many clinical trials that take longer than one would hope to fill, and the FDA and I know scientists have been seeing well, gee, how do we know what we’re testing applies to people maybe with different ethnic, gender backgrounds, a variety of situations, and often we can’t find people who fit those categories to be in. What is that‑‑how poorly have we been doing in the past with diversity in trials, and what does that mean for developing new medicines?

Dr. Schilsky:
Well, we don’t do well in almost any dimension. We don’t get enough underrepresented minorities in clinical trials. We don’t get enough older people in clinical trials. You have to remember that 60 percent of cancers occur in people 65 years and older, and yet only about 10 percent of people participating in clinical trials are 65 and older. So we are having to treat the majority of older people, and I would say the majority of minority people, with data derived from participants in clinical trials who are not like them.

We need to change that for a whole host of reasons. It’s historically been very challenging, and the problems really sort of boil down into three big areas that I think we can discuss a little bit further.

First is awareness. Many people are not aware that clinical trials are even an option for them. Many people think that a clinical trial is a last resort, and I want to dispel that myth right out of the box. Clinical trials can be a very good option for patients right from the time of their cancer diagnosis even if it’s their very first treatment. So clinical trials may be a last resort, but they don’t have to be, and there are many clinical trials that are appropriate for people right following the initial diagnosis of their cancer.

So there’s the awareness issue, and sometimes, frankly, not even the doctors are aware of what clinical trial options are for their patients. And the one thing we know for sure is that the most influential person as to whether or not a patient goes into a clinical trial is their doctor. If the doctor does not recommend it, if the doctor is not aware of it, it’s not going to happen.

But then you get into the more technical issues. There are things, there are rules for clinical trials because they are research studies. They are experiments. There are very well defined rules, most of which are in place to protect the people who are participating in the study. Some of these rules are called eligibility criteria, and they specify the characteristics of people who can enroll in the study. Well, historically, they tend to be very rigid and very limiting, and you’ll often hear people talk about how the only people who can get into clinical trials are Olympic athletes. That may be the case, but it’s not Olympic athletes that we’re treating in the clinic every day, so we need to make our clinical trials more representative so that they’re more applicable to the typical person that a doctor sees in their office.

And then there are the logistical or operational issues of the clinical trial. The clinical trial can be very burdensome. Mel just described how he had to travel from his home in Atlanta to Houston to participate in a clinical trial. Not everybody can afford to do that. Not everybody can take time away from work, time away from home. And the clinical trial requires not only that you travel sometimes but that you travel on a specific schedule because of the requirements of the trial.

So all of these are issues that are‑‑can limit participation in trials, and many of them are magnified in minority populations or in populations that don’t have the economic resources to be able to meet the requirements of the trial.

Andrew Schorr:
Right. Let’s talk about that for a minute. So, Cecelia, you go out in the community and speak to people, and you probably meet some people who maybe are diagnosed with a blood cancer, like you’re active with the Leukemia and Lymphoma Society so you may speak to them, and they say even if you brought up about a trial, they say, hey, Cecelia, I’m working two jobs. Or my husband or spouse is working two jobs, and we’ve got two kids, three kids. How could we ever participate? We just can’t get away or we don’t have the family support or whatever. Those are real issues, right?

Cecelia Mann:
True. True, those are real issues, and I try to direct them towards resources that Mel and I found out about along the way. The Leukemia and Lymphoma Society, they have resources to help with travel, and American Cancer Society has resources that help with the hotel and lodging. And there are a number of other different types of funds that can be assessed to make that a possibility.

But you’re right, Andrew. I had one young lady at a health fair and a second cancer had popped up, and she was coming there to get information, and she was saying that they were saying it wasn’t too much they could do, and I brought up MD Anderson. And she immediately said, I can’t go out there, I have to go to work, and she turned around rough. And so when they listen to me then always glad to tell them about it and let them know there is an option and that clinical trials work, and I point to Mel, my husband, as a success story also.

Andrew Schorr:
So, Dr. Schilsky, you talked about physician awareness. It’s also about more physicians participating in trials at I think what you call the community level. So, in other words, MD Anderson and where you work, at the University of Chicago, those are big city centers and where they are in Atlanta there’s Emory and some other mainly centers, but what about out in the hinterland, if you will? Can somebody who lives there diagnosed with a cancer, how do they have access to a trial that their doctor knows about and maybe that’s more close to home, if you will?

Dr. Schilsky:
Right. So of course we know that anybody with cancer prefers to be treated in their community, and most are. So one of the goals is to be sure that oncologists practicing in all sorts of community settings have access to clinical trials. Now, one of the ways that happens is that for more than 50 years now the National Cancer Institute has actually been operating and funding a community‑based clinical trials network. It used to be called the CCOP program. That’s an acronym that we don’t have to go into. They’ve recently changed the name. It’s now called NCORP program, but‑‑that stands for, I think, the National Community Oncology Research Program.

But the point is that the program, which is in most but not all of the states in the United States, funds community oncologists to participate in NCI‑sponsored clinical trials, and there are at least 65 or 70 such clinical facilities around the country right now. So in those medical practices patients can find those clinical trials in their communities without having to travel.

There are also other community‑based networks that are active participants in cancer clinical trials. So I think at the end of the day the critical thing for patients, and this is sometimes easily forgotten because you’re so, you know, your thinking and your time and your emotion are all taken up in dealing with the cancer diagnosis. It’s really important, though, to ask the doctor, do they have access to clinical trials. Do they have a clinical trial that might be appropriate for you? And if not you might want to consider where else you could go, hopefully still relatively nearby to get access to a clinical trial.

Andrew Schorr:
Mel, so for you, you went around to some doctors who were not aware of anything new to do for you, right? And that’s still true in so many areas of cancer. Now, what do you say to patients about speaking up because Dr. Schilsky just referred to it, people are terrified. They really just want the doctor to have the answer. What do you tell people so that they maybe advocate for themselves?

Mel Mann:
Okay. So if you’re looking for a clinical trial and I’m out at, like you say, a health fair, we have a‑‑the Leukemia and Lymphoma Society has something called the clinical trial support center, and they have nurses who work early in the morning till late at night. And you call them up and you tell them about your illness, and they check the availability for what clinical trials are out there, and then they narrow it down to what you actually qualify for, and then they take into consideration your finances and other issues.

And then you’re left with a number of possible clinical trials that you have, and you can take that back to the doctor and you can discuss that with him. So that’s one of the things I talk with them about.

Andrew Schorr:
I could mention, now this is really more broadly across cancer, too. So there are breast cancer groups, there are lung cancer groups, and I would just make a pitch to find out, is there a local chapter or national number for you, for the cancer you or a loved one been diagnosed with, and that’s the question.

Mel Mann:
Yes.

Andrew Schorr:
Say, look, A, I don’t want to feel I’m alone, and, B, how do I get‑‑how do I get connected with what could be lifesaving or life‑extending treatments for me and that I could discuss with my doctor. And understanding‑‑and then, boy, if there are obstacles like financial issues, logistical issues, travel issue, is there support for that.

Mel Mann:
Yes.

Andrew Schorr:
So let’s go back to the inclusion/exclusion or eligibility issue you spoke about, Dr. Schilsky, because, you know, somebody who has cancer may also have heart problems or diabetes or some other issue. Maybe they previously had another cancer, and so for the companies developing new drugs they may be happy with narrow inclusion criteria because they don’t want to have anything get in the way, some previous thing you’ve had, to affect their ability to have a new drug go on the market.

So what kind of work is going on between government and the drug manufacturers so that the criteria, not so tight, but you can still get legitimate scientific answers?

Dr. Schilsky:
Right. So, as you alluded to, Andrew, there are good reasons that there are eligibility criteria. One of them is to protect the patients in the study from circumstances that would increase their risk of participating in the study. Another is because the companies or whoever is sponsoring the study wants to be able to isolate the specific effect of what they’re studying without having a lot of confounding factors that could muddy the water and makes it difficult to interpret the results. But that said, the bad thing about all that is that the results of the study might not be applicable to the majority of people who could benefit from the treatment because they weren’t included in the study to begin with.

So one of the things that my organizations has been working on very hard over the last couple of years now, and we’ve been doing this collaboratively with people from the Food and Drug Administration and the National Cancer Institute and a lot of clinical experts and a patient advocacy group, Friends of Cancer Research, is to try to expand or broaden or simplify some of these eligibility criteria that tend to keep people off of trials and in particular tend to keep minority populations off of clinical trials.

So, for example, it’s not unusual for someone who has a new diagnosis of cancer to have previously had some kind of cancer earlier in their lives. So we might see a patient who has lung cancer who 15 years ago had a diagnosis of prostate cancer. Well, for that lung cancer patient to go on a trial that has the typical inclusion and exclusion criteria that doesn’t allow for this previous malignancy, they would be excluded even if they had been cured of that prostate cancer 15 years ago.

We also see, and you mention what we call in the medical profession comorbidity. So if someone’s got cancer and they also have heart disease, they also have diabetes, high blood pressure, anything that affects the functioning of your normal organs, can also exclude people from participating in trials, and there are certain limits that we feel can be expanded and still allow the treatment to be given safely.

So just about a year ago now we came out with a set of recommendations for how eligibility criteria can be modified to make clinical trials more inclusive. And now just recently, I’m really pleased to say, the National Cancer Institute expanded their sort of template protocol document that many investigators follow to incorporate our recommendations, so now their standard protocol includes these broader inclusion and exclusion criteria. And the FDA now is working on what they call guidance documents to advise commercial companies that are running clinical trials to do just the same thing. So we are very optimistic now that we’ve got this ball rolling. We’re going to be removing these obstacles, and we’re going to be able to have more inclusive and diverse population of patients who participate in cancer clinical trials.

Andrew Schorr:
Great leadership. I hope it works great, and we’ll be happy to support you. So, Mel and Cecelia, let’s talk about the money part of it a little bit. So you were making trips to Houston, Mel, Cecelia was home with a five‑year‑old, and so admittedly there may be hardships, financial hardships, being away from family if you have to go to a trial somewhere else, checking back. What do you say to people when they say, well, I’m just going to go with the traditional stuff. It’s close to home. In other words, if there can be programs that can help them it still takes courage, if you will. So what would you say to people about investing in their life, if you will?

Mel Mann:
Well, you know what the standard, what the current treatment is and the outcome of that, so if you want to have a different outcome then you have to try something new which is probably going to be a new drug. So you have to weigh that with the cost and the travel. Some people may not have the support, the caregiver support to go a long distance, so you have to take that into consideration. As far as the Leukemia and Lymphoma Society they do have certain funds where they can help with travel pay, co‑pay, insurance premiums, that could help alleviate some of it. So there’s a lot of stuff that’s involved, and it’s an individual decision.

Andrew Schorr:
So we’ll just make a comment, though. So, many people have a church or synagogue, friends, neighbors even if they’re living alone, but yet people are sometimes hesitant to ask for help. And I would say speak up. People do want to help you.

Dr. Schilsky, let’s talk about another reality of trials. There’s a history certainly and some fear still in the black community of whether they were tested on, without their knowledge even, going back years and the general thought, you’ve heard it through your career, I’m sure, people say, well, I don’t want to be a guinea pig for a couple reasons. One is we don’t know if it’s going to work. And second of all if there are different arms of a trial I don’t know if I’m going‑‑I’m going to go to all this trouble and expense, I don’t know if I’m going to get the good stuff. So maybe you could speak about that a little bit. First of all, the fears of being experimented on, and then also about whether you will get what could be a breakthrough.

Dr. Schilsky:
Yeah. Well, for sure, you know, there is this sort of sordid history of inappropriate experimentation on people, and clinical trials are a form of experimentation. They are a form of research. There’s no doubt about that. But clinical trials these days are highly regulated, overseen by independent groups that include patients and clinical experts that come together in committees called IRBs, Institutional Review Boards, and they evaluate on both the risks and the benefits to patients who participate in clinical trials.

They make sure that the trial has an appropriate consent process associated with it, that it’s explained in plain language to patients, so I think these days a lot of those concerns no longer exist. And I hope that people can get beyond the history that led to some of those concerns. The‑‑sorry, I lost a train of thought on the rest of your question.

Andrew Schorr:
The issue about are you going to get the good stuff.

Dr. Schilsky:
Oh, yes. So a couple points there. One point I want to make clearly is that in most cases cancer clinical trials do not include a placebo or an inactive treatment. That’s not always the case, but it’s true most of the time. So patients are always going to get at least the standard of care treatment, and of course the standard of care is what is at that time known to be the best available treatment.

The whole point of doing the research is to determine if the new thing is better, and of course we always hope it will be. It’s not always better, but sometimes it is, as in Mel’s experience. And I think this has to be clearly laid out to patients. They have to clearly understand why the research is being done. In many trials nowadays even if the patient is assigned to get the standard of care treatment there still may be an option to get the new treatment at a later point. So if the standard of care doesn’t work many times there’s still the opportunity to get the new treatment following the standard of care treatment.

So the trial really boils down to not standard versus new but new versus standard followed by new. So eventually everybody may have a chance to get the new treatment. That’s not always the case, but I think the key‑‑my key take‑home, in a sense, is that we’re doing the research because we think and we hope the new treatment is better, but we have to do the research to prove that. And everybody in a clinical trial I think can be assured that they’re going to get, at the very least, the best available standard treatment.

Andrew Schorr:
Mel, when you signed the papers to be in a trial, and you probably shared them with Cecelia, especially back in the late ’90s and I participated in one trial in 2000 and another in 2011, there’s a lot of paperwork, things in bold face written by lawyers. I didn’t always understand it. What propelled you beyond that? Was it just that, oh, my god, if I don’t get something I’m going to die? Or how did you two deal with the paperwork and feel comfortable signing on the dotted line?

Mel Mann:
Well, I saw a lot of hope in the paperwork. For example, one trial I was on was peginterferon, and I had been taking interferon every day, injecting myself, and I had to keep it refrigerated and when I travelled it made it difficult. So with peg I can take one shot a week, so that would make the cancer journey easier. It may not make me live longer, but it will improve my quality of life, so I saw my quality of life improving with that clinical trial. And I looked at the paperwork, and I went through it, and I felt comfortable with it.

Andrew Schorr:
And how about you, Cecelia? I mean, your husband says, well, I’m going to be in a trial and I’ve got to sign all these papers. Did you say at any point, wait a minute, that’s scary?

Cecelia Mann:
Well, no, I didn’t. I didn’t because with Mel, he had three years to find a marrow match, and he was at the end of year two and no match in sight. And so when he had the opportunity to go out to MD Anderson and be on a clinical trial or several, I was okay with that. I was okay with that. And I looked at it as actually being a blessing. And it turned out to be, and we’re grateful.

But I would say to anyone else who is contemplating a trial and that person and their caregiver, their spouse, to just educate yourself, and get as much information as you can, ask as many questions as you can, but please don’t just throw it away out of hand. It’s definitely worth considering.

Andrew Schorr:
Dr. Schilsky, so we have more than 50 million people with a Hispanic background in the United States, and even if many people are speaking English they may speak Spanish at home. And then when you are diagnosed with a cancer there’s a whole new language of stuff that comes into play that even if you’re fluent in English it may not be either what you easily understand or even aligns‑‑what’s being asked of you aligns with your cultural background. Okay? So how, beyond, let’s say, the African‑American community, when you look at the Hispanic community, how do we encourage participation there and get over some of these cultural or language nuances, if you will?

Dr. Schilsky:
Yeah. So it’s much the same thing in the sense that the same information has to be conveyed but it may have different meaning and different interpretations in different ethnic and cultural groups. Most clinical trials now will have a consent form that is fully translated into Spanish. But, of course, there are many different languages on the globe. When I was practicing at the University of Chicago for many years on the south side of Chicago, we had Polish‑speaking people, we had Russian‑speaking people, we had people‑‑Chinese‑speaking people.

So the requirements actually are that there must be a consent form, at least some reversion of which is translated into the first language of the patient. So if you’re a native Spanish speaker, a native Chinese speaker, you have to have, be able to see a consent form written in that language, and generally speaking you have to have your native language interpreter present in the room to help you go through the consent form and respond to your questions. And that person has to be someone who is independent from the research team so they can give you the straight answer and not be influenced by any member of the research team. So I think all of that certainly helps.

But, again, what helps a lot more is to have members of the care team who look like the patient. So we have problems with diversity in our profession as well. We have very few African‑American oncologists. We have more Spanish‑speaking oncologists, but again we have few Asian oncologists. So we need to do a better job of improving the diversity of our profession, improving the diversity of the care teams. We need nursing staff and research staff and other people who work with our patients who represent them and gain their trust, who look like them, who talk their language. And I think that will go a long way toward making people feel more comfortable about participating in clinical trials.

Andrew Schorr:
I was at a conference last week and I heard some of the patient experience, people from different drug companies talking about how they were trying to simplify their forms because I know in 2000 when I entered a Phase 2 trial there were all kinds of black boxes, you could die, you could this, everything in the kitchen sink was in it. I’m still here, and I think because of the trial, and most of the side effects I didn’t have or they were definitely handled extremely well.

So right now, where are we, Dr. Schilsky, with participation? And why is it important? In other words, in this age of personalized medicine why do we need more black people in certain trials? Like, I know in multiple myeloma, one of the areas we cover, there’s a higher incidence in the black population, right, but yet few black people are in the trials for myeloma drugs. Or maybe there are differences with Asian populations or other populations. So is it that you can’t really get a clear scientific answer on the differences? Is that it?

Dr. Schilsky:
That’s part of it. First of all, we want anybody who could potentially benefit from being in a trial to be able to be in the trial for their own personal benefit. Secondly, we need to learn about the performance of the drug or the intervention in all the diverse populations in which it might be used. And one of the things we have learned is that not all populations respond the same way. Some treatments are more toxic in certain racial or ethnic groups. Some are more effective in some racial or ethnic groups.

And, you know, since you brought up this whole new world of precision medicine, I’ll give you the example of the lung cancer drugs that are used to treat the specific mutations in a gene called EGFR. So that’s a gene which has mutated in about 15 percent of Caucasian patients with lung cancer, but it’s mutated much more commonly in Asian patients. And in fact one of the clues that there was even a gene mutation that was important in determining whether these drugs worked or not was because it was observed that the drugs worked better in the Asian patients in the clinical trials even before the genetic abnormality had been discovered. And the clue was what’s different about the Asian patients than the other patients in the trial.

So the diversity is critical to our learning and critical to our application of the therapy in all the diverse populations that we serve.

Andrew Schorr:
If you’re in our viewing community and you have a question, send your questions into questions@patientpower.info, questions@patientpower.info. We’ll continue our discussion of course, but we invite you to join in.

So, Mel, when you get to talk to people, what do you say? Somebody is sick, diagnosed with a cancer, what do you say? Dr. Schilsky was talking about not seeing clinical trials as a last resort, and you weren’t seeing it that way (?) Inaudible, but today what would you say to people when you talk to them about it?

Mel Mann:
Well, I will say explore your possibilities because there are all different opportunities at each phase. You may not go into Phase 1 but you could do a Phase 2, 3, 4‑‑or Phase 3, and you don’t know what’s going to happen in each of those phases. So you just have to see what’s out there. And I’m exhibit A, so they look at me and they say, well, I can work, and then not as suspicious, you know. We have Tuskegee, and that was 1972, and it was that dark period of cancer history so that kind of rolls around in their mind, but you can’t let that jeopardize the opportunity such as Gleevec that I took advantage of. So we know that Gleevec worked, and there are other drugs that have improved the quality of life and the lifespan of cancer patients. So definitely research those drugs.

Andrew Schorr:
Did you lose heart when you were first in one trial and the medicine wasn’t working for very long? Some would say, well, all right, I tried a trial, forget about it, you know. But you then pursued other trials. What propelled you to do that?

Mel Mann:
Well, I was still in the game, so I saw that these trials took‑‑well, first of all, I could not find a bone marrow donor, but a bone marrow transplant was pretty drastic in itself so I was looking at these other opportunities as maybe not even having to take part in‑‑have a bone marrow transplant. So that was another incentive. So‑‑and I knew that if I didn’t find one‑‑there was a very small chance, there was only about 5 percent of Americas who are on the marrow registry, so basically I was helping to build a list, maybe not for myself but for people in the future who needed a transplant.

Andrew Schorr:
Dr. Schilsky, let’s talk about the pace of research. So, first of all, if we don’t get enough participation in trials how does that slow drug development?

Dr. Schilsky:
Well, it slows it down enormously because we have to have a certain number of people in each trial to be able to get a reliable answer. And these days it’s becoming even more challenging because as we’re developing drugs that only target a specific genetic abnormality in the tumor which sometimes is very rare so we may be looking for a genetic abnormality that only occurs in 2 or 3 percent of all people with a certain kind of cancer. First you have to find the people who have that genetic abnormalities, then you have to be able to enroll them in a clinical trial. They have to be willing. They have to meet the enrollment criteria. So it can take a long time, and even a global effort to find enough people to fill out a clinical trial.

And most clinical trials in order to produce a reliable result are going to require a minimum of 50 to 100 patients. Some require many hundreds of patients or even many thousands of patients depending on the question being asked. So you can see if people are not participating it’s going to take long time to get those answers.

Andrew Schorr:
Now, Mel, you got Gleevec in a trial at least three years before it was approved, and it was approved fast because it was such a breakthrough, right? So you literally got tomorrow’s medicine today, and it saved your life in the process, right?

Mel Mann:
Yes, because I was past the three years. I was about three years and eight months in my diagnosis, so you add another three years onto that and I would not have been here.

Andrew Schorr:
Right. In my case I was in a trial related to chronic lymphocytic leukemia, three‑drug combination, and I received that in a Phase 2 trial 10 years before that was approved. So it was a long time.

So I have a question for you about personalized medicine, Dr. Schilsky. So for instance in chronic lymphocytic leukemia I’m not‑‑I’m Caucasian but I’m Ashkenazic Jewish, okay? So where we’re going with personalized medicine, are we beginning to find subsets among Caucasians, among African‑Americans, among Asians, where there are even more narrow slices to help us understand targeting of medicines and what’s effective for whom? Is that where we’re headed?

Dr. Schilsky:
Absolutely. And, as I said earlier, we’re seeing that all the time. So almost every common cancer now is being broken down into a whole basketful of rare cancers under the broad umbrella of whatever the cancer type. So lung cancer, there’s probably six or eight different kinds of lung cancer now that each have a specific genetic abnormality, that each requires a specific treatment. And many of those treatments now are FDA approved, but the first thing you have to know is does the cancer have the genetic abnormality and then what is the appropriate treatment to use. We’re seeing that in breast cancer, in melanoma, in many other kinds of cancer types.

There’s another‑‑there’s a related issue here, though, of course, which is that not everybody metabolizes drugs the same way, and so another reason to have diverse populations in a clinical trial is to learn about side effect profile of the drug, learn about the right dose of the drug to use. And we know full well, for example, that African‑Americans metabolize some drugs differently from white people, and so, depending upon how the drug is working in the body, an African‑American person might require a higher dose or a lower dose of the same drug that a white person would require in order to get the same therapeutic effect.

So it just speaks to the point again where if you don’t have diverse populations in the trials you can’t learn this stuff so that doctors then have the information they need to be able to prescribe the drug in the best way for their particular patient.

Andrew Schorr:
Okay. Here’s some questions we’ve been getting in. Kaitlin wrote in, Mel, she wants to know, do you still participate in follow‑up activities related to the trial you were in? So tell us about your participation and sort of follow‑up.

Mel Mann:
My follow‑up is I go out to MD Anderson twice a year, and it’s just a one‑day, one‑hour doctor visit where they take the blood work and they check and see if everything’s stable. And then when I’m back home, twice a year I have my blood work checked back at home, and that’s the extent of the follow‑up. I still have to take medication, one pill a day.

Andrew Schorr:
Right. And is that still covered by the trial?

Mel Mann:
Well, it’s covered by the trial, but my insurance also covers it. I did Gleevec for life because of the trial.

Andrew Schorr:
Okay. Dr. Schilsky, let me just ask you, is that a benefit typically of trials? Like with these oral cancer medicines which you know can be so expensive, if you’re in a trial for one do you get it for life or an extended time or how does that work?

Dr. Schilsky:
Depends a little bit on the trial and the sponsor for the trial, but the one thing for sure is when you’re on a trial whatever the investigational drug is, whatever is being researched, that’s provided for free. And any testing that would be considered to be for research purposes is provided by free‑‑for free. So that’s a benefit of participating in the trial.

Typically the drugs continue to be provided for free for as long as the patient continues to benefit. Now, sometimes if the drug ultimately gets FDA approved then it may be necessary at some time in the future for a patient to switch over from the research drug to the commercial drug, but of course at that time the drug is FDA approved and if the person has insurance it will generally be covered by their insurance.

Andrew Schorr:
Okay. We got a question in though for you, Dr. Schilsky, from Darrell. We were talking about genomic testing to understand what version of a disease we have either because of our ethnic background or some other thing that’s going on with us. As you know, insurance companies for a while have been balking at some of these more sophisticated tests, yet we and our doctor need that for us to get what may be right on target for us. So maybe you could talk about work that ASCO’s doing at all related to that. We want the testing done, but we also want to get it paid for so we can get that right, precise care.

Dr. Schilsky:
Yeah. It’s a complicated question because the testing is done at different points in the person’s illness. And so typically a test on a tumor specimen that’s necessary to determine a standard of care treatment, and many of these tests are referred to as companion diagnostic tests. Those tests typically are covered by insurance because the treatments themselves are also covered by insurance and the only way to know if you can get the treatment is to have the test done.

Now, where it gets a little bit uncertain is when you get into this sort of large‑scale genomic testing where a patient’s tumor might be tested for many hundreds of genes not really knowing what you’re looking for and not really knowing what you’re going to do when you find it. And that is where you’re beginning to bump up a little bit against, research and that’s where the insurance companies, sometimes some are reluctant to pay for that kind of testing.

Now, at least one of those large genomic profiling tests earlier this year was approved by both the FDA and Medicare and now will be reimbursed. So that’s the good news, and I think that’s the direction that most insurance companies are heading in.

One of the things that my organization is doing to try to understand how best to use these tests and how best to use targeted cancer drugs is we’re doing our own clinical trial that’s available in 20 states around the country, so not the entire country yet, but has already enrolled more than 1200 people on the study over the last two and a half years. And we’re doing this study to understand how this kind of genomic testing is done, what kind of treatment is recommended based on the results of the genomic test and whether or not that treatment actually works.

Andrew Schorr:
Cecelia, you mentioned earlier about the lady walked into the health fair and when you started talking to her about trials she said I got to go to work, and she walked out. And Dr. Schilsky was talking about eligibility requirements, but there are other issues where the study may be asking you to come back to some place or have multiple tests with some frequency so it’s not just leaving work one time. It may be leaving work 20 times. Have you had people voice that concern to you, that it’s just‑‑what’s being requested is just too much?

Cecelia Mann:
Yes. I think the lack of convenience for people who don’t have the funds or don’t search out the funds would definitely hinder them being on a clinical trial or being open to hear about the clinical trial. So, yeah, convenience and awareness. We try to spread awareness that, yes, after we talk about the disease, the myeloma and the symptoms then we go into the resources. And, you know, I make sure I tell them about calling the information line and talking to the masters level oncologist professional there and finding out about the latest trials, the latest treatments in addition to what they’re doing currently with their doctors or their family members or friend that is diagnosed with one of the blood cancers.

Andrew Schorr:
Dr. Schilsky, she’s getting at helping people sort out what trials are available. So medical science is a moving forward, and I’m sure you as an oncologist say, hallelujah, there are more trials than ever, but it’s often not only daunting to understand any one of them but to go through a bunch of them to understand what might be right for you. So how can ASCO help that? Is it just simply educating your doctor, or what can we do for families affected by cancer so they can get at what could be a match for them?

Dr. Schilsky:
Yeah, it’s a great question. So there are a lot of resources available, as Cecelia has mentioned. We can help patients understand and even begin to sort of, you know, wade through and winnow down the potential clinical trial options for them. One things that we’re working on and very interested in doing is sort of flipping the current paradigm by which trials are done. So right now, typically speaking, the patient has to travel to whatever site has the trial available. If they’re fortunate enough that that’s their own doctor’s office, that’s great, but, as in Mel’s case he had to travel to MD Anderson to get the trial.

The technology these days is at a point where we think we can flip that. Instead of making the patient go to the trial we’re going to work toward making the trial come to the patient. So if your doctor is aware that a trial exists somewhere in the world it should be possible for the doctor to basically just go to a website, find the research study, find the consent form, find the other documents that are necessary and present that to the patient. And if the patient qualifies just sign them up instead of making the patient travel hundreds of miles away to the one place that has the trial available.

Some trials have already gone down this road, and they’ve actually been recruiting very successfully, but it’s still not the usual way in which things are done, and we think we need to try to flip things around a little bit to make it easier for the trial to come to the patients. Let the trial travel. Let’s not make the patient travel.

Andrew Schorr:
That sounds great.

Mel Mann:
Can I add something to that, Andrew?

Andrew Schorr:
Sure.

Mel Mann:
Okay. As a veteran, I was part of the VA system, and I had to go out to MD Anderson, but this summer they started something called Navigate, the VA had started something called Navigate, which partners with the NCI. And it’s at 12 sites around the country, and it’s to bring the clinical trials to the VA. It’s right there. So if it’s an NCI clinical trial then the veteran can get on that clinical trial. And there’s a lot of African‑American veterans who can take advantage of that.

Andrew Schorr:
For sure. That’s terrific. So we’re going in the right direction. One other thing I think that needs to change is we talked about the scientists, whether they’re federal government scientists or drug company scientists, and they want to get answers to a whole bunch of scientific questions. So they may say, as you write the protocol I think it is, well, you have to get so many CT scans and you have to get so many blood tests and stuff like that. And it can become onerous, Dr. Schilsky.

What’s happening in trial design so that, A, we talked about eligibility, you can get into the trial, but the things you’re asking of me may have logistical hurdles as well that you’re kind the lightening up on it to get to the key scientific question without all these other bells and whistles that make it tough on me.

Dr. Schilsky:
Yes. I like to think of it as the need to know and the nice to know, right? There are certain things you need to know in the trial to be sure that the treatment is working, the patient is safe and not having any severe side effects and things of that sort. A lot of that stuff is the same stuff that doctors order every day on their patients as part of routine clinical care, and so much of what needs to be collected in clinical trials really aligns pretty well with standard of care.

Now, that said, because clinical trials are research and because there’s always new frontiers to explore, sometimes testing in a clinical trial extends beyond what the standard of care is. Sometimes patients are asked to give extra specimens of their blood, of their normal tissues, of their tumor tissues. Extra biopsies might be required, things of that sort. Patients need to understand why they’re being asked to do that, what those specimens are going to be used for, how is it going to advance research.

And, frankly, they’re very important to expanding the scope of the research. So, for example, oftentimes those specimens are used. If the treatment doesn’t work in a patient having those specimens can help the scientists understand why the treatment didn’t work, and that opens up a whole new horizon to explore to potentially make the treatment better in other patients.

Andrew Schorr:
Mel, do you recognize that by being in a trial and the work that you and Cecelia have been doing that you’ve probably helped thousands of patients by first being in a trial and then you and Cecelia talking about it?

Mel Mann:
Yeah. Yeah. I guess that’s kind of hard sometimes. You don’t see yourself in that role, but as I look back on it, yes.

Andrew Schorr:
Cecelia, you’ve probably talked to a lot of people. Have you seen a change where‑‑you’ve been doing this for a number of years where earlier on people said no, no, no. Are people more receptive? Do you see a change going on? Let’s say in the African‑American community, do you think people are a little more receptive?

Cecelia Mann:
Yes, I think so. I think they are more receptive, and this has a lot to do with education and awareness, and that’s what we are out there doing when we are out there in the community. And the more they hear about it and the more they read about it and the more they can see examples like Melvin, and we know one or two other people that we’ve met that were also on a clinical trial. One is in our church, and he had a type of leukemia, and we didn’t know why he was sick. But he is doing very well.

And so the more we can get those examples out there in the community of successful clinical trial patients, it really helps and goes a long way toward helping people of color relax and come aboard. And I just say, please, do your research, educate yourself and ask questions and please stay open and don’t dismiss clinical trials out of hand.

Dr. Schilsky:
And, Andrew, if I could just add to that. I just want to make the point that it’s people like Mel who are creating the future. Everything we know about how to treat cancer we learned from the people who participated in the clinical trials. We’ve been doing clinical trials in cancer for at least 70 years, and all of the standard of care treatments that we have today came from the participation of people in clinical trials. And that’s how we make progress. That’s how we’ll continue to make progress.

So it’s the clinical trial participants who, sure, they’re in it for themselves. We understand that. They’re looking for a new treatment, a better outcome, but they are the heroes of oncology because they are paving the way, trying the course and ultimately making a better future for every cancer patient who follows them.

Andrew Schorr:
Amen. Let me just recap a couple of things, and correct me if I get anything wrong, either of you. So, first of all, Dr. Schilsky, I know there are more trials now than ever before, and they’re now looking at these rare subtypes as well, and so if we participate we may get the benefit of tomorrow’s benefit today. Cecelia was talking about assistance programs, people to help you sort it out, that you are noticing how there are difference among us about the ways that drugs are effective or not, and that’s so important to learn.

If we partner with you, Dr. Schilsky, and the many thousands of oncologists and researchers that you represent, can we get to the goal line faster? In other words, are you hopeful that if we really consider trials and participate in trials and stay in trials and the different groups that we can get closer to cancer cures?

Dr. Schilsky:
Absolutely. I mean, we have more and better cancer treatments today than we’ve ever had before. We have all sorts of new and hopeful treatments on the horizon. We have to prove that they are safe and effective treatments to get them out there into routine clinical practice, and that’s where the clinical trials come in. So the more people who participate in trials the more quickly those trials can be completed and give us an answer, then the more quickly those drugs will make their way into standard clinical practice where everybody can benefit from them.

Andrew Schorr:
Okay. So a couple of to‑dos for our audience if you’re a patient or a loved one or however you hear this. Ask your doctor about whether trials apply to you or your loved one who is diagnosed with cancer even if it’s on day one. You don’t have to be at death’s door. You’ve been diagnosed or a diagnosis is suspected, what tests can we do, how do we know what we’re dealing with, and when we look at the treatment options is a trial a possibility if that makes sense, right?

Dr. Schilsky:
Absolutely.

Andrew Schorr:
Okay. Step two, are there resources to help me overcome any obstacles I may have to participation, first understanding the trial, understanding it in my first language, sorting out is it right for me, getting to where it is and then staying in the trial because many people, unfortunately, don’t stay in the trial and so how do be help the trial get to the goal line?

And then lastly, Dr. Schilsky, it sounds like you’re doing a lot at the community level to have more doctors have an easier time of the bureaucracy that we’ve had with trials before and the understanding of this flood of trials that’s happening, right?

Dr. Schilsky:
Absolutely. And, you know, to be perfectly honest, the clinical trial community has‑‑we ourselves have created some of the bureaucracy, some of the excess regulation, some of the barriers to participation. It’s up to us to strip those away and solve the problem and make clinical trials more broadly available. We are working very hard now to make that happen.

Andrew Schorr:
Okay. So whatever community you’re in. I’m in the Ashkenazic Jewish community. Mel and Cecelia are in the African‑American community. We have people watching who are in the Hispanic community, the Asian community. If you have benefited from a trial, talk it up, right? Cecelia, people should talk it up, correct?

Cecelia Mann:
Exactly, yes. Please, talk it up.

Mel Mann:
Yes.

Andrew Schorr:
Mel, thank you. I wish you really continued good health. How many years has it been since you were diagnosed?

Mel Mann:
Well, in about two months it will be 24 years.

Andrew Schorr:
Twenty‑four years, and for me it’s 22 years. And had there not been trials either that we were in or somebody else was in we wouldn’t be here.

Mel Mann:
Yes.

Andrew Schorr:
So, thank you. And also, Cecelia, thank you for being a community activist when it comes to trials and being supportive of Mel as he’s been in a trial because some other people would say, you can’t go there, you can’t do this, and you’ve been supportive every step of the way. Thank you for that.

Cecelia Mann:
Oh, you’re so welcome. It was a pleasure to do it.

Andrew Schorr:
Mel and Cecelia Mann from Atlanta. And Dr. Richard Schilsky, you’ve devoted your life to this, Dr. Schilsky, and I just want to say on behalf of the cancer patient community thank you and thank you for the leadership that ASCO is trying to do, both with changing research requirements, working with government, working with industry, and you thank you personally for your devotion to us. I really appreciate you being with us.

Dr. Schilsky:
It was my great pleasure. And, again, congratulations to Mel and Cecelia.

Andrew Schorr:
Okay. Thank you all. So this is what we do with our Clinical Trials Mythbusters program. Please tell others about it. The replay is available very shortly and all kinds of little highlights that we’ve done today. But what’s important is wherever you are is remember we can’t develop new medicines unless all of us work together to participate to get the scientific answers that apply to us, whatever our unique situation is, and then we can work with government to approve new medicines, get them on the market and so many people can benefit in the US and worldwide.

Thank you so much for being with us on this Patient Empowerment Network program. I’m gratified to be part of it. Thanks too to our financial supporters AbbVie, Astellas, Celgene and Novartis and their dedication to drug development and supporting and sponsoring clinical trials. I’m Andrew Schorr near San Diego. Remember, knowledge can be the best medicine of all.


Please remember the opinions expressed on Patient Empowerment Network (PEN) are not necessarily the views of our sponsors, contributors, partners or PEN. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Clinical Trial MythBusters: Actionable Advice for Knocking Down Obstacles to Trial Participation

Actionable Advice for Knocking Down Obstacles to Trial Participation

Downloadable Program Guide

Clinical trials offer tomorrow’s medicine today, but more often than not, only a small fraction of patients ultimately enroll in a trial due to barriers posed by financial logistics, distrust and travel, to name a few. In this MythBusters program, we will examine the barriers to enrollment, evaluate patient needs and discuss resources to help guide people through the clinical trial process with the help from two experts, Dana Dornsife of Lazarex Foundation and Myeloma Survivor Reina Weiner.


Transcript:

Andrew: Hello from Carlsbad, California, near San Diego. I’m Andrew Schorr from Patient Power. Welcome to today’s Patient Empowerment Network program, clinical trials myth busters and actionable advice, resources for knocking down obstacles to trial participation. I wanna thank the companies that have provided financial support for this program. They have no editorial control, but we definitely thank them for their support. Those supporters are AbbVie Incorporated, Astellas, Celgene Corporation, and Novartis.

Okay. We have a lot to talk about. First of all, I’ll just say I’ve been in two clinical trials; one Phase 2 many years ago at MD Anderson for the leukemia I have, chronic lymphocytic leukemia. And that gave me tomorrow’s medicine today. It worked, but I had travel far to do it and there were costs involved.

And then I was in a second Phase 3 trial close to home, and that was good too, and discovered another cancer that I have, myelofibrosis, through the monitoring in the trial. So, I’m a believer, but there are obstacles, and let’s talk about some of these. And we’re gonna give you some very specific resources to overcome these obstacles, so that hopefully, if a clinical trial is right for you, you can participate, you can feel good about it, and you can move medical science along to help everyone who is dealing with that condition.

So, what are some of the issues? Financial, of course; logistical issues, of course; distrust, are they really gonna take care of you or are they gonna protect your safety? Is it really right? And are you being given the straight scoop? What about travel costs? I went from Seattle to Houston, Texas a few times. Costly, okay? Stay in a hotel. It’s costly. Get a babysitter, leave work; costly.

The guinea pig syndrome; you’ve heard about it so many times. Are they gonna experiment on you, and are they really protecting you, and are you a number, or you are a person with cancer, or your loved one? And then is your medical team that you’re talking to about your treatment, are they informed about clinical trials? Or are they pooh-poohing clinical trials because they don’t wanna do the paperwork, or it’s happening down the road and not at their clinic. Lots of issues; we’ll talk about that.

Okay, I got some great helpers. So, first let’s go to Asheville, North Carolina, and you are used to live in Charlotte. Reina Weiner joins us. Reina, welcome to our program today. There we go. Say that again, Reina, you were muted.

Reina: Thank you.

Andrew: Okay. Now we should tell you that last June, well, June of 2017, Reina had a autologous transplant for multiple myeloma. And along the way, leading up to that, over many years she was in four trials. So, first of all, Reina, let’s start with what’s most important. Post-transplant, how are you feeling today?

Reina: I’m feeling very well. Thank you, Andrew.

Andrew: Okay, and what’s coming up at the beginning of September?

Reina: What is coming up at the – oh, a big party is coming up. Our children are throwing us our 50th wedding anniversary party, so that’s been cool.

Andrew: Yeah. Well, congratulations. And you’ve been dealing with what became multiple myeloma since 1999. We’re gonna come back and track that in a minute, as far as the steps along the way, the concerns you had or not at different times about being in four clinical trials.

And now let’s go up near San Francisco in the East Bay of San Francisco Bay, Danville, California. Dana Dornsife. And Dana is the Chairman of the Lazarex Cancer Foundation. Dana, thank you so much for being with us.

Dana: Thank you, Andrew.

Andrew: Okay. Now ladies and gentlemen, I want you to know Dana and her husband and her family, overall, they’re incredibly philanthropic across a number of issues that are faced globally, and also in the US. But one of them is helping people with the financial issues that prevent them from being a clinical trial. So, Dana, this is a personal story for you, so maybe you could just tell us why did you start the foundation? It was a family issue.

Dana: It was a family issue, and that family issue really revealed to me a gap that exists in cancer care for advanced-stage patients who want to remain in their battle with cancer through clinical trial participation. My youngest sister’s husband, Mike, was diagnosed with pancreatic cancer in his early 40s. He was given one half of one percent chance to live, and at the time 35,000 people a year were diagnosed, and 35,000 people a year were dying from pancreatic cancer.

So, we decided as a family that if Mike wanted different results that we would need to do something different. And Mike and Erin went ahead and pursued standard of care, and I was tasked with identifying clinical trial opportunities for Mike. And, of course, that sounds very linear, but in fact, for a layperson it was a very difficult task to undertake. I did identify some trial opportunities for Mike. He did participate in a trial and responded well for a period of time, he had good quality of life.

And during that period of time he was meeting people who were asking him, “Hey, what are you doing? I wanna do what you’re doing.” And he would say to them, “Oh, just call my sister-in-law, Dana. She’ll help you.” And that’s literally how this organization began. Through those phone calls that I was receiving from other pancreatic cancer patients, I began to understand that Mike was able to take advantage of medical breakthroughs in clinical trials because he had a family who could afford to support him through the process. And all of these other families that I was talking to, they just didn’t have the financial wherewithal.

So, we started Lazarex in order to fill that gap and help people identify clinical trial opportunities, and then provide financial assistance to them to help cover the out-of-pocket expenses that create huge barriers for patients who are already experiencing financial toxicity due to their disease.

Andrew: Well, thank you for what you do. And we’re gonna talk a lot along the way about resources. There’s a downloadable guide that you’ll be provided with, along with a link to the replay of this program. And that’s gonna have specific resources that you can access, whether it’s financial issues, other issues you may be facing. So, look for that.

Today we’re really focused on actionable resources. So, let’s go to Reina for second. So, Reina, you had been in the pharmaceutical industry.

Reina: Yes, I had.

Andrew: So, you knew about drug development, and you understood about clinical trials. So, I’m willing to bet you were pretty proactive. People who weren’t in the field, they don’t know from clinical trials, and maybe they’d been worried about it. They’ve worried would they be experimented on, would they be a number and not name, would they get quality care. But you were probably, I have a feeling, pretty proactive. And you write about that. I know you have a book as well. So, is that Step 1 for people to speak up for themselves?

Reina: It is Step 1; absolutely, Step 1. And what I found is, first of all, people don’t know about trials. And if you go to a small community practice where they’re very busy, they don’t have the time, they don’t have the staff to really educate patients about trials, the best, best step for patients to take is to ask, “Is there a clinical trial that might be appropriate for me?” That’s huge.

Even when I went to a very well respected hospital and there was a researcher who was following me as I had smoldering myeloma and the numbers kept going up and up and up. I said is there – because I was living close to the NCI – is there a trial that would be appropriate to me at the NCI. And he said just a minute, turned around, went to his computer, found the trial and that’s how I got in.

Andrew: But it wasn’t at where you were receiving care or being monitored at that time. It was somewhere else.

Reina: It was not. It was at somewhere else.

Andrew: Okay. Dana, is one of the obstacles, not just financial, or maybe it’s even the business of cancer where if an oncology practice that you’re going to that’s maybe close to home is not doing the trial, maybe it’s not even in their financial interests to tell you. I mean, is there an awareness issue, do you feel?

Dana: There’s a huge awareness issue there, Andrew. And it all starts with knowledge is power, right, so I completely agreement with Reina’s comment about one of the first questions you need to ask is, is there a clinical trial out there for me because many doctors who are in community environments don’t offer that information. It’s not what they do every day. They’re there to administer standard of care. Only 6 percent of doctors actually engage in conversation with their patients about clinical trials, and that’s usually the 6 percent who are associated with research universities, right?

So, knowledge is power. If the patient doesn’t know about a clinical trial, they’re never going to participate. But once you find out and once you’ve identified an opportunity, the second biggest hurdle is that out-of-pocket expenses associated because most patients have been dealing with their disease for a longer period of time, and they’re basically broken in every way: physically, emotionally, spiritually, and, sadly, financially.

So, patients start to make decisions about the outcome of their care based on the size of their checkbook, and not focused on what’s best for them. And so, Lazarex eliminates that financial barrier as well to help patients say, “Yes, I can participate,” and we can get them where they need to be when they need to be there.

And that’s just the tip of the iceberg, Andrew, because there are many other barriers that exist; socioeconomic barriers, language, culture, historical barriers. And we are tackling all of those barriers one at a time. But really, the biggest two barriers are knowledge and financial.

Andrew: Right. And you mentioned about the historical barriers. Some people know about the Tuskegee experiments with African-American people, so in the African-American community, there still is a distrust among some people. Yet if you think about it from the FDA’s point of view where a company that’s developing a drug, or the NIH, they say okay, how does it work on broader populations or different ethnic groups or different ages or genders, et cetera?

They want to understand that data, and so not just having a number of people participating in the trial, but having it reach people who are in different situations, if you will. And so –

Reina: And if I may – ooh, I’m sorry.

Andrew: Reina, please, go ahead.

Reina: Well, if I may say that because people don’t know about it and the trials, the best trials, are trials with a variety of patients, but they do try to accrue populations who are certain ages, certain genders, ethnic groups, whatever they can get. And only 3 to 5 percent of patients participate, cancer patients, participate in clinical trials, and so much is lost if people don’t participate.

Andrew: Here in San Diego it’s sort of a pocket of a lot of medical research. There’s a lot up in your area, Dana, in the Bay Area, San Francisco Bay Area. I mean, it’s in North Carolina in the research triangle where that’s home state for Reina. And not to disclude others, and then certainly up around Boston. There are like companies all over the place and many of them are in earlier drug developments.

So, when you talk about immuno-oncology now, can we harness our immune system with the help of some medicine to fight the cancer, and I know some people who’ve received it; lung cancer patients who are living, et cetera, melanoma patients who are living for an extended time. These companies can’t move forward unless there’re people who are in the trials. So, the FDA says where’s your data? And they’re saying well, we’re trying, but we haven’t been able to complete this trial. Right, Dana? So, we can’t move towards cures unless we all come together.

Dana: That’s exactly right. So, let me just throw a few statistics out at you that I found astounding when I learned of them. So, we have a 48 percent failure rate of clinical trials, and it’s not because the drug didn’t work. We will never know, quite frankly, if the drug would have worked or not. And we will never know because there weren’t enough patients enrolled in the trials to find out.

So, 11 percent of trials never enroll a single patient, if you can believe that. So, here we are with an almost 50 percent failure rate, and yet we have 600,000 patients a year in this country who are dying from cancer. So, there’s this incredible disconnect between the thousands of patients who would participate in clinical trials if they could, and the thousands of clinical trials that need patients to participate in order to succeed. And without successfully completing those trials, those drugs are never going to get market to help the cancer patients that they are intended to serve and help.

That’s why Lazarex Cancer Foundation exists, and that’s why removing the barriers to clinical trials is so important. Our process does not lend itself well to that. And I just want to take a step back, Andrew, to address the minority participation in clinical trials. We all understand because of epigenetics and, yeah, advances in medical science that we need to have the full spectrum of our population participating in clinical trials. But that doesn’t happen. When you look at the 5 percent of patients who actually participate and you break it down ethnically and racially, less than 5 percent are from minority communities combined.

So, in theory, though we say we understand the importance of that, we’re actually not in practice doing what needs to be done. And so a lot of our work is also focused on reaching out to those socioeconomically challenged and racial and ethnic minority communities to raise awareness and help people like you’re doing on this program dispel the myths around clinical trials, so that they’re more inclined to ask better questions.

Andrew: Right. So, so important, and I applaud for that work. We’re gonna talk about the financial process in a second. Reina, so you were involved in a National Institutes of Health or National Cancer Institute trial.

Reina: Yes.

Andrew: A couple of them, I think, and one at Memorial Sloan Kettering in New York’s premier resources. So, we talked about your tip was you gotta speak up and ask about trials, where they’re at that center, wherever you are, by XYZ oncology in a suburban area, whatever it is or not. So, what’s Step 2? So, for instance, now I understand there are people – and Dana, I’d like your comment on it too.

At some clinics now where there are clinical trial – there are nurse navigators, but often sometimes there are clinical trial navigators too, but often you gotta ask about that too, right, Reina? I mean, it’s speaking up and looking for the resources that are available to you there or wherever you choose to go, right?

Dana: Yes, and there are organizations like Dana’s who help people do clinical trial searches because that’s a bit overwhelming when you are already frightened, you already have the financial issues coming up. And like you mentioned, logistical issues. So, there is Dana’s organization; therefore, myeloma, the SparkCures. There’s the MMRF. There’s the International Myeloma Foundation. There is something called Cis Crypt. And so, they will help you find a trial.

And there are lots of regional trials groups, so you may not need to go to the big, big research center. They might be able to do it locally for you. But I always want to bring up the fact that there’s so much misinformation about trials and what it entails. There’s a tremendous amount of fear. And when I went on the first trial, as I wrote about a little blog recently, everybody said to my husband – well, not everybody, but an awful lot of people said why would you let your wife going in a clinical trial? She’s definitely gonna be a guinea pig.

And I can tell you very, very, very clearly that you get so much care. There’s so much documentation. And the patient’s health is never sacrificed for the research ever. And so, and you sign a consent form, so you’re very clear about what is going to happen. And yes, there’s more there’s more bloodwork. Yes, there are more biopsies. And it’s part of research. And when you sign up, you sign up. And I had more than I’d like to even talk about, but I feel very grateful and very humbled for the care that I received.

And I can tell you, too, that I talked to other people on the trial. And yes, they hope to gain better control of their cancer. But, in addition, they really hope to help the next group of patients who are coming up, so that these new treatments actually happen.

Andrew: I feel the same way. I was in a trial at MD Anderson in 2000, and the three-drug combination I got was not approved till ten years later, but they learned a lot. And you were on a three-drug combination, which I think still has not been approved for first line, but it’s is widely used, I think.

Dana: Right.

Andrew: So, in multiple myeloma. I wanted to mention some other resources, the Leukemia & Lymphoma Society also has a resource center. You can call them. So, there’re these different groups that help you identify a trial, and doctors who specialize, so let’s say pancreatic cancer, you mentioned earlier Dana. I got a call from a friend in Miami, “How do I find a pancreatic cancer specialist?” And I connected them with PanCAN, Pancreatic Cancer Action Network in Los Angeles, who knows who are the doctors who have the most experience with that.

Now, Dana, so then the next thing comes up is alright, I’ve identified the trial, but it’s not where I am. So, now we talk about logistics and finance. So, let’s say somebody calls your foundation. Tell us how it works. So, I don’t have the resources. Maybe they live in Northern California and the trial is in Southern California or in Salt Lake City. What happens next?

Dana: So, Andrew, in some cases it’s not even that distance. In some cases it’s getting from Sacramento to San Francisco, which is literally a one hour, one-and-a-half hours without traffic, in your car. And sometimes it’s a tank of gas, a bridge toll, and parking. I mean, we’re not talking about thousands of dollars in some cases, but it’s still the difference between life and death.

When someone calls Lazarex Cancer Foundation, they can contact us directly. We have a financial application that we use to determine the degree of eligibility for patients to get their out-of-pocket expenses reimbursed. Or they can be referred to us by their social worker at the institution where they are receiving, or thinking about participating in a clinical trial.

We take a look at the household income of the patient, and I believe our guidelines are very generous. We go up to seven times the federal poverty guidelines for patients. And we arrived at that number through trial and error. Our goal is to help as many patients as possible participate in clinical trials, and turn away as few as possible. And then we reimburse on a sliding scale from 100 percent to 75 percent to 50 percent depending upon your household income.

And it’s a pretty easy process to go through in order to be enrolled and receive the reimbursement. And then we reimburse our patients monthly, on a monthly basis. And in some cases, we’ve been working with patients, we follow them, like Reina, through two, three, four clinical trials. And we’ve been supporting them in trials for years. And without doing what we do, they may not be here with us today.

Andrew: Well, I’m sure you’ve saved some lives and lengthened some lives. Reina, so you were in the pharmaceutical industry and in oncology, I believe, before all of this started happening to you. And you’ve continued teaching nurses and devoting yourself to education and your book and your blogs. Thank you for all that. Maybe that’s what life’s about.

But knowing on the inside there are pharmaceutical programs, in some cases, I think, particularly for rare cancers where they may provide assistance. They can’t pay you to be in the trial, but there are at times assistance and travel logistics, particularly for rare cancers where maybe the trial is not, not one hour away. Am I right, Reina? Are you familiar?

Reina: Oh, there are. And sometimes when I was working, there would be a patient who had a cancer that really was not aligned with a particular treatment that would be effective for them. And so, the doctor wanted to try an off-label use of a product, and so then they would come to me and asked me if I could get the pharmaceutical company to provide the drug for free.

And sometimes it takes a little doing, but I was concerned about the patient and hoping to get them a better quality of life, if not an extended period of life. And so, yeah, the company would do that. Not every day, not all the time, but if the company had evidence that this was a patient who would benefit from the off-label use of a product then they would help them out.

Andrew: Okay. So, Dana, related to other organizations providing assistance, and I recently interviewed someone from the Family Reach Foundation where they help with rent or things, groceries, things like that. So, somebody says, “Oh my God, I’m afraid of a trial, I can’t go there,” or if they hear about it and they say, “Hmm, well, maybe I could, but I’d have to leave work, or maybe my spouse would have to leave work, we’d have to find somebody to pick up the kids from school, oh my God.” There are organizations that can help with some of these family processes, aren’t there?

Dana: Absolutely. And I think we’ve provided the Patient Empowerment Network with a list of those. 21st Century C.A.R.E. is an organization that provides patients with immediate financial assistance for expenses related to active cancer treatments. Cancer Care provides assistance for cancer-related costs. There’s a Cancer Care Co-Payment Assistance Foundation. We get that question a lot.

We’ll help with the out-of-pocket travel expenses, and in fact, some of the medical and diagnostic expenses that aren’t covered by insurance. When you’re participating in a trial, sometimes you have to get more stems than insurance will cover or whatever. But co-pays are a big deal for people to be able to afford those, and so, that is another organization that can help. Patient Advocate Foundation, which is an underinsured resource directory.

So, there are a lot of you nonprofits out there who exist to support patients through the fifth process. It’s just a matter of helping patients really understand and put together all of those resources in a way that they can access them.

Andrew: Okay. So, Reina, you’ve been through it four times, and you’re a pretty savvy person. Not all of us know as much, so help us now. So, one of the questions in a trial is, and in cancer, am I gonna get what I describe as the good stuff, knowing that the good stuff that’s being tried may not be good. I mean, it may not work out. There are trials that go bust. Not just for not getting people, but they got people, but it wasn’t as effective as they hoped it would be.

But let’s say we’ve done our homework and we go to a certain clinic, but it’s some sorta controlled trial. We don’t know whether we’ll be in the arm. So, was a concern for you? Were you gonna get the good stuff, and why do it?

Reina: Well, no, really, Andrew, because I know that like if it’s a Phase 3 trial, so you’re comparing standard of care versus the newest and hopefully the latest and greatest. If it turns out that one arm of the trial really shows a significant improvement, patients are always switched to the more effective arm of the trial. They don’t leave you on this arm of the trial thinking well, what the heck, we’ll just leave you there and see how the research pans out. So, they are always switched over to the most effective.

So, I wasn’t really concerned about that. And in the Phase 2 trial, it’s just seeing if the product was effective. And so, that was obviously not a concern for me. So, it worked out, and I do think, though, like what Dana does is absolutely wonderful at totally, totally, totally past wonderful.

But I always try to let people know who have friends and family who are facing some chronic significant illness that don’t just call and say let me know, let me know if I can help you because that’s so ambiguous. And most people will not call because they have pride or they think they can do it all by themselves.

So, I always try to suggest to people that if you’re calling somebody who you think might need some help, be specific. Call and say, “Can I walk the dog? I’m going to the grocery store in an hour. Is there something I can pick up for you? Can I mow the grass?” Anything that will help, but make sure that you are specific in your offering.

Andrew: I want to talk about a related issue. You use the word pride. Some people, maybe in some cases it’s even shame. They developed a certain cancer. Where these are maybe middle-class people who’ve had some resources. They’ve been paying their mortgage. They’ve been paying their expenses, making do. But now they get hit with a cancer diagnosis, which is catastrophic, and there is help available, Dana, but they’re too proud to ask for it when this could happen to anybody. And maybe you’ve even countered that along the way or know there’re people out there. What would you say to people, to not be shamed and to speak up?

Dana: Yeah. Well, sadly, one in three women will be diagnosed with cancer, and one in two men. And so, this is not an uncommon scenario, right? The likelihood of knowing someone who will receive a cancer diagnosis is very likely. So, I think that patients have to understand that pride doesn’t help you in your process with battling this disease. You have to take advantage of every opportunity that’s out there in order to come out on the positive side of this experience. And if you don’t take advantage of every opportunity, you may not.

And so, it’s one of those things that we just have to deal with right from the beginning, and just say okay, again, knowledge is power. I’m going to surround myself or engage with the people that are around me who want to help me. And you have to put that team together because you will need your team with this disease.

Andrew: Okay, so great advice. Reina, part of your team maybe could be the first doctor you saw who gave you the diagnosis, but they might not be the one where a trial was offered. So, first step is you talked about speaking up, but it takes a lot of courage to say to the doctor in the white coat with all the letters after their name, you know, thank you so much, Doctor, and I’ve either found out about a trial, or your turned and typed it in somewhere else. I hope you don’t mind, but I am going to go over there. Maybe you can advise me along the way.

But that takes courage because people are terrified, and they may be bold in principal in that situation with the person in the white coat. What advice would you give?

Reina: Ooh, well, that’s a big one for a lot of people. And, really, you know what, I imagined that it would be people who are older, who come from a generation where the doctor has the final word. But what I found out when I was writing my third book is that there were younger people who also feel very uncomfortable speaking up, asking a doctor, and so forth. But really, what to really put in your little mind and in your heart is this your life.

This is not just kind of a trip to the mall. This is really important for you to either improve the quality of your life or extend the quality of life, so take a deep breath, be very polite, and I think most doctors who are professional and open-minded will hear what you have to say if you present it in a way that they can hear. And if they really don’t hear you then it might be time to have a look around to see who will.

And, really, the bottom line is you need to trust yourself. And if you feel that this is really right, that there is a clinical trial that you would be eligible for and you can participate in with Dana’s help, with the financial, with the logistics, and so forth. Like I said, you just take a deep breath. And most doctors, like I said, really want the best for you.

Andrew: Okay, let’s talk about something that comes up. One of the things for people is the criteria of different trials. Dana, I don’t know if this is in your area, too, related to financial, but people let’s say okay, I wanna be in a trial, but the criteria are so narrow that I really wanna be in the trial, but they say I can’t.

Dana: Yeah, so that is a sad reality in many cases. And I refer to this as Clinical Trial Nirvana Syndrome where as a drug sponsor for trial, you want to attract the healthiest patients you can to participate in your trial, so that you have the greatest chance of success. But, unfortunately, in many instances, in most instances, a cancer diagnosis is accompanied by other comorbidities like heart disease or diabetes or other maladies that would preclude a patient from being able to participate in a trial.

So, that is an area that we are looking into and trying to – we have several proposals out there with various aspects of our government to try and really take a closer look at that, to try and make the trial makeup in relation to patient participants better mirror the realities of our situation because the likelihood of someone, if the drug gets approved, taking that drug and having a comorbidity is pretty likely.

And yet we won’t know what will happen there, right. So, we have to drill down on these issues and it’s a great, great issue to bring up. So, we’ve got a lot of work to do ahead of us.

Andrew: Right. And another thing that comes up too, and Kevin sent in a question. Kevin, thank you for this, matching what’s available in clinical trials to where you are in your journey with an illness. So, on Day 1 you’re diagnosed. I know Esther and I, we were crying and almost on the floor. And I thought I would be dead the next day. And it really took a while to overcome the terror of the diagnosis. And so, we were not even – well, the doctor wasn’t talking about trials; we wouldn’t have been hearing it anyway.

And some of us, thank God, with some trials, with some cancers now, are blessed with living longer and we start to learn. And then we want to know, in our situation, what applies to us. So, I know there are a lot of efforts being made to match trial offerings to where you are and what you might need to know now, what might need to be offering.

And some of you have heard this term, artificial intelligence, where we in the Internet business are all trying to fine tune what we’re suggesting or putting in front of you based on who you are and where you are, recognizing privacy and all those kinda things to make it more manageable.

We still have a long way to go. I mean, we have clinicaltrials.gov, but it’s not tied to where I am, who I am, where I am in my journey. It’s just what’s being done in a certain illness, right, Reina?

Reina: Yes.

Andrew: So, we have to refine our tools.

Reina: Absolutely.

Andrew: We have to refine our tools. Well, we’ve been getting in a number of questions. So, here’s one. So, David; so, he says as the excessive use of CT scans in clinical practice moves away from being the norm, have they lessened their use in clinical trials? In other words, this is about testing, and maybe it’s about the requirements.

Dana, I don’t know if you have feelings about it, but the scientists who are doing these trials, they wanna know everything. They would like to test us. So, the CT scan, and I mean I’m gonna have one next week, but it has radiation, right?

Dana: Right.

Andrew: So, let’s do a bunch of CT scans. No, let’s do a bunch of bone marrow biopsies. No. So, I’m saying I’m sorry. Not just do I have to pay something for these tests, or is there a co-pay or whatever, but also am I gonna be radiated? Am I gonna be poked? So, what about those issues? Is there dialogue going on, not just to help us financially, but also make it less onerous, I guess?

Dana: Yes, in fact there is dialogue going on about that, and it’s good, heartfelt dialogue. And it’s coming from a myriad of stakeholders, right, not just from patient advocacy organizations, but also from within industry insurers. And the whole goal is to okay, let’s stop looking at patients as a chart or a number on a piece of paper, and let’s understand that these are living, breathing human beings who are voluntarily participating in this clinical trial process for the benefit of not only themselves, but future patients to come and our industry.

And let’s start treating patients as humans who are participating, and let’s see what we can do to lessen the number of visits or minimize the number of scans and blood work, et cetera. So, there is active dialogue around that, and I think there’s a much higher degree of sensitivity on behalf of the teams who are actually putting the protocols together now.

Andrew: All right, I think so. And I know in some cases they’re doing what’s called trial simulations with a panel of patients and saying okay, we’re trying to answer these scientific questions and see if this drug that’s in development can do better for patients and would require so many office visits. Or so many, you come to the site, but so many could be done, maybe with your local doctor if that’s closer to home. So many blood tests, so many CT scans, so many biopsies. Imagine lung cancer patients with another lung biopsy. Not fun, and often not available.

So, there are all these kind of questions. And I think that’s going on although it needs to happen more. Now Dana, do you talk to the pharmaceutical industry? We had a question from Vi Life wanting to know related to trial awareness. Beyond the financial, do you work with pharmaceutical companies at all, as you are now, today? I mean, what we’re doing here is just to raise awareness about trials or other programs that you may do.

Dana: So, we are engaging with pharma right now. We were very fortunate to work with the FDA earlier this year in securing language around reimbursement of patients’ out-of-pocket expenses associated with clinical trials. There was some very nebulous language out there that was really preventing pharma from being able to support programs like ours.

And what we’re doing now is, in addition to we’re bridging this gap for patients that exist every day by reimbursing patients, but that is not a sustainable business model. It’s noble, but we have to have our tin cup out every day. And the number of patients we can help is directly related to the amount of money that we have in our account, right?

So, in addition to that program, our Lazarex Care Program, what we are also doing is trying to fix this problem and do it in a sustainable way. And in order to do that, we actually have to shift the burden from the patient back into industry, right, and help industry understand why they should include these out-of-pocket expenses as part of the clinical trial protocol every time, right, so they can enroll trials on time, on budget, save R&D dollars, preserve patent years, right?

I mean, there are a lot of reasons why pharma would want to participate in a program like that, in addition to the fact that it’s the right thing to do, right? And then we get more drugs to market faster, and we provide a platform of equitable access for everyone. So, we are engaging pharma in discussions right now about funding this program, we call Lazarex our IMPACT Program, that’s being rolled out at comprehensive cancer centers across the country. And it stands for Improving Patient Access to Cancer Clinical Trials.

It has been received very well and I’m happy to say that Amgen actually stepped up and funded this, so we are rolling it out here in California, and we are hoping that we’ll have similar opportunities in a couple of other areas in the United States. So, they are interested, and they want to improve clinical trial enrollment retention, and especially minority participation.

Andrew: Right. Boy, that you. Again, I keep saying thank you for what you’re doing, but you’re a real leader in the field. I’m going back next month to the Biden Cancer Initiative Summit continued by Vice President, Biden, former Vice President Biden, and his wife who continue to do leadership in this. And there’ll be a lot of senior people there and I’m hoping we can talk. And I know this issue of how can we advance cancer care through research in partnership with patients is a big one. So, Dana, thank you for helping lead the way in getting this going. And thanks to Amgen just as an example.

Reina, so, we talked about the cultural differences of people being in trials. We talked about the pride people may have in asking for assistance, the fear people have maybe participating in trials. You still have a – not now. I mean, you’re doing so well and you’ve been through trials and it’s worked out well. But there must have been some bumps along the way. Were there any misgivings at different times? And if so, how did you overcome it?

Reina: Oh, yeah. Well, there were definitely misgivings, I am sure. The first trial was when I asked the doctor if there’s something going on at the NCI. And there was no misgivings about that because that was a very observational trial. The second trial was much more progressive and I felt kind of a little uncertain about it, and so I asked the researcher at this well-known institution if I should participate because the trial, I should back up a little bit, that was for either smoldering myeloma patients or active disease patients.

At the time I was smoldering, and most physicians didn’t believe that that was a good idea to treat smoldering and wait until it became active. So, I asked this one researcher and he said absolutely, not, do not participate in the trial. And then I called someone else also from a very respected institution where I had been, and he said well, if you join that trial you’ll be crossing the Rubicon, which I didn’t even know what the Rubicon was at the time. I had to go look it up.

But, basically, once you start treatment, you kind of go on that journey and there’s no way to step off. But then I thought about it, I thought about it, thought about it, and finally I decided to trust myself because I had been to the NCI. I felt very safe there. And I decided to move ahead with it.

So, yes, I had plenty of misgivings about that. The other trials, not really because that trial changed my life and it gave me a very reasonable complete response. And the other ones, like I said, they just kinda fell in with the collecting a good amount of stem cells for a transplant and so forth.

Andrew: I wanna talk about family issues. So, the decision to be in a trial affects the family, whether it’s somebody’s driving you to the doctor, somebody’s taking off work, their worry, how they feel about trials, their own view of it, family logistics, costs, et cetera. We’ve talked about that. So, you wrote this blog about people questioning your husband, I guess, was your wife gonna be in a trial? So, how did you overcome that, whether if not with your husband, just with your community that you weren’t like crazy?

Reina: Well, they already know I’m crazy, so that’s a total aside. But, really, trying to educate people about the misinformation about trials; say, look, I will never be a guinea pig because that’s not what trials are about. And it’s very well controlled and there’s a lot of data that follows you. The care that I got was excellent.

And I try to dispel, like I said, a lot of the myths; that you signed consent form, which clearly explains what the trial is about, what your commitment to it is, and you can also drop out for any reason. There was hope that you don’t because they would like to have some results that then will lead into future treatments for patients. But you can drop out, so, really, taking that opportunity to educate people about what a clinical trial is like and that there are no guinea pigs.

Andrew: I wanna just – oh, yes, please, Dana.

Dana: Yeah, if I could just offer something in that regard. For people who haven’t gone down this path, the journey with cancer, having a cancer diagnosis is not like other chronic diseases, right, like diabetes, for instance, that you can typically control with insulin or whatever, right? For a cancer patient who has failed standard of care, who’s gone through maybe second- or third-line treatment options, but still has progressive disease, that patient will die if they don’t do something, right?

And so, clinical trials offer tomorrow cures today in some instances, right, and we don’t always have positive results in clinical trials. But for a patient who’s at that crossroad where their doctor has delivered those words, “You need to get your affairs in order,” right, it’s not a matter of am I crazy if I participate in a clinical trial. What it is a matter of is do I wanna live? And if so, what clinical trial can I participate in? It’s a very different decision tree.

Andrew: Right. And I certainly say that all the time. I got a call, as I mentioned, from a friend in Miami. The mother has a very serious cancer. And I said part of the initial discussion, even the initial discussion, Dana, can also be are there clinical trials that we should consider along with standard therapy? So, certainly, if you’ve failed or they’ve failed you, the treatments no longer work, what is the 360 degree view? And if you don’t do it here, so they do it down the road, or do they do it across the country? And what are the issues for you participating?

So, a lot of thinking, but it’s gotta be part of the discussion. So, so sadly now, what are we seeing; 3 percent, 4 percent, 5 percent of adults participating in cancer clinical trials in the US. Not good at all. And are we hurting ourselves with the chance of future therapies that can be more effective, or even cures because some of these companies sometimes are venture-backed. They don’t have money forever, you know, and they’re trying to get to the goal line to go the FDA.

Look, here is another question we got in. Tamara, our producer, just sent in. She says well, what happens when you join a clinical trial and it doesn’t have a beneficial impact? So, Reina, they didn’t know that the trials would necessarily work out for you. So, what happens then? Do you go on another trial? What do you do?

Reina: Well, if it doesn’t and you don’t seem to be responding to the therapy on the trial, or you find it intolerable yourself, then they will always return you to your oncologist who you had been seeing previously. But, on the other hand, they may offer you another trial that’s available that you would be eligible for as well. So, I mean, I really try to stress to people that the researchers are looking out for you. They want the best income, in addition to accruing the data that they hope.

And I can tell you that when I was on a trial at the National Cancer Institute, when I had questions, especially about the trial with smoldering versus active disease for myeloma, they would spend a couple of hours for me, explained with me, can I say that, right? Yes, spent a couple hours with me explaining all of the aspects of the trial, so yeah.

Andrew: I wanna point up an example that some people have heard of a woman I’ve become friendly with in the myeloma community, Reina. Cherie Rineker. So, Cherie’s down in Houston, and she was dying of myeloma. And she’d been in trials and treatments. She was at MD Anderson. Bob Orlowski is one of the top doctors in the world, and her doctor. And she was in different trials and then things were not working.

And she was put in touch with another researcher doing this CAR T investigation for multiple myeloma, which is pretty new, pretty new. And they’re learning a lot. It’s not a slam dunk, but so far it’s worked for her. It saved her life. She went to Nashville, Tennessee from Houston where she lives, and maybe I’m not sure the financial issues, Dana, about going. But that’s where she’d been in successive trials. And some were not working or no longer were working. There was another approach.

I wanna ask about another concept I’ve heard called siteless trials. And I don’t know, Dana, you’re nodding your head. Maybe you are familiar with this. One is a siteless trial because we talked about these trials going on at these academic medical centers, but not much elsewhere.

Dana: So, I have tell you, I don’t have a lot of experience with siteless trials, but there is a lot of dialogue taking place around rather than having the patient go to the trial, bringing the trial to the patient, and I think that’s the impetus behind a siteless clinical trial.

I think cancer has some unique challenges, especially blood-based cancers in clinical trials, and the oversight of patients participating in those trials that make siteless trials a bit of a challenge. But I think the place to start is in other diseases, or perhaps where you have a cancer diagnosis that’s not a rare form of cancer, whatever that requires, a high degree of oversight.

But the whole goal in doing this is to understand how we can get more people into these trials and make it less obtrusive on their life, right, so that more patients would be inclined to participate, increase our enrollment retention, our minority participation, and, ultimately, reduce the burden on the patient to participate.

Andrew: Esther and I’ve given a lot of talks at different conferences, and we said you have to see patients who might be considering or are in a trial as investors. So, they’re gonna invest with their body, their time, sacrifices, and other things in their lives for the hope of being cured if they could, or doing better.

And there needs to be the communication, financial support, logistical support in really treating people with a lot of respect as a person. Reina, do you agree with that, that we have to get to that concept where we’re taken care of? And you felt that way, but we need to do it for more people and have more people feel confident that it’ll work out that way.

Reina: Oh, certainly. Certainly, I do. And the education is really essential. And after I was in the first trial, I talked to everybody who would listen to me. And even if they didn’t, I would talk anyway just to try to say this is a place where you can go where you will receive what is hopefully the newest and the best treatment that’s available. That you will be cared for as well as you can possibly be, and that everything is documented. You know all the options that you have staying on the trial, giving consent, making sure you have all the information that you need to feel comfortable.

And Dana’s organization, hopefully, helping people out financially and logistically. There are ways to get into trials that at times are very successful. For me personally, I don’t know that I would be alive now if I had been on that trial, and that’s really my claim to fame, what can I say?

Andrew: And, Reina, I would say the same thing. Had I not been in a Phase 2 trial for chronic lymphocytic leukemia in 2000, I wouldn’t be around to have had retreatment last year, which has work quite well; 17-year remission. And I wouldn’t have been able to do this, and really have a purpose in life. So, I’m very grateful for being in the trial.

Dana, I can’t tell you – we were talking about gratitude, for you; came up in your family. You saw the gap for, not so much your family, but so many other families. The issues, financial issues, and you’ve been very philanthropic and, obviously, trying to have leadership in getting at some of these – we have a very imperfect system right now, so we have a long way to go. But for our viewers, if you’re living with cancer now, if your loved one is living with cancer, there are resources, people like Lazarex, people have been through it, like Reina.

We’re gonna give you this downloadable guide. And you’re gonna connect with these resources. Don’t… Put your pride away. Dana said it so well. There’s a very high likelihood we’re gonna be affected by cancer in our families, and there is help to navigate what’s kinda complicated right now, but is doable and can offer you the chance of doing better. Dana, did I say it right?

Dana: You did. You did. You did a great job, Andrew. Thanks.

Andrew: Okay. Well, thank you. And thanks to the Lazarex Cancer Foundation and, really, all you’re doing. And let’s hope that we can improve this process, increase participation, and have so many of these companies and the government that are trying to get scientific answers. We participate as respected patient investors. And we do better well. Reina, any final words from you with your 50th wedding anniversary coming up?

Reina: I’m very grateful. I’m very grateful to be here. I’m grateful for all the clinical trials, all the physicians who have taken care of me and who listened to all my concerns and fears. And I am super-duper grateful to my husband who has supported me, helped me, been there, been my caregiver, and washed the food for me when I had the transplant, and really, all the people who have been on the journey with me. So, if you are considering a clinical trial, if there is one that you might be eligible for, give it some thought. It’s a really important choice for you to make.

Andrew: Reina, thank you so much, all the best. Happy anniversary, early. Dana, best to you. Dana Dornsife, joining us from the Lazarex Cancer Foundation in the San Francisco Bay area. Dana, good health to your family, and thank you for all you do. Thanks for being with us, Dana.

Dana: Thank you.

Andrew: And, Reina, all the best, and thank you for those great words of wisdom. And we’ll meet in person sometime and I’ll give you a big hug, okay?

Reina: I hope so. I hope so. You take care of yourself, Andrew. Thank you so much.

Andrew: Thank you for joining us for this Patient Empowerment Network program Clinical Trials Mythbusters. We hope to do more. I wanna thank the companies that have helped provide funding for it; Abbvie Incorporated, Astellas, Celgene, and Novartis, for their support.

Thank you for joining us. I’m Andrew Schorr from Patient Power down near San Diego. Remember, knowledge can be the best medicine of all.

Exercise and Nutrition Before and After Myeloma Treatment: What You Should Know

Living Well with Multiple Myeloma

Exercise and Nutrition Before and After Myeloma Treatment: What You Should Know from Patient Empowerment Network on Vimeo.

Downloadable Program Guide

Exercise and nutrition are important components to long-term health for everyone. But as a myeloma patient, are there specific tips for exercising safely? Can incorporating simple lifestyle changes improve and maintain good bone health? This webinar, featuring physical therapist Melanie House and dietitian Alexa Welch, both from University of Iowa Hospitals and Clinics, will provide guidance for individuals looking to increase their overall health through diet and exercise.


Transcript:

Andrew Schorr:
Greetings. I’m Andrew Schorr coming to you from Quebec City, Canada. I’m delighted to be here, and hopefully some of our Canadian friends are on with us. We’re going it go around the US as well with some leading experts in this important Patient Empowerment Network program produced by Patient Power. And the program is Exercise and Nutrition Before and After Myeloma Treatment, What You Should Know.

And we have some very knowledgeable experts who will fill you in, so take notes, with stuff you can discuss with your caregiver if you’re a patient, with your family members and for you to know so you do as well as you can living with myeloma whether you are going through treatment like transplant or on multiple drugs. Okay.

Lots to talk about, and we have received many of your questions already, but if you have a question send it in to myeloma@patientpower.info, myeloma@patientpower.info. I want to thank the companies that have provided financial support for this program. We’re very grateful to them. They have no editorial control, but they want to support the myeloma community. Those sponsors are AbbVie Incorporated and Celgene Corporation and Takeda Oncology. So thanks to them. All right. Ready to get started?

Let’s go first to Cleveland, Ohio, where he is joining us by phone, and that is my friend Jim Bond. Welcome back to our program. Thank you for being with us, Jim.

Jim Bond:
Oh, you’re welcome. Thank you for having me. It’s good to be here.

Andrew Schorr:
So, Jim, you were diagnosed with multiple myeloma. What is it, like 26 years ago?

Jim Bond:
Yes, in 1992.

Andrew Schorr:
Okay. You’ve had a variety of treatments and clinical trials, and you’ve had transplant, and then you also developed a second very serious cancer, AML, so you’ve had altogether I think four transplants. Is that right?

Jim Bond:
That’s right.

Andrew Schorr:
Whoa. Okay. Now, we should mention that in a couple of days, Jim, you are going to once again be on your bicycle four days riding 328 miles. What is that ride that you’re doing now for I think the 12th time?

Jim Bond:
It is the 12th time, and it’s the American Cancer Society Pan Ohio Hope Ride, which my wife Kathleen founded and leads. She got me to ride, and I’ve been able to do it 11 straight years and I’m done training. I trained an hour this morning and I’m ready to go, so in two days we start from Cincinnati and four days later 350 of us will arrive in Cleveland, Ohio.

Andrew Schorr:
Okay. Well, we’re all riding with you, Jim. Exercise and physical fitness has played a big role for you, and we’re going to come back to that in a minute, and you’re going to tell your story how your commitment to exercise has really helped you survive myeloma and also get the treatment you needed for acute myeloid leukemia, they call it. So we’ll be back to you, but I want to introduce our other experts.

So let’s go to our experts, medical experts who are in Iowa City, Iowa, at the University of Iowa Hospitals first bringing back to one of our programs oncology physical therapist, a veteran in the field, Melanie House. Melanie, welcome to our program today.

Melanie House:
Thank you, Andrew. It’s great to be here.

Andrew Schorr:
Okay. Thank you. And, Melanie, just to understand, you’ve been working with oncology patients including on the transplant unit there for about how many years?

Melanie House:
Well, oncology patients actually for most of my career. Probably in the early 90s I started working on some of the oncology floors, but I’ve been specific been overseeing the bone marrow transplant unit since January of 2010.

Andrew Schorr:
Okay. And they have some myeloma patients who come through there, right, who have transplant?

Melanie House:
Yes. Actually, that’s a significant part of our population, is the folks with multiple myeloma.

Andrew Schorr:
Okay. We have a lot to talk about. Okay. But you have a colleague I’d like to introduce who is a dietician with oncology patients and also works on the same floor as there with people who are going through a lot including transplant. So Alexa Welsh, thank you for being with us also from the University of Iowa Hospitals.

Alexa Welch
Glad to be here today.

Andrew Schorr:
How long have you been in the dietician field?

Alexa Welch
I have worked as a the dietician for three years, and then I have worked on the same floor as Melanie with the bone marrow transplant patients now for two years.

Andrew Schorr:
Okay. Wow. All right. So let’s start with exercise, Melanie. So, you know, I’ve interviewed a number of myeloma patients over the years, and there are some people who find out they have myeloma when a family member gives them a hug and then they have like cracked ribs, and they never knew that they had this illness they never heard of. They never knew that their bones were at risk, and then they go in and they get this diagnosis. And it’s terrifying. So you think, well, gee, if somebody giving me a hug can crack my ribs and I have myeloma how on earth can I exercise? What do you tell people related to these bone issues?

Melanie House:
Well, I always take time to educate my patients on where their lytic lesions or pathologic fractures may be located. In my experience that’s actually an area where patients often don’t realize, perhaps they’ve never viewed their imaging. And I encourage my patients to better understand that because if you don’t realize where those lesions are then you wouldn’t have good information to guide other activities or precautions that you might need to take.

Andrew Schorr:
Okay. So at step one, know where you have lesions. Step two‑‑but that would freak me out. I’m a leukemia survivor myself and I haven’t had those bone complications, but if I did I would be just terrified to do stuff. But yet, exercise is good for us, right?

Melanie House:
Well, I think‑‑yes. Exercise is good for you as long as it’s in the proper dose, right? And so it needs to be the right intensity, the right frequency, the right load. And so that’s where you really need to work with a professional who has good understanding of where your lesions are and understands the different biomechanical principles. You know, how the muscles might pull on that bone, that could be good or bad. How posture or lifting technique might impact your fracture risk.

So it’s important that there is a professional who’s knowledgeable working with you, a physical therapist that has access to those films or those scans to help inform them giving you the proper prescription for exercise.

Andrew Schorr:
A couple more questions for you now. So some of us know my friends Jack Aiello, who was treated with transplant years ago. He’s doing great. Also like you, Jim, a long‑term survivor of myeloma, but he was left with neuropathy, so he walks with a cane, sometimes he uses a scooter. But yet, you know, he’s aging like all of us and he needs exercise for his body. So what about if you have that complication of neuropathy, which some people do with myeloma?

Melanie House:
As far as exercise, we can find some form of exercise that’s safe anywhere along that spectrum. That all depends on the person’s balance response, their tolerance for weight bearing through their legs because some people have not only those sensory changes but they have more painful kinds of sensory changes with weight bearing.

So, again, it’s very specific to the patient, but the one thing I do want to emphasize about neuropathy is it is not a‑‑I have a lot of patients who say to me, well, I know my balance is bad because I have neuropathy, end of story. And I say to them, well, actually, you know, we have the potential to improve your balance because fortunately your brain is still connected to your muscles through your nerves, and we can recruit other muscles and help them work more efficiently together to improve your balance response.

And so I actually train my patients with neuropathy so that they can improve their balance and have heard countless reports back from patients who were discharged from the hospital and gone on to do outpatient therapy and recovered balance that they never thought they could.

Andrew Schorr:
Wow. How do you do that? Is it like practicing standing on one foot, or give us a clue?

Melanie House:
Well, that is actually‑‑I’m a very practical person, and I work with people that are laborers. You might work with a truck driver or somebody who is a farmer, and these aren’t individuals that are typically going to see (?) a gal at tai chi or something like that, and it is that simple. But if you can challenge yourself in single‑limb balance and do it safely that is really going to force your nervous system to have to respond more quickly and efficiently.

That is actually the test that I do and the exercise that I prescribe, but I set them up to do it safely. So if you can do this test and this exercise standing in a corner in your home where two pieces of dry wall come together with a chair in front of you then you’ve got the walls that can catch you behind and to the sides with the chair in front of you so that you can catch your balance if you need to and when you need to.

Yes, single‑limb balance is a great way to challenge ourselves. And you might get the feedback, well, I never stand on one leg, and to that I say, actually when we walk we’re standing on one leg over and over. So it does prepare a person to be better on uneven surfaces, slopes and conditions like that.

Andrew Schorr:
Okay. And we were talking about bone complications, and obviously if you’re worried about these lesions and you fall, which you might if you don’t have the best balance‑‑

Melanie House:
Right.

Andrew Schorr:
‑‑and that triggers more bone issues.

Melanie House:
Correct.

Andrew Schorr:
So we don’t want to really understate balance is important, and many of us and the people typically, not always, with myeloma are older, where balance isn’t as good anyway. So balance, we got to think about balance, right?

Melanie House:
Very important. Very important.

Andrew Schorr:
Okay. All right. Let’s talk a little bit about nutrition, Alexa, for a minute. So there you are in the transplant unit, and Melanie was mentioning a number of patients who come through are people being treated for myeloma. When you get blasted or even with less intensive transplant there are a lot of issues about feeling like you can eat. Maybe you have mouth issues, pain, etc.

So first let’s talk about somebody getting ready for transplant because that’s still used in myeloma in some quarters. How can somebody fortify themselves if they’re told, well, transplant is what we’re recommending for you?

Alexa Welch
So one of the most important things we want patients to be aware of before transplant is maintaining your weight. Try not to lose any weight. We don’t want you losing muscle or losing strength at all before transplant. So eating a well‑balanced diet while you can, while your appetite is still good. Eat from all the food groups. Get your fruits and veggies in. Get your proteins in. Keep your muscles strong. Keep your weight up. That’s pretty much the coming into transplant being prepped and as strong as possible.

Andrew Schorr:
Okay. But you’re sick going into transplant, so is this like I don’t want to say force feeding, but mean if there’s a care partner there, are they saying, George, eat your vegetables. You’re 72 years old. I mean what‑‑is it‑‑you have to make an effort I guess.

Alexa Welch
Yes, so actually most of the time when I see patients present on day one of hospital admission they are usually feeling pretty well and have been eating well at home and actually have not been losing weight usually. So sometimes when they’re first diagnosed they’ve lost some weight. They weren’t eating well. They were tired, they didn’t know why. That is usually behind them before they come in for transplant.

So typically actually when they get here they are feeling pretty good and have been eating pretty well. It’s going into their admission where they’re getting the chemo and they’re getting transplant that they start to not feel very well again.

Andrew Schorr:
Okay. Let’s talk about that. So people‑‑and of course we have groups in myeloma going through different kinds of treatment. Let’s talk about transplant for a second. If they’re on your unit how do you help them with their diet when, let’s face it, this is rough business. And Jim’s been through it four times. We’ll talk to him about it. But from your point of view how do you help people stay strong?

Alexa Welch
Yes, nutrition is very individualized just like Melanie was saying can for exercise. You just kind of have to figure out where the patient is and what they’re struggling with most. Some of the most common side effects are going to be loss of appetite, mouth sores, nausea, vomiting. We kind of take each of those individually.

So loss of appetite, typically we recommend doing smaller meals more often throughout the day instead of forcing yourself to eat three big meals. When you don’t have an appetite and you’re not hungry and you’re forcing food down sometimes it’s easier to force a smaller amount and try that every couple hours instead of sitting down to a big, overwhelming meal that you can barely even get three bites down and then you just feel hopeless because there’s no way you can finish all that food at once.

So sometimes just having snacks like peanut butter and crackers or fruit and cottage cheese or something small like that and breaking that up throughout the day helps get in enough calories and protein so that you’re not losing weight or losing strength. So usually that is what we do for loss of appetite when you are kind of force feeding. And then when you get to that point we’re not really super worried about eating from all the food groups, so if you’re not able to get your fruits or your veggies in for those few days I’m not going to be super concerned. Or a milkshake is the only thing that sounds good, then absolutely we want you getting your calories and getting your protein that way.

Andrew Schorr:
I’m glad to hear you say that because my‑‑my little kid when I went through chemo would bring me a great chocolate milkshake and I didn’t feel guilty at all. So that’s okay. You’re giving us permission.

Alexa Welch
Absolutely, yes. Absolutely. And I think most of my doctors and team agree with that, that if that’s the only thing that they can get down, then we’re definitely not telling them that they cannot have that.

For nausea and vomiting, usually our pharmacists and our doctors have medications that they can get on board to help, antinausea, antivomiting medications that help control that. And then from my end I just make sure my patients know that right after they get a dose of that medication is when they should try to order some food or eat some food so that that’s fully kicked in and they can try to get as much food down and keep it down as possible. Obviously, if you’re force feeding yourself and it’s going to come back up, it doesn’t do any good. So medication does usually help control the nausea. We’ve just got to make sure that we find the right cocktail for them.

Andrew Schorr:
All right. Post‑transplant, and this may be for people who are on these two‑, three‑, four‑drug combinations now for myeloma, what are you recommending now for a healthy diet? We’re doing some recipes on our website and people say try this, try that, but what are you recommending so that people can regain their strength or be as strong as they can because they’re probably getting some ongoing medicine?

Alexa Welch
Yeah, so appetite usually is kind of slow to come back after transplant. I do hear from my patients who have left and then either come back for a second transplant or hear from our outpatient dietician that works with them that going home just helps your appetite too. Being able to eat your own food in your own home helps a lot. Usually as soon as appetite comes back patients are able to kind of eat, you know, back to normal, back to three meals a day instead of snacking throughout the day.

Recovering, honestly, is still just adequate calories, adequate protein so that they’re still not losing weight. I still never encourage weight loss even after transplant is done because that can be muscle loss and can affect your strength overall. We want you to not be losing weight after transplant as well. And then in general I do a food safety education with patients before they leave the hospital, so making sure‑‑you know, because after transplant your immune system is still not perfect, and we want to make sure that we are eliminating as much bacteria from the food you’re eating so that doesn’t cause any issues, you don’t get any food‑borne illnesses. So we go over that kind of stuff.

Besides the food safety and then adequate calories, adequate protein, you know, weighing themselves, making sure they’re not losing weight. That’s pretty much it. We just want you to stay strong and make sure you’re eating well. And then once you are feeling a little bit better focusing again back on that balanced nutrition, so eating from all the food groups and getting your fruits and veggies in and all that.

Andrew Schorr:
One last question for you now. So there are these products you can get at the supermarket, you know I don’t know the different brands, Ensure and I’m sure there are other brands, high calorie. Do you recommend that to people if they’re not eating a plateful of food?

Alexa Welsh:
Yes, absolutely, especially when they’re in the hospital and their appetites are bad and they’re not eating very much food or they can’t force down solids sometimes liquids do go down better. We use Ensure here. That’s just who our contract is through, but Boost is an equivalent. Equate, or the Walmart brand make their own. That’s an equivalent. I think Costco and Sam’s will have their own.

They all essentially serve the same purpose, which is higher calorie, higher protein in a smaller amount and so that you’re not again having to force feed yourself all day long when you don’t feel well. I would say those are indicated again when your appetite is not very good or you’re having issuing with nausea and vomiting and maybe that’s the only thing that stays down. But once your appetite is back and you’re eating better those aren’t really necessary as soon as you’re able to maintain your weight on just food.

Andrew Schorr:
Okay. We have a lot more to talk about about food and exercise, but Jim’s lived this. So, Jim, you’ve been through transplant. You’ve been 26 some‑odd years. You’re riding a bike, but you’ve been in and out of hospitals and you’ve had your highs and lows. First of all about exercise. Jim Bond, what would you say to people about the benefit of exercise when you have this diagnosis?

Jim Bond:
I’d say it’s one of the key reasons that I’m alive today. And, Alexa, I agree with everything you said, and I’d just like to add a couple personal notes on my diet. I have gone through four stem cell transplants, and what I made myself do is get out of bed, starting with the first one, and it was hard because I was knocked down with the drugs they gave me. But I found that by getting out of bed and then when I was able take a few steps, and then walking around the floor pulling my IV behind me, it gave me‑‑it gave me‑‑it would tire me out, keep me from sleeping in the afternoon, and it actually helped stimulate my appetite. So I would recommend that you try that as much as you can.

If you can’t get out of bed yet just make yourself‑‑I made myself sit up in the bed as long as I could, and that sounds trivial but at times it was not trivial. And, Alexa, my wife, Kathleen, who is my caregiver, she found a high‑calorie, high‑protein drink that she brought in, and I found different flavors work for me. Orange was my favorite. But that was‑‑to me is what was key.

I found what appealed to me food‑wise, and I just ate as much of that as I could. I didn’t worry too much about three food groups. I was too sick. But when I found something that worked for me I would do it. I would also order all three of my meals when I woke up in the morning, and when they arrived that gave me the motivation to, okay, try something. If I put off ordering, then I might not even have the desire to order. So that was a little bit helpful for me.

But exercise has been key throughout my battle with cancers. In fact, exercise saved my life, as you referred earlier. I was‑‑I was 64 years old. I had lived with myeloma successfully for I don’t know many years, and then I got leukemia. And it was the kind of leukemia that’s treatment related and they said, hey, Jim, the only way you can live is by getting yet another transplant.

So they threw me in the hospital for what turned out to be three months solid. They got my leukemia down. They found a match on the matching database, and they came in my room, and I was thrilled. I said, great. When do I get the stem cells? And they said, well, we’re not sure you can live through another transplant, and I said, but that’s the only way I can live. And they said, but we can’t kill you.

So I pleaded my case. They came back and they said, Jim, the doctors who were voting against you on our committee, they changed their mind and voted yes when they heard that two months ago you cycled 328 miles, four days in the American Cancer Society Pan Ohio Hope Ride a month ago. So the exercise of not only training and riding in the bike but just every day doing something, that saved my life because they were not going to give me that‑‑turned out to be a German woman’s stem cells. They said I was not a good risk until they heard what exercise did for me.

And that’s really been true all through this thing. By exercising, doing something every day, I think it made my body able to take more and more treatments because, as we know, today myeloma is still not curable so when it comes back I want to be as strong as I can to make myself tolerate another one. Now, each day what I think of as my mantra is to be on my feet not on my seat. And right now I’m standing up talking to you because I think even standing is better than sitting. And Melanie’s great guidance at a seminar we were at helped me understand that walking is really good for us and standing is better than sitting. Sometimes it’s hard, but I make myself do that.

Andrew Schorr:
Right. Oh, boy, what a great story. And now let’s go to the guru here, Melanie. So, Melanie, I got as a Father’s Day gift a Fitbit. Somebody may get a bigger one, a smaller one, an Apple watch or just count their steps somehow. So today Esther and I are in Quebec City, where we are partly on vacation. We did 11,000 steps. And I’m a two‑time cancer survivor, chronic lymphocytic leukemia and myelofibrosis.

So, Melanie, just walking, is that good? I mean, I didn’t jog and I didn’t lift weights today, but I walked.

Melanie House:
So that is a huge accomplishment, especially when you think about what you achieved by walking. Something that people don’t realize is that‑‑earlier you mentioned the importance of load bearing to the bones in order to stimulate bone density. Well, people don’t realize that when we’re walking because of our body weight and the influence of gravity when your foot hits the ground your bones actually experience about one and a half times your body weight. So you are actually doing an appropriate dose of loading in those long bones in your legs, for example. So you’ve gotten some weight bearing in. You’ve gotten some endurance exercise in. Helps to build your cardiovascular system.

And the other thing is that walking I do want to mention because a lot of my patients, they’re very fixated on walking and I applaud them, but if we are trying to prepare people to be able to do other things like climb their stairs, then we do have to add a different type of exercise to prepare them for that.

Andrew Schorr:
Okay. What’s that? So how do I‑‑or our friend Cindy (?) Chimileski and some of the other myeloma patients have even done these mountain climbs, which have been incredible. But how do you prepare for climbing? Steps or mountain?

Melanie House:
So as it turns out, you practice for the test for most things. So if what you need to be able to do is climb stairs we need to either be climbing stairs while you are in the hospital, or in our case because we know that our patients are prone to getting low blood pressures while they’re here, it’s usually I think a side effect of the chemotherapy, then we have gone to what what’s called the NuStep. That’s the name of an exercise machine that is basically a seated stepper. So that is one way that we’re able to get people working on their stair climbing muscles in a safe with way while they’re hospitalized.

But even an exercise like bridging that’s something that can be done lying in the bed. For my patients that can’t get in the hallway we’re doing a bridging exercise which is working all of the same muscles at zero percent risk of falling down because they’re already laying in bed.

And some people like to do squat exercises which can be done and should be done over a chair or over the bed. But the one precaution there if you are dealing with fluctuations in blood pressure is if you’re doing that sit‑to‑stand motion repeatedly that could bring on that sense of light‑headedness or weakness because of the drop in blood pressure.

Andrew Schorr:
We talked about bone complications, and we were talking about people going through transplant, different medicines. So we have highs and lows with any of these blood cancers. So you and I were talking before the program and you were talking about people being aware of their numbers, their blood test numbers.

Melanie House:
Right.

Andrew Schorr:
So talk about that a little bit as to us having a clear idea of where we are, not just do we have a lesion in a bone somewhere but about our blood.

Melanie House:
So the most common complaint that I hear people say is I’m just so tired, or I get short of breath when I’m doing stairs or walking, and I think there were each some participants today that sent in some questions asking about what can I do to address my shortness of breath. And the first thing that I think about as a clinician is where are your numbers at for your hemoglobin or your red blood cell count, because our red blood cells, they are the vehicles that actually deliver oxygen to our muscles and to our brain.

And one of the most important muscles that must get oxygen is actually your heart, and so it is important to recognize whether you’re anemic. If you’re anemic I can tell you right now there is not a single reference that I could find that would support you or support me prescribing you vigorous aerobic exercise because anemia means you’re at about half of your normal amount of red blood cells yet you’re trying to do vigorous exercise. The muscles that are doing the work are going to aggressively be pulling those oxygen molecules off of the red blood cells, but you only have half the number of red blood cells that you should have to deliver oxygen.

So it doesn’t matter if your oxygen saturation probe says you’re 100 saturated. That just means that those half of your red blood cells that you have happen to be fully loaded, but there’s not enough of them to safely do vigorous aerobic exercise, and your heart could suffer the consequences. I’ve had patients who actually did induce a heart attack just from walking at a time when their hemoglobin was very low and when their blood pressure was low.

Andrew Schorr:
Okay. So let’s go over a couple things we talked about with you. One is related to bone complications, understand where you have bones that are at risk.

Melanie House:
Correct.

Andrew Schorr:
Right? Okay. That’s the first thing. And hopefully there are bone‑‑there are medications now that some people have discussed with their doctor that can try to slow the progression of those bone complications. Okay. So that’s part one. Part two is you talked about balance. That’s so important. Even if you have neuropathy don’t be freaked out that you can’t develop balance. And then related to knowing your blood counts so that what you’re pushing your body to do is healthy.

Melanie House:
Correct. Right.

Andrew Schorr:
Okay. All right. Got it. We’re going to come back for some more. I want to get some specific exercises. So walking is good. Climbing, if you have stairs in your house, those kinds of things, or if you’re training for one of these myeloma challenge trips, whatever it is, we’ll talk about that more in a minute.

Alexa, so we talked‑‑you keep saying, you know, fruits and vegetables and balanced diet and all that, but patients we have are friends in the myeloma community say, well I’m going to do this special diet in their effort to take back to control where cancer has kind of tried to take control away from them. So how do you feel about special diets, whether it’s meat, vegan, you know? How do you feel about that?

Alexa Welch
So some of those diets just end up being overly restrictive or totally cut out certain food groups, which is not‑‑I mean, there is just not enough evidence out there to support any of those restrictive diets actually really helping. Cutting out food groups like that sometimes results in weight loss, which, as I have mentioned a few times before, that’s definitely not the goal. We don’t want you losing weight. Don’t want you losing muscle.

And a lot of times when you’re sick and you have cancer and you’re going through treatment, any time you’re losing weight unfortunately it’s muscle loss. It’s not fat loss. And so then again that results in weakness and poor outcomes as far as response to treatment and recovery. So, yeah, some of those special diets, I mean, I would have to take it patient by patient if they feel very strongly about it, but, yeah, a lot of times they’re just really restrictive on certain food groups that they can’t have or should cut out totally. So I don’t usually recommend those.

Andrew Schorr:
Okay. So a couple of questions. Maybe these are myths or not. So some people have wondered, does sugar intake feed the cancer cells?

Alexa Welch
So a lot of the foods that we eat, all carbohydrate food, so whether it’s fruit, grains, rice, milk has carbohydrates in it, any carbohydrate that we take in will break down to a molecule called glucose, which are‑‑all of our cells in our body need glucose to function properly. It’s the energy that they use. So whether those carbohydrates are coming from sugar, artificial sugars or added sugars or natural sugars from fruits, they all break down to glucose.

We cannot control which cells get the glucose that we take in. Once we eat it, our body does with it what it will, so the cancer cells just happen to be very glucose hungry all the time, so they will take up and use a lot of that glucose. That being said, if you’re not eating enough glucose or not eating enough carbohydrates in general your body will break down your muscle stores to get that glucose.

And that is why you don’t want to be restricting certain food groups, especially carbohydrates because the rest of your body still needs the energy to carry on the normal functions of everyday life. So you shouldn’t be cutting out some of those food groups like the carbohydrates that are fueling the rest of your body too.

Andrew Schorr:
Okay. Another question, juicing. So people have all kinds of‑‑there are juicers you can buy, and your best friend down the street will say, oh, you’ve been diagnosed with cancer and you should be juicing, carrot juice and this juice and that juice. Any comment about that?

Alexa Welch
Yeah, so I just don’t see the issue with eating the whole food is. The whole fruit or the whole vegetable that you’re juicing, you’re taking out a lot of the fiber. You’re taking out a lot of what keeps you full, the substance to it, so then you’re having to spend a ton of money on groceries relies to get less benefit, if you ask me, because you’re taking out, again, that fiber that’s very beneficial for keeping you full, helps cholesterol.

So those are not things that you want to be leaving out of those foods that you’re taking in. You still get all the vitamins, all the minerals from those fruits and vegetables, but, yeah, eating the whole thing is more beneficial.

Andrew Schorr:
Okay. You were being conscious of our diet at the grocery store. What about the health food store, the pharmacy about dietary supplements? Comments about that?

Alexa Welch
Yeah. So one thing to be careful about any over‑the‑counter supplements like that are not FDA regulated. So you want to be careful that if you’re taking any dietary supplements, herbal supplements, any extra vitamin, C, A, whatever, that you’re clearing that with your doctor, your physician, your oncologist, your pharmacist, talking to your medical team about that and making sure that they are okay with you taking those extra supplements. Again, they are not FDA regulated, so just because they say something is in it, that hasn’t been tested. So you want to be very careful about that.

And some of those supplements can interact with certain chemo drugs. There are certain medications that you might be on every day, so you want to again clear that with either a pharmacist or a physician to make sure that it’s okay if you’re going to take any supplements like that.

Andrew Schorr:
Okay. So when I go to the gym they have a little store in the front, and they have those huge jars of protein powder. So you’re saying even that, check with my doctor.

Alexa Welch
Yes. Especially‑‑I mean, you want to make sure that if you’re going to do the protein powders like you want to make sure that it’s a brand that you trust. So in general bigger brands like Walmart’s brand or some of the‑‑like Abbott, who we get Ensure from, they have their own brand of protein. Some bigger brands like that are going to be ones that you can trust because if they were putting‑‑you know, you hear myths about people having like actually sawdust in their protein instead of real protein powder.

So those are the kinds of things you want to avoid. Usually big companies like that are more trustworthy because if they were found to have bad ingredients in their protein powders they would have more to lose essentially than some of the little companies you’re buying online that you don’t want to necessarily trust. Generally, if it says 100 percent whey protein 100 percent soy protein, those are a little bit more trustworthy.

And always, again, good idea to just run it by your doctor make sure they’re okay with it, or ask the dietician to read the label for you. Some grocery stores have dieticians that work there. Some gyms have dieticians, so use your resources.

Andrew Schorr:
Right. I will mention to people now, so we go to this ASH, American Society of Hematology medical meeting, thousands of doctors talking about myeloma among other cancers from around the world, and so now we’re talking about often four‑drug combinations for people with myeloma. So if you go into a store they don’t know that you’re taking drug A, B, C, D. They probably never heard of them nor know the profiles of those drugs and how it will line up with something they’re going to offer you. You’re not just a super healthy person who is taking no drugs coming off the street, so you have to check.

Okay. So, Jim, you’ve been listening, and you’re about to ride in a couple of days again 328 files. Now you’re of course just a subject of one, but, Jim, what do you eat? What is your diet, whether it’s when you’re doing these rides or just day in day out?

Jim Bond:
I get asked that a lot, and there’s a lot of people that really do focus on special diets. I do not focus on anything special in my diet. I focus on trying to maintain my weight. I do exercise, and for some reason since I’ve had cancer and the transplants I really have to make myself eat as much what I consider healthy food. For example, my lunch today consisted of a meat sandwich, potato chips and an apple. And that’s typical. And for breakfast I eat eggs, meat, toast and potatoes, which is‑‑turns out to be my best meal. It’s my best appetite. And a normal dinner, you know.

Yeah, we have vegetables. We have meat. I love corn on the cob in Ohio. It’s great. But I don’t worry about anything really special. I want to keep my weight up. So when I go in, and I do go in monthly for a bone strengthener I’ve been getting for 24 years now, the biggest surprise for me is, okay, how did I do on weight this month. And when it’s higher I’m happy. And typically the nurses frown at me because they’re trying to lose a little weight, but I’m always trying to maintain or keep my weight.

Now, another reason‑‑I do pound a lot of liquids. My kidneys, I was told, because of the type of myeloma I had, I was told, look, Jim, your kidneys and your bones are at risk. So they said drink, keep yourself well hydrated especially when you’re riding your bike in the summer in July in Ohio. So I drink a lot of water with something in it, you know, a Gatorade or something flavored, not just pure water. But that’s really important to me.

And yeah, it’s inconvenient. Gets me up a lot at night, you know, going to the bathroom, but I believe it’s worth it. And it drives my sodium down. When I get my chems every quarter my glucose and the rest of them are fine, but it’s all I can do to get my sodium into the normal range. So, believe it or not, even with the doctor’s okay they said, Jim, eat more salty foods, which I know is kind of weird, but that’s the way I roll, and so I really don’t worry about that.

It’s the bones though. I do worry about my bones. I’ve had a lot of bone involvement. I’ve got metal holding some of them together, but I’m lucky enough to be able to walk on my own, ride my bike. But it’s taught me, Jim, cut down on the risk. Stay off ice. Stay off step ladders, stay off stools. It’s just not worth it. So I try to do that.

But one comment you made is be sure to check with your doctor on what seems like it’s something that’s not worth it. Green tea is a good example of something a friend of mine who is a myeloma patient had no idea he should have cleared that with his doctor. Because he thought green tea, that’s fine, only to find out from his doctor, no, the medication he was taking was actually nullified by the green tea. So it’s really a good idea to run what you think is not very harmful, run those things by your doctor or nurse and make sure they’re okay with that.

But every case is different, like you said, (?) Jack, and for some reason it’s worked out pretty well for me. But I do take a few‑‑I do take a few vitamins that leading hospitals have recommended, and they’re for neuropathy and hopefully to keep the myeloma away. And I’m happy to share things, but you can get me‑‑you can find me on the internet or through somebody.

But the thing is you can’t just willy‑nilly take things. You’ve got to run them through your medical team because your case is, you’re own case, each case is different, and, sure, it’s great to talk to people but just run it by somebody.

Andrew Schorr:
Right. All good points. Okay. We’ve been getting in questions, and if you have a question now send it in to myeloma@patientpower.info, myeloma@patientpower.info.

Here’s a question we got in from Laurie. Laurie says, my husband has 13 vertebral fractures from his myeloma. He’s not a candidate for the various surgeries (?) Inaudible, kyphoplasty, etc., to do repairs, so he’s been doing plank exercises for two years, and he has a brace and support.

So one of the things he’s wondering is could tape, Melanie, like athletes do, kind of some kind of taping when he does exercise be supportive for him? Would that be a good idea, like athletic tape?

Melanie House:
Well, actually it’s a little bit different. I think the tape we’re referring to is a little different than athletic tape. It’s called Kinesio tape, and it’s been around since the 70s actually, and it is something that has been shown to help with musculoskeletal types of pain, so it could be worth exploring. If this is the same question I’m thinking of, this individual complains of the pain that radiates around the bottom of the ribs.

Andrew Schorr:
Correct.

Melanie House:
That sounds like it’s probably one of the intercostal nerves that could have some compression on it perhaps due to where the vertebrae has lost its height and therefore the rib is getting compressed and maybe pressing on a nerve. So yes, there’s some potential there. If there could be some lift appreciated on one of the ribs or just to create a little more space there.

The other thing that I thought about is when we’re laying flat‑‑I hear this all the time. I just had a patient today say to me, well, I was six‑foot‑three but not anymore, and this individual just had some back surgery done, fused his lumbar spine. And so I explained to him that it’s best if we put his back brace on when he’s laying down because that’s when the vertebrae are off‑loaded so your disks are at their maximal height, and if you can put a brace on laying down and put it on so it’s comfortable but snug, once you sit up you’ve done the supporting that you’ve needed to before everything tries to collapse.

Andrew Schorr:
Okay. Good points. Mike Furlow sending this question. He said he discovered myeloma when a plasmacytoma broke my humerus near my shoulder. My bone scan and CT scan showed no other significant lesions, but he later found significant damage to my right ankle during the surgery. So he’s wondering, is it safe to assume I have damage elsewhere? He just doesn’t know what to do. And so do I have to be particularly careful about bone injuries going forward. He’s worried. What do you say?

Melanie House:
Yeah. This sounds like a classic case where you know there’s got to be‑‑there could be some other problem in there but you can’t see it, you don’t know about it, so that fear creeps in, and that could paralyze somebody really from doing exercise that could be benefitting them.

So I would definitely recommend that he meet with his doctor or primary care provider who has access to his films, his recent scans, so like a whole‑body MRI or the PET scan, and go through, where are the lesions that I should be concerned about, and how would that guide my exercises or working with a physical therapist to come up with a safe program. Because if you don’t know where they are and you fear that there’s something electric there, I’m going to do the same thing. I’m going to think it’s safest to stay in my recliner probably.

Andrew Schorr:
Okay. So Jim mentioned a couple‑‑he mentioned a lot of significant things a minute ago, but he was saying that he knows given his bone complications there’s certain things that he’s going to avoid. He lives in Cleveland. In the winter he’s going to be real careful about ice. And if his wife says, gee, can you change a light bulb up there and it means going up on the step ladder, he’s not doing it. Okay?

Melanie House:
(?) And she probably wouldn’t ask.

Andrew Schorr:
She won’t ask, right. So the point is what about changing sort of activities in daily living so you can be active but be safe?

Melanie House:
Well, the first thing that comes to mind, and this is again going back to where I am most concerned for my myeloma patients, and that is the vertebral fractures because I‑‑it’s just‑‑it’s so sad to me when I see folks losing, progressively losing their height knowing it’s because these vertebrae are literally collapsing, and the biggest force that causes the collapse is flexion.

So when you think about in your daily life how often do you have to flex. Oh, I have to bend over to put my shoe on, I’m pulling my sock up. Oh, I dropped the paper, or maybe I’m picking something up off the floor that normally sits there like the food bowl for my cat. And so these motions can result in significant pressure forces going down the front of the vertebrae that actually lead to their collapse.

So one way that a person can change the way they’re moving throughout the day is hamstring stretching is a good start because the longer your hamstrings are the less you have to flex through your lumbar spine. But for others it’s beneficial to even use adaptive equipment. Like our occupational therapist will train people on how to use something called a reacher, and that just allows you to be able to bend over safely but not bend too far and still pick an item up so you’re at less risk of losing your balance and falling but also less risk of causing those flexion compression fractures of the spine.

Andrew Schorr:
Well, so you’re saying don’t bend down for the cat’s bowl. Maybe there’s some grabber or something will help you do it?

Melanie House:
Well, in that case‑‑I mean, there’s different ways to approach it. If you can squat rather than bend. The thing is that we all have our habits, and we don’t even realize what we’re doing until we see a video of ourselves or someone points it out. But if you know you’re at risk for compression fractures in your spine, going through some training to actually learn what ways could I move differently, what strategies could I use that are safe and still let me do the things I need to do, there’s always a way to accomplish it. It’s just that it’s very individualized for each person.

Andrew Schorr:
Okay. Remember, send in your questions to myeloma@patientpower.info.

Here’s another one again for you, Melanie, from Paula. Any thoughts on interval training or other techniques to help my body use oxygen more efficiently? So interval training, that would be like running for two minutes and then walking and running or longer. If you kind of start, stop, right?

Melanie House:
Yes. And interval training, I use interval training for patients in the hospital who can only walk 10 or 15 feet and have to sit and rest. We can call that interval training. Or, like you just said, it could be something like being on a bike or walking or jogging where you’re just doing that higher intensity and then you do the lower intensity.

So for each individual you have to find that right combination of exercise that’s still safe for you, but the first thing that I would think of in this question is again back to, okay, what are your lab values? If you’re hemoglobin is low, if you happen to be anemic, then you really do have to listen to your body. If you’re feeling short of breath, you should not be pushing through that.

So, yes, interval training would actually help you to build your endurance and your total distance that you could walk, and that to me suggests that you’re listening to your body and you’re slowing down when your body is telling you need to.

Andrew Schorr:
Okay. Alexa, lab values are not just about hemoglobin, but they’re also about creatinine. Jim was talking about kidneys. Certainly patients are at risk for kidney problems. My doctor says every time I see him, Andrew, drink more water, drink more water, drink more water. Jim was talking about that too. And also looking at whether we’re getting enough of different minerals as well. So that can show up in our lab values too. We should be aware of those, right?

Alexa Welch
Absolutely. Your doctor can test you for any vitamin deficiencies as well. Yep, your labs are very telling for, you know, if you’re hydrating properly, if you’re well nourished. But yeah, so definitely I think you’re doing the right thing staying hydrated and making sure you’re getting enough liquids. That’s definitely the best thing, one of the best thing for your kidneys.

Andrew Schorr:
There you go. I’m going to drink some more in a second. But I‑‑so, Alexa, and this is for you, Melanie, as well, but first you, Alexa. So what do we do? So you all are at the big university medical center, but even at clinics they often have a dieticians. Increasingly now some of the larger clinics have a physical therapist or maybe consulting one nearby.

Should we consult with you, not just if we’re having a transplant but we’re there for a clinic visit? Can we say, I’d like to see the dietician? I’d like to see the physical therapist because I want to be strong, I want to exercise, I want to eat right. I mean, that’s something we can request, correct?

Alexa Welch
Absolutely. I know here we have a dietician who works out patients specifically just for our cancer patients who are here for clinic visits. Usually her schedule is pretty flexible, and she is able to add patients on same day. So obviously I don’t know how it works everywhere, but every patient should be an advocate for themselves and how they want their treatment to go. So if they’re losing weight and they know they’re not supposed to, then you meet with the dietician and see what they can do differently for calorie boosting, for protein boosting, and same thing I’m assuming with physical therapy as well. You need to be an advocate for yourself. Ask for those consultations if you’re not offered them.

Andrew Schorr:
Melanie, you’d say that?

Melanie House:
Yes. I would agree. And the other thing is actually for physical therapy in most states it is a direct access option for you so you don’t often‑‑depends on what state you live in, but you don’t always have to have a doctor’s referral to be seen by a physical therapist.

That said, you’ve heard me say over and over, if I’m recommending that you see a physical therapist I want that therapist to actually be well informed of your past medical history, any of your lab values, any of your films and your imaging. So some facilities will still request PT counsel just so they have that physician connection and can get all those types of things that they need to know.

Andrew Schorr:
Right. Right. So, again, wherever you may be in the sound of my voice, if you will, all physical therapists are not equal. Melanie is an oncology physical therapist now, and she’s going to understand the risks you have in myeloma. We talked about bone, balance, the lab values, etc., maybe even complications from treatment you’ve had or medicines you’re taking. So somebody needs to see the whole picture. Same with a dietician, right?

Alexa Welch
Yes.

Andrew Schorr:
So trying to see people get the whole picture is important. You’re not going to have that at the health food store. You probably won’t have that at the pharmacy. You need to seek out somebody who’s knowledgeable about this.

So, Jim, a little bit about cancer patient consumerisms. You’ve had to really speak up for yourself. First of all, play a role in your care and speak up for yourself so you get the care you need and deserve. What you would you say to our listeners today so that when you think about diet, exercise, and going on with their life, which many people, and you’re a great example, now with myeloma can, what would you say to them so that they advocate for themselves to get consultants like these on their team?

Jim Bond:
Everybody’s different, and I believe everybody should handle their case the way they’re comfortable. Here’s what my wife and I are very comfortable with. I want to be an equal partner with my medical team. I don’t want to be the boss, and I don’t want to be bossed around. I want to have an equal vote.

And a good example of that is at about the 10‑year mark I was told here in my home town, Jim, you’re done with any treatments available. So you have to go to a hospice. You’re all done. And I said, no, I’m not going to a hospice. I said‑‑and that made the doctor leave the room, angered, but before he left I was able to say I know of a clinical trial that I had gotten word of in an out‑of‑town second opinion in those 10 years, and I said, I’m going to try to get in that clinical trial. And he told me I was wasting my time. I got in it. I was lucky enough to get in it anyhow. Had the leave town.

And I think that’s one of the great examples of being an equal partner. Okay? The doctor had certain advice, and it’s happened before in the 26 years. But I spoke up with my wife’s support, and I said, look, what if we tried it this way? What if we tried that three‑drug mix but without the steroid because I don’t really like to take steroids once I learned it caused one hip to have to get replaced. And the doc says, you know, I’m really not that keen on that, Jim, but I’ll go along with you if you want to run the risk. I said, yeah, I really do, and if it doesn’t work, if the numbers go up, we can always add the steroid later. And, you know, two months later the very popular myeloma doctor called me back and said, Jim, good call on your part. It worked fine without the steroids.

So advocate for yourself. Don’t be afraid to get educated. There’s lots out there. But if you don’t want to that’s okay too. If your way of handling it is different than that, I believe that’s‑‑your way is right for you.

One thing I’d add to the stretching and the back stuff. I’ve got severely curved spine. I’ve lost at least three inches of height, and I’m sure I’m at risk for something back there. But they don’t tell me, Jim, don’t bend or don’t do this. Well, I do stretch my hamstrings daily. That’s very important to me. And what I’ve learned to avoid is lift‑‑I don’t lift heavy objects. So how do you go along with your life? Well, you figure out ways. You know, it’s great that we have luggage that has those rollers on them. I have a briefcase that has rollers on it because I feel the pain. If I lift I’ll feel the pain the next day. So I stay away from lifting.

But, no, I just go ahead and do things. And I try to ask the doctors and nurses, tell me what I should not do, and I listen real carefully and being an equal partner I got to weigh all that, and I let them know where we’re coming out on things. But it’s fine to advocate for yourself and the longer each of you live with myeloma the more you’re going to realize, hey, there’s a lot of flexibility here. No one’s got the answer or we’d have a cure. So there is some flexibility, but you’ve got to use your good judgment and that of your medical team.

Andrew Schorr:
Great advice. So, Melanie, what do you want to leave people with on the importance of exercise wherever you are in your myeloma journey and having the right consultation so you can do what’s wise and what’s safe?

Melanie House:
The most important thing I can say is there’s no better time to start than now, and finding those things‑‑you know, think about what is it that’s important to me? What am I missing out on in my life that I want to get back to, and consult a professional to help them‑‑they will help you achieve those goals to get back to doing those things as best and as safely as possible.

Andrew Schorr:
I’d agree. You know, I have to get immunoglobulin treatment once a month for my‑‑related to my leukemia. Some other people may get that too. And yet in those times like now, in between, I travel. And, you know, so I’m going on with my life and thank god have energy and can do those steps I talked about. So I would urge you to go live your life. And your healthcare team will help you. You can do this exercise or that. And there’s Jim who’s not going to get up on the step stool, but he’s going to do that 328‑mile bike ride for the 12th time. Okay.

So, Alexa, a final comment from you about what you’d say to people about proper nutrition wherever they are in their myeloma journey. What would you say to them?

Alexa Welch
I would say listen to your body. If your body is telling you I’m hungry, eat. If your body is telling you I’m not hungry, maybe not eat but also recognizing that if that’s all day long that you’re not hungry maybe you need to set an alarm on your phone to make sure you’re eating properly. Wherever you’re at in your journey it’s important to listen to your body until your body can’t tell you what it needs anymore, and then after that then you need to start kind of taking over‑‑your mind has to take over and listen to what you need.

Maintaining your weight. And then, like I said, whenever you can eat from all the food groups, and then in the meantime when your struggling to maintain your weight or eat enough overall you want to use those supplements when necessary while talking to your medical team. And supplements I mean by the high‑calorie, high‑protein shakes, not necessarily the herbal supplements.

Andrew Schorr:
All right. Great information. And one great thing I take away from you too is should I need a transplant some day or I’m going through chemo again a chocolate milkshake is okay.

Alexa Welsh:
Yes, absolutely.

Andrew Schorr:
All right I won’t feel guilty about the ones I’ve had in the past. So, Jim, we’re going to leave it with you. So you have this bike ride coming up, the 12th one, for fund‑raising for the American Cancer Society that you wife started in Ohio, god speed to you, Jim. Are you feeling pretty good?

Jim Bond:
I am. I am. I had to shake off a bout of pneumonia a month ago, but I got the clearance to get back on my bike about three or four weeks ago. And they said, take it easy, and if you get tired, you know, get off your bike‑‑and I will‑‑and, you know, I’ve proven I can do it, but, you know. I think we have to all use our common sense on this stuff and live your life like you said. And we can do it. We can be long‑term survivors.

Andrew Schorr:
All right. Well, all the best to you. We are all riding with you, Jim Bond, okay?

Jim Bond:
Thank you.

Andrew Schorr:
All the best. Well, I want to thank Jim joining us from Cleveland getting ready for the bike ride in a couple days. Alexa Welsh joining us from the University of Iowa Hospitals in Iowa City, thank you so much, Alexa. And also Melanie House, joining us once again also from the University of Iowa Hospitals. Great information. Thank you so much.

I want to thank the Patient Empowerment Network for letting these programs flow and what a wonderful service it does to our myeloma community. And I want to thank the companies that have helped fund this program, AbbVie, Takeda and Celgene, thank you for being supporters of the myeloma community.

Remember, there’s a replay that will be available to you soon. Share it with others you know in the community. There will be video clips and sound clips with Jim that will be coming up. There will be a transcript, all coming your way. And discuss it and make sure that you connect with not only other people in the community but people like Alexa and Melanie who are very specialized, who can help you have the right diet and the right exercise for you.

In Quebec City, Canada, I’m Andrew Schorr. Thanks for joining us. Remember, knowledge can be the best medicine of all.


Please remember the opinions expressed on Patient Empowerment Network are not necessarily the views of our sponsors, contributors, partners or PEN. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

How Can Patients Learn About New Myeloma Treatments?

Living Well with Multiple Myeloma

How Can Patients Learn About New Myeloma Treatments? from Patient Empowerment Network on Vimeo.

Downloadable Program Guide

Getting the right cancer care calls for sound, up-to-date information and open dialogue with your healthcare team. As a patient, how do I stay informed about new treatments in development for multiple myeloma? What are the considerations when choosing treatment that’s right for me? In this video, experts will help you better understand the latest multiple myeloma treatments for patients who are actively seeking the best care available.


Transcript:

Jack Aiello:
Hello and welcome. Thanks for joining today’s Patient Empowerment Network program. We thank AbbVie Incorporated, Celgene Incorporation and Takeda Oncology for their ongoing support.

We have a lot to cover, and we’re so happy that you joined us. My name is Jack Aiello, and I am a 23‑year survivor, myeloma survivor, this is. I learned that getting the right cancer care calls for sound, up‑to‑date information and an open dialogue with your healthcare team.

Some questions to ponder as a patient: How can I stay informed about new treatments in development for multiple myeloma? What are the considerations when choosing treatment that’s right for me?

We have already received a number of your questions today, and we’ll get to some of those answers, but first I’m really pleased to introduce our distinguished guests. Dr. Amrita Krishnan is an M.D. She’s the director of the Judy and Bernard Briskin Myeloma Center. She’s a professor of hematology and hematopoietic cell transplantation at the City of Hope. Dr. Joshua Richter is the assistant proper of medicine at the division of hematology medical oncology at the Tisch Cancer Institute of the Mount Sinai School of Medicine. And Kristen Carter is the advanced practice nurse at the University of Arkansas Myeloma Center.

Before we begin answering and addressing some of the questions, I want to make sure you are aware that this webinar is not a substitute for medical advice. You really need to refer to your medical healthcare team. And if you have questions during this webinar you can e‑mail them to myeloma@patientpower.info, and we will try to get to as many of those as possible.

I mentioned I was a 23‑year survivor. I was diagnosed in 1995, and back then treatment decisions were pretty easy because there weren’t many treatments. Either you took melphalan prednisone, a couple of pills, or you went the transplant route, which I ended up doing. I’ve learned an awful lot in 23 years. I facilitate our local Bay Area myeloma support group, and so the questions I’ve seen you already asking today and the questions we’ll be asking our doctors are the same questions that are asked in our support groups as well. So let me begin.

The first question has to do with‑‑we hear about new drugs that are out there that have recently been approved, but how do I learn about drug approvals? What’s the process for approving a new therapy, and should I attend as a patient these medical conventions I hear about like ASH or ASCO, and if not, how do I learn about these new drugs?

Dr. Krishnan:
Yeah, I think people are welcome to attend meetings such as ASCO, but you know there are 30,000 people there and so it’s a fairly overwhelming experience, and it’s very hard to drill down. And, to be frank, a lot of what gets‑‑the mix of what gets presented at most of the national meetings in regard to very, very early‑stage drugs that are only available in clinical trials. And then, yes, we do have Phase 3s that are randomized trials presented where drugs are pretty much ready to be approved or already approved and that’s confirmatory data for those drugs. So it’s a big mix.

I think, you know, for patients in terms of getting the most sort of bang for their buck is sort of doing things like you’re already doing such as the seminar you’re hosting right now I think is invaluable because it really helps drill down all the data for those meetings. And some of the other patient education forums I think are‑‑again because I think we’re happy, we’re always happy to speak at those types of events to help sort of synthesize that data in a more kind of (?) coherent, how‑can‑I‑help‑you forum.

Jack Aiello:
Any additional thoughts on that, Dr. Richter?

Dr. Richter:
Absolutely. I think that the patient support groups from different programs run by the MMRS and the IMF as well as the Leukemia and Lymphoma Society are extremely helpful. There are many of these programs, and if you go to these organizations’ websites there are frequently programs that may be near where patients are.

The other thing that I think is key as part of a patient’s and their caregiver’s myeloma journey is at some point during your treatment it’s really worthwhile to come to a center such as the people represented here. You know, University of Arkansas and City of Hope and Mount Sinai are all extremely advanced in terms of their myeloma knowledge, but there are many others across this country. And I think as patients it’s important to have a deep connection with your care team, and you can still receive all the care with your local team, but at least one point during your journey going to one of the centers like the ones on today’s panelists I think is worthwhile to find out what is on the horizon and how they can work with your local physician and nurse practitioner team to form the best plan for you.

Jack Aiello:
One thing I’ll add on to what you said about the information provided by organizations like the International Myeloma Foundation, like PEN empowerment network, like the Multiple Myeloma Research Foundation is that they have videos and webinars very quickly after ASCO or ASH meetings that will summarize what the major outcomes were at those meetings. And they are intended for patients, and they really are excellent, excellent vehicles for learning.

Kristen Carter, so I have a question targeted for nurses, I think, and that is as a patient how do I communicate or partner best with my doctor on treatment decisions? What do you find that works regarding being diagnosed with something called myeloma, which you’ve probably never heard of, hearing all of these overwhelming terms of IgG and too high a level of protein, which sounded always good to me, how do you‑‑how should patients be interfacing with both their doctors and their nurses?

Kristen Carter:
Well, all my patients have my cell phone number so they tend to call me if they have any questions, but I always tell my patients make sure you write down questions because you know as well as I, when you get in there in front of a doctor who’s got a whole list of patients for the day and they’re seeing you, they’re giving you all this information, and I always call it the deer in the headlight look from the patient because they’re brand new. Writing down questions that you think of is always very important.

Having a family member that’s right there with you, that maybe they’re thinking of things that you haven’t really thought to ask. As we’re going over side effects and treatment decisions, taking notes is very important because I always have patients, and I will have patients four or five years later, go, remind me, what is my subtype. And we go over this every time and you go, I thought we were doing a really good job of educating. So if you don’t understand something ask to repeat the information. And I always repeated back to the patients and have them repeat it back to me. That way I can see if they really understand what we’ve gone over.

And just make sure there’s an open dialogue. I always tell my patients don’t suffer in silence. If you have a side effect we need to know about it. If there’s something you don’t understand we need to know because that way we can ensure that you’re not only understanding but getting appropriate treatment, and if there’s side effects that we need to know that we can make adjustments.

Jack Aiello:
The doctors especially seem very busy and sometimes in a little bit more of a hurry than you as a patient want them to because it’s difficult for you to absorb the information that they are providing you. How do I slow them down? How do I make sure that I do understand what they are saying, Kristen?

Kristen Carter:
Having a list I think is a really good way to slow down.

Jack Aiello:
I agree.

Kristen Carter:
I have patients that come in and they’ll have their list, and I usually go in first. So I work for Dr. Van Rhee, and we have‑‑we manage, actively manage about 700 myeloma patients from all over the country. And so these patients will come in sometimes a thousand miles to see us. We don’t want them to be shortchanged on their time because they’ve travelled all the way from Arkansas, and they’ve done all the workups, and we certainly don’t want them to feel like they didn’t get the time after spending money and travel time to get to our academic center. So usually I will go in first and answer any questions that I can answer, and the list is always so important. And we’ll say, sit back down, we have the list, and what I can’t answer the doctor will answer.

And again I do provide an e‑mail or a cell phone, and I have patients that will e‑mail me a list of questions that I can turn around and answer for them if they didn’t get the information. So I think definitely having a list, having family support if it’s available to come with you, I think that does kind of slow the doctor down.

And if you don’t understand something you just stop the doctor before they leave the room. Hey, I didn’t understand that. You are your own patient advocate, and you’ve got to make sure that you speak up if there’s something you don’t understand or if there’s something you’re not sure about. Or if there’s something you’re not comfortable with in the treatment planning you need to vocalize that with your doctor or nurse.

Jack Aiello:
Dr. Krishnan or Dr. Richter, any other things that patients have done when you meet with them that you want to pass along to patients on this call?

Dr. Krishnan:
I think the one thing to be honest I’ve started writing down stuff that the patient said myself because a lot of patients start getting focused on taking notes, and they don’t want to miss anything, but then it’s very hard to absorb and take notes at the same time. So having someone with you to be your scribe is very helpful. Some doctors, you know, don’t mind patients recording them. Some are less comfortable with that. So that’s something else you can consider is asking your doctor if that’s an option.

I think the other important thing to remember is all the information we get, especially when we talk about transplant, that’s not going to be the first time you hear it, so don’t‑‑it’s not like you need to understand it all right now. This is just information gathering, and that information is going to be repeated again and again by multiple different people.

Jack Aiello:
And Dr. Richter?

Dr. Richter:
I think everything that’s been pointed out is great. I would encourage patients that if they want to record to ask first. We’ve definitely had some patients where all of a sudden their purse starts beeping and I ask what that is, and they say, oh, I’ve been recording you. I have no problem, and most of us don’t as long as we’re told about.

I think it’s also‑‑as much as the care teams set goals for each appointment it oftentimes can be a good idea for patients to set goals of what they want to get out of the appointment. So not every appointment is going to be soup to nuts, everything from the diagnosis to the whole treatment, but this appointment, what is going to be my next step with treatment and how do I deal with my toxicity. This next appointment I want to find out about transplants. So setting a couple of discrete goals I think really helps both sides to accomplish what we need to.

Jack Aiello:
Yeah, I agree. Dr. Richter, you brought up some of the resources earlier. I don’t know, when I was diagnosed in ’95, back then we weren’t sure if the internet was even going to be a success, so resources were quite limited. What do you find patients today using, and how do you‑‑you know, some doctors, patients will tell me, will say stay off the internet, don’t go there, and that’s not the right answer. So how do you advise patients today about that?

Dr. Richter:
So I think that this is something that we can’t avoid. It’s definitely a double‑edged sword. What I always encourage patients when we talk about different things is I direct them to certain sites that I know have vetted information that’s been created by the myeloma community, and it’s very accurate and realistic. So sites from the imfatmyeloma.org and the MMRF, Multiple Myeloma Research Foundation I found to be very important, and both of these organizations have handouts that we often give patients to augment things.

One of the other resources that I‑‑you know, is definitely another double‑edged sword, is clinicaltrials.gov. And I even hesitate to mention this, but I think it’s a valuable resource. I think as patients with myeloma are extremely savvy and oftentimes come in knowing data even before I’ve even heard it. It’s quite amazing. The benefit of clinicaltrials.gov is it lists all of the trials that are done in all of these institutions. It provides some overview about it, about whatever the trial is, some information as far as who may or may not be eligible, and it lists the institutions and sites that are running the trial with contact information.

So I think one of the benefits there is that people start hearing about all of these different trials on sites like myelomacrowd, LLS, MMRF, and if you’re interested in seeing if there’s an institution by you it’s a great way to drill down and find out the closest institution and a contact that may get you the right place.

Jack Aiello:
There are some good front ends for clinicaltrials.gov as well. Something called SparkCures, S‑P‑A‑R‑K cures. Something called the myeloma matrix from the IMF. Something called Smart Patients. And there are also organizations like the Leukemia and Lymphoma Society, like the IMF and MMRF who have clinical trial specialists that you can talk with, that given your situations they will help you to direct you to the right clinical trial as opposed to starting off with clinicaltrials.gov. So, I agree.

Same question for Dr. Krishnan.

Dr. Krishnan:
I think‑‑

Jack Aiello:
Resources that you have found particularly useful to provide to your patients.

Dr. Krishnan:
I think actually Dr. Richter pretty much covered them in terms of the IMF, the MMRF and what you have added to it actually. I learned some more resources too, so thank you guys.

Jack Aiello:
And same question for Kristen Carter.

Kristen Carter:
I always tell my patients to go to reliable resource sites like the IMF and the MMRF because I definitely have had patients call me later. I had a patient that was looking up fatigue in myeloma and he called me, and he goes, you didn’t tell me that I was only going to live five years, because he looked on the internet and it said five‑year survival is 48 percent at that time. He’s nine years in complete remission at this point, but I had to talk him off the ledge because he had been on the internet and he had read that, and after we had already gone over kind of the statistics and things and his individual myeloma. I said don’t worry. Don’t look at those statistics. Let’s worry about you.

So definitely, like Dr. Richter said, go to resource sites that are reliable, like you said. Leukemia and Lymphoma Society, IMF, MMRF, those are the reliable sites to go to.

Jack Aiello:
For those listening, don’t forget you can e‑mail us questions at myeloma@patientpower.info.

Doctors Krishnan and Richter, let me ask you another question. In June it’s always a big month, ASCO happens, the clinical oncology conference in Europe they have something similar, EHA. Can you give us some insights? I’ll start with Dr. Krishnan. What were some of the highlights that came out of those large cancer conferences for myeloma patients?

Dr. Krishnan:
Sure. So I had the honor of giving the ASCO highlights actually at ASCO. It was 7 a.m. Sunday morning, and surprisingly we had a full house, which tells you the interest in myeloma. So the highlights in that session were really focused around relapsed myeloma, not surprisingly. So combinations of venetoclax, the drug approved for CLL, using it combination with carfilzomib, proteasome inhibitor, so we know venetoclax work the best when it is combined with proteasome inhibitor. Most of the data we’ve had so far has been with bortezomib, so this was the first trial presenting the data with carfilzomib, and that included patients who have had prior bortezomib or who were bortezomib refractory. So that was exciting.

Jack Aiello:
Just to clarify, if patients aren’t aware, Velcade is the same thing as bortezomib.

Kristen Carter:
Thank you.

Jack Aiello:
Yep.

Kristen Carter:
You know, the caveat in that trial was that patients had to be carfilzomib naive, so, you know, we clearly don’t know when patients have had prior carfilzomib exposure if they received the same degree of response, but the response rates were very high, and patients who had a particular translocation that venetoclax targets, the (11;14) translocation, the response rate was 100 percent. Again, these are small numbers of patients, but it is interesting data both in regards to the targeted therapy as well as in the idea that we can combine venetoclax with different agents.

The other thing I would highlight was the CAR T‑cell data, which I think of huge interest to patients. This is now an expansion cohort. So the initial data we saw was in about 20 patients. Now we have data‑‑it’s still not huge numbers, 40 patients, but what we did see was that the response rates remain very high, about an 80 percent response rate.

We learned some interesting things that previous trials and the CAR‑T in this construct, the Bluebird trial, targeted BCMA. And the initial phase of the trial required that the patient have a certain amount of BCMA expression on their plasma cells. And that was actually a hard target to get. Some patients were excluded. What we learned in the expansion phase is that the percent of BCMA expression on the myeloma cells really didn’t matter in terms of response. And that as an (?) Inaudible criteria is no longer an issue moving forward.

We learned that the cell dose of T‑cells infused matters in terms of response, that there is a certain minimal threshold of T‑cells needed. And we also did learn in terms of toxicity signals that we do see cytokine release. Fortunately in the majority of patients it’s been mild. I would think those are the two biggest highlights.

And the other one I wanted to briefly touch upon was the study looking at weekly carfilzomib. So it looked at weekly compared to a traditional carfilzomib schedule, and showed that a weekly higher dose was tolerated well. Interestingly, we actually saw a better progression‑free survival in the patients receiving weekly compared to the twice a week. I haven’t drilled down enough yet in that trial to know is that because of toxicity, or what are the reasons, but it just shows us that you can give weekly higher dose carfilzomib.

Jack Aiello:
And, Dr. Richter, do you want to follow‑up on any of those?

Dr. Richter:
So those were absolutely the big highlights. Everyone is very excited about the potential for CAR‑Ts and myeloma.

The other studies that I would high rights that came out of EHA and ASCO this year focused on combination therapies. It is still a goal if we can in patients to put them on multi‑drug combinations using multiple different mechanisms of action to treat the different types of subclones within the disease. So there has been data recently on three‑ and four‑drug combinations and how they may benefit patients.

So the combination of elotuzumab, pomalidomide and dexamethasone, the data was presented at EHA and was very encouraging as a really great option for patients with relapsed myeloma as well as that same combination, elotuzumab, pomalidomide and dexamethasone with bortezomib added to that. A four‑drug combination, but again in the right population this can be both tolerated and efficacious, as well as the three‑drug combination of Velcade, pomalidomide and dexamethasone.

And I know a lot of this may seem like, you know, they used to call it word salad where you’re just mixing up different letters and combination and it doesn’t all make sense, but that’s part of what our collective job here is to look at all the different options and all the data and drill that on what the exact correct regimen is for an individual patient. For some patients four or three drugs may be too many and two drugs may be appropriate, but in the right patients we may need to combine three or even four drugs to get the response needed.

Jack Aiello:
Can you say a little bit more about what makes the right patient for the right drug combination?

Dr. Richter:
So I think that’s‑‑there’s three different factors. There is treatment factors, disease factors and host factors that we take into account.

Treatment factors means have we given a previous line of therapy and did it cause toxicity. So if we’ve given drug A and the patient had horrible neuropathy I would not utilize that drug and may think twice about drugs that are similar. Host factors are things such as the patient’s age, their frailty, other co‑morbidities that they have that may affect the choice of drugs that we give. And disease factors are crucial. How quickly is the disease progressing? Is it taking other forms such as forming tumors such as plasma cytomas? Is it involving other areas of the body?

And as Dr. Krishnan pointed out, we’re starting to understand that certain drugs may have better efficacy in certain subgroups of patients. So for example venetoclax in patients with that (11;14) translocation or something called Bcl‑2 overexpression, we may utilize a drug like that in a patient earlier rather than later because that‑‑realistically, they’ll have a higher response rate.

Jack Aiello:
Thank you. We have a question from a caller named Mona who is a myeloma patient and did an allotransplant. Kristen, I’m going to ask you this question. She did an allotransplant in 2012. She’s been on Revlimid maintenance now for six years, and she’s a university instructor and leads a very active working life. Her question, though, has to do with does she take‑‑and this will be actually for all of you‑‑do I take‑‑in fact, let me ask this of Dr. Krishnan. Do I take Revlimid, continue to take Revlimid indefinitely, or is there a time when I can actually stop taking it?

Dr. Krishnan:
So the allo setting is a little bit different and because we really have no large trials. The only trial we have using‑‑two trials using Revlimid after allotransplant, one in the US, one in Europe, it was actually quite a challenge. A lot of patients developed graft‑versus‑host disease, so really only a minority of patients were able to tolerate it, and to say on it for as long as she has is actually quite impressive. So, honestly, in her case we don’t have any clear recommendation.

In the autologous setting we have differences right now. We do have‑‑

Jack Aiello:
I misspoke. Hers was an autologous transplant. I’m sorry.

Dr. Krishnan:
Okay. In the autologous setting we have the US approach which was based on the CALGB CTN trial, which randomized patients after transplants or observation or to lenalidomide indefinitely unless they developed toxicity or the myeloma progressed.

The French had a trial that actually started out with the same idea, indefinite lenalidomide. They ended up abrogating it because of their concerns for toxicity. The patients in that study had about 18 months of lenalidomide.

And then lastly there’s a big trial that’s going on right now that the IFM Dana‑Farber trial that in this French part patient after transplant might get lenalidomide only for a year. The US part patients get lenalidomide indefinitely, so it tells you that, you know, we can’t really‑‑don’t know and we can’t agree.

The last point I would say is a trial, which you’re very familiar with, Jack‑‑you’ve been hugely instrumental in getting it off the ground, is trying to answer that very question which is (?) Inaudible transplant get randomized to lenalidomide or lenalidomide and daratumumab, and then after two years if they’re MRD negative, so really looking very, very deeply at their myeloma, patients will have a second randomization, so a group of patients both stop therapy, so that will answer the question can you stop therapy if you’ve had a very, very good response.

Jack Aiello:
Kristen, I know you have lots of patients that come from really all over the world to the University of Arkansas there. There is a patient named Renee who is South African who says, I don’t have access to many of the newer myeloma medicines, and I wonder if there are assistance programs out there to remedy this. Are you familiar with being able to help someone like that?

Kristen Carter:
We have actually had several patients that this is a big issue with. I have a guy that is from Trinidad and he can’t get a lot of the medications there. And I have someone from the Bahamas saying they have a lifetime cap on their insurance, and then that becomes a big problem especially assess to medications in other countries. We actually have had people fly in to get medications and fly out, and we were actually able to get it through patient assistance here in the United States, but not everybody has the means to do that.

Jack Aiello:
Yeah.

Kristen Carter:
And so‑‑I mean, it is a big issue. I mean, even to try to get Revlimid in some areas or Velcade in some areas, it’s just not on their protocol in that country. And even here in the US dealing with the VA and certain places like that where different combinations have to be approved before they can get that. So that’s always a challenge, is access to medication and different regimens that may not be approved overseas, Canada, the Bahamas. European countries still are not utilizing the medications. So we’re very fortunate to live in the United States and have the access to the different combinations that we have here.

Jack Aiello:
Do any of you hear patients who have those problems trying to access generics, and do you have any feeling for whether that’s a good idea or not?

Dr. Richter:
I think it’s a difficult thing to ask because unfortunately there are well known disparities in terms of access to care within this country and in other countries, and a lot of the patient advocacy groups are trying to do what they can to help a lot of these patients. In terms of what patients ought to do if they can get access, I think it depends on the source. There’s obviously some legitimate channels that people can utilize to try to get access to drugs that may not be readily available.

Obviously, in the day and age we’re in I think there are some probably shadier ways people can get drugs, and it will be unclear how real they are. So I think that if you have access to any of these things it is probably best to bring them to a pharmacist to evaluate to ensure that if you are able to get these drugs from some other means other than the purely legitimate routes that you are taking the correct things and nothing that’s dangerous.

Jack Aiello:
There were a couple of maintenance questions that came in, and I’ll try to summarize them. David asked, rather than starting maintenance at 10 milligrams or 10 milligrams every other day of Revlimid, why not start at a lower dose, you know, two and a half or five milligrams or no treatment. And maybe you do that when you look at someone’s age and quality of life. And another person, Greg, just is flat‑out asking what is the best maintenance therapy to remain cancer‑free.

So, Dr. Krishnan, can you talk about how do you recommend maintenance treatment?

Dr. Krishnan:
Some of it is (?) imperious, but we do know that there is a dose response with Revlimid because we do see patients who, for example, were on maintenance at a lower dose and their M spike starts trending up, we increase the dose and we do see a gap but sometimes patients respond. So the dose that was picked was sort of a balance of trying to get a fairly active dose but understanding toxicity.

In newly diagnosed patients we use 25 milligrams, but in the maintenance setting we use 10 to 15 milligrams understanding there’s more hematologic toxicity after stem cell transplant so it would be hard for patients to stay on 25 milligrams for any length of time. So I think we’ve tried to balance that in our sort of initial recommendations for the starting doses of maintenance therapy.

In regards to the question what’s the best maintenance, I mean, that’s a great question and the answers still remain unknown. We just saw a press release from Takeda about ixazomib. We don’t have any details yet, but that it’s the oral proteasome inhibitor compared to placebo after transplant improved progression‑free survival. Again, don’t know anything yet about those patients within a certain subgroup, how big a benefit was it. So we’ll all waiting for the ASH meeting this year to hear that. But, again, it speaks to the question what is the best maintenance, and we’ll continue as we get new drugs study them both in relapsed, up front and in maintenance.

Jack Aiello:
Dr. Richter, as patients, though, get older and look at that quality‑of‑life issue, how do you adjust maintenance dosages, or do you decide maybe they shouldn’t go on maintenance?

Dr. Richter:
I go back to what Dr. Krishnan said which is true, which is the dose that was picked and was studied in CALGB study showed a progression‑free and overall survival, so that is our base from where to start from, but ultimately we then have to individualize from there. There are definitely patients that maintenance therapy absolutely benefits. There’s patients that unfortunately in order to provide a benefit from maintenance they have own toward toxicity, either hematologic with lowering of blood counts or other toxicities.

And on the flip side there are some patients that we feel may have higher risk disease where giving one or two drugs may not be the ideal maintenance, but there are some ongoing clinical studies looking at three drugs as a maintenance approach. And although this may seem quite extreme to some for those subset of myeloma patients with such high risk disease that we need to start enrolling in these trials to look at ways to offset their risk of having early recurrence. So I think we have what is the standard.

As Dr. Krishnan pointed out, there is the press release which we haven’t seen the hard data from yet with ixazomib, but this is going to be changing over time, and it needs to be individualized to the actual patient, their side effects, their type of disease along with the most up‑to‑date data.

Jack Aiello:
Kristen, a person named Donna from Nova Scotia asked, and I’m sure you are asked in a lot. And that is, I have severe neuropathy from Velcade. What treatments are there for severe neuropathy? And anyone can chime in, but I’m guessing you get this question.

Kristen Carter:
Yeah, that’s actually one of the most frequent questions I get. Especially when starting maintenance because we actually do do triple therapy maintenance utilizing Velcade. And the good thing is now that we have subcutaneous Velcade definitely the neuropathy is a lot less so we don’t have to worry about as much. I always tell my patients that we need to know about neuropathy before it gets grade 3. If you have grade 3 neuropathy I did not do my job.

We need to dose modify early. We need to start drugs like gabapentin or Lyrica. I’ve used Cymbalta. There’s several different ways to treat peripheral neuropathy, but the main big thing is dose modification and dose interruption if you have a grade 2 or more neuropathy. That’s when you start to need to think about dose modification. We do not want it to get to painful neuropathy and continue treatment.

And then you look at the clinical research on the newer drugs like Kyprolis or ixazomib that does have less‑‑less neuropathy associated with those drugs, so I’ve definitely used Kyprolis when someone had neuropathy with Velcade with not having further neuropathic symptoms with utilizing that drug. There’s lots of other options out there that does not have the associated neuropathy symptoms.

But the big takeaway would be let’s not let it get to grade 3 before we’re talking about neuropathy. So actually every visit, we talk about neuropathy at every visit. I ask that question at every visit, so preemptively educating the patient that these are the symptoms that you may develop, and also letting the patient know, hey, let me know if you’re having symptoms.

Jack Aiello:
Doctors Krishnan and Richter, any added insights in terms of how to fix bad neuropathy? And, by the way, if you do have any of that will definitely fix it, I will be in your office tomorrow.

Dr. Krishnan:
Absolutely.

Dr. Richter:
I think there’s a few‑‑the number one thing that Kristen brought up, and this is literally the biggest issue, is open dialogue with your care team. That is‑‑she is 100 percent correct. It is a lot easier to prevent than to treat. Unfortunately, the drugs that we utilize do not work in everyone. The other modalities that could be tried, I’ve had some success with Cymbalta, which she mentioned, also some of the tricyclic antidepressants drugs, like amitriptyline, nortriptyline may offer some help there.

But, again, this is really all about trying to prevent it and picking the right drugs and the right dosage. There are some newer‑‑we’re starting to work on some clinical trials here for some novel approaches, but nothing as a cure‑all just yet.

Jack Aiello:
Dr. Krishnan, anything else?

Dr. Krishnan:
No, I think we’ve covered every single drug that we’ve tried for neuropathy.

Jack Aiello:
I’ve had a few patients tell me that maybe acupuncture has helped them, cocoa butter has helped them, acupressure, acupuncture, as I said. But as you say there’s nothing for everyone, and it can be really debilitating if it gets too bad.

Dr. Richter:
There’s one other‑‑and again, neuropathy can come in a variety of ways. There’s a numbness but there’s also a pain. For people who have extreme pain there are compounding pharmacies that can a make certain combinations of lidocaine and some other medications that may help numb it. The other one‑‑and I know this sounds very extreme and not all places do this‑‑there are various studies looking at compounds of ketamine. And I know this sounds crazy, ketamine, which is also known as Special K, which is used in a variety of other nonclinical settings. There’s been some conflicting studies looking at the use of ketamine in peripheral sensory neuropathy, and I’ve had a few success stories in patients with severely refractory peripheral neuropathy working together with our pain management colleagues to compound the right dosage, but it can be tricky to use.

Jack Aiello:
Okay. Want to thank people who have already sent in questions, and for those you just joining questions can be sent in to myeloma@patientpower.info.

I thought that one of the questions came in from an individual named Jack‑‑that wasn’t me, but he asked a really good question. And he said essentially I don’t understand why newly diagnosed patients are often given the standard myeloma treatment regimen called RVD, Revlimid, Velcade and dex, from the beginning. Would it not be equally or better to maybe start treatment at lower dosages to see what the initial response is and then titrate up to the higher doses if needed? And perhaps if they were just as effective this would reduce side effects and toxicity.

I know, Dr. Richter, this question interested you as well, so how do you answer that?

Dr. Richter:
Again, it’s a patient‑by‑patient basis, and although RVD is an extremely common initial therapy if you look at the MM connect data about most utilized therapies in up front patients it includes RVD, Velcade, Cytoxan, dexamethasone but it also includes a fair amount of Velcade‑dexamethasone alone or Revlimid‑dexamethasone alone in up‑front therapy.

The rationale to give more drugs up front comes from our knowledge of the biology of myeloma and that we recognize that myeloma is difficult to kill in a human being, that plasma cells are very robust, and we do have evidence that the deeper responses that we can achieve, so getting patients to a partial remission, very good partial remission and down to the levels of complete remission with MRD or minimal residual disease negativity seemed to impact overall outcome, and patients who achieve those deeper remissions tend to do better.

So that’s the reason why we tend to start these multi‑drug combinations at fair doses is to attempt to achieve those deep levels of remission because those tend to be the patients that have better outcomes. Now, this is not wholly true. There are patients who can get two drugs and do extremely well, but as we have just the data that’s out there to go on, this leads us to choose this approach.

Jack Aiello:
Yeah. Do you agree, Dr. Krishnan, I presume?

Dr. Krishnan:
I do. I do want to make one comment that it’s not that every patient gets RVD, but, I mean, frankly, our interest is not taking away drug it’s in adding more drugs because‑‑and we get high response rates and we want to actually‑‑we think that the toxicity profile is manageable for the gain you get from deepening it responses.

Jack Aiello:
Dr. Krishnan, I thought Greg asked a million‑dollar question here. After achieving remission and completing maintenance what are your best resources for options to maintain the remission and avoid relapse?

Dr. Krishnan:
I think a lot of that depends on what treatment you had originally, as Dr. Richter said, the biology of your myeloma. Some people have a more aggressive cytogenetic profile, for example, so we tend to treat them more aggressively and continuously. I think just, again, that’s a very individualized to the patient, but think the one take‑home message I would say is that myeloma is different than a lot of other cancers in the sense that we really don’t stop treating, that we continue therapy, and this concept of maintenance is very sort of germane to myeloma.

And, frankly, other diseases are starting to adopt it more now. We see in the lymphoma space more in the concept of maintenance now. And you could argue in breast cancer with hormonal therapy patients are on a drug for extended periods of time. Those are eventually stopped, so we hope in myeloma we get to be stopped, too.

Jack Aiello:
Kristen, I have had people ask me since I’ve been diagnosed a long time ago, what do I do nutrition‑wise? And I don’t ever have any good answers for that, and I’m sure you’re asked that question. Do you have any good answers for nutrition to help benefit myeloma patients?

Kristen Carter:
I tend to be‑‑I like to look at the whole body. I’m definitely a person that adopts a very clean diet and exercise program myself personally, and so I think that nutrition makes a huge difference just in everyday life. Now, do we recommend an alkaline diet and a ketogenic‑based diet? Absolutely not. If you want to do that, we’re welcome to let you do whatever you feel comfortable, but I do tell patients that it’s very important to continue to eat good, nutritious‑good nutritious diet.

And also exercise. I think it makes a huge difference in fatigue and overall well‑being to get good exercise and have just a well-balanced diet. But we still do not adopt, you know, specialized diets, sugar‑buster diets for myeloma. I have actually had patients that we’ve gotten after they’ve done two years of alternative therapy, and if you want to complement your treatment with alternative therapy we say as long as it doesn’t interfere with the type of therapy that we’re prescribing, go for it.

But as far as doing alternative diets and therapy, we still have not adopted that or seen a huge benefit to the patients. So I just tell my patients to live your life, have a good nutrition and exercise program.

Jack Aiello:
Yeah, I agree. Well, I think Lonnie asked a question that’s been asked for 15 years at least, and that is whether to get a stem cell transplant or not. And specifically how does one make a sound decision about that? I’ll start with Dr. Richter.

Dr. Richter:
I appreciate starting with me, although for what it’s worth Dr. Krishnan is actually director of transplantation services. But I think this is a personal decision. As drugs have gotten better and better it is definitely come into question about the role of transplant. Many years ago when the only options we had were steroids, melphalan as pills and a combination called VAD I think it was very clear that autologous stem cell transplant was very much the way to go.

As novel therapies have come outed it continues to come in question. That being said, the data to date has shown that for those patients who are eligible to undergo autologous stem cell transplant there continues to be a benefit for patients who are able to undergo that. Now, what that means is fairly vague, and it differs from country to country. In the United States there’s no absolute age limit, but physiologic age comes into play as much if not more so than chronologic age, so I still think that it’s an important part of therapy.

This will‑‑you know, I think we always ask as we get new therapies is transplant going to go away, and what I always say there’s two reasons why I think transplant is going to be here to stay. Number one is patient selection. There are certain patients that we can give a stem cell transplant who will remain in remission for many years if not longer, so it still represents the best therapy to get those really long‑term remissions. And as we get better data behind us we’ll know, be able to select out who is the correct person to transplant who is really going to get that great benefit.

And one of the things that’s evolving in terms of new technologies is post‑transplant therapy or give‑back. So the question is in the next five to ten years are we going to start to see things like post‑autotransplant (?) carts or post‑transplant placental‑derived national killer cells or some other give‑back post transplant to augment their therapy so that once we get that deep remission with a transplant we can give another immune‑based therapy to push them even farther and achieve an extremely long remission if not potential cure. But I absolutely would love to hear what Dr. Krishnan has to say about the subject.

Jack Aiello:
I do too, but that’s why I asked you first. Dr. Krishnan, you are director of transplantation at City of Hope, so how do you answer this lady’s question?

Dr. Krishnan:
Well, first of all, thank you. You both gave me a promotion. I’m actually not director of transplantation. I’m director of the myeloma program. Still, I guess a couple points. Number one is if you look at the CIB in terms of the (?) international bone marrow transplant registry really only 20 to 30 percent of patients in the United States who are eligible for transplant are referred for transplant, so it’s very underutilized. As Dr. Richter said, we now have trials using modern drugs comparing them to transplant, and transplant still seems to offer us longer remissions.

And then the third point is transplant has become safer so we know in that comparative trials, again, obviously patients selection. You’re going to stack the odds in your favor if you’re going to offer a therapy that you want a good outcome, that the risks now are equivalent to the initial induction RVD therapies for patients getting an autologous transplant. And so much so I can tell you at least at our center we’ve moved our transplant to the outpatient setting.

So when we started patients were in isolation, boy in the bubble kind of thing, and now we recognize most of the infections patients get are from their own body not from everyone bringing it in to them. There’s a lot to be said for, as Kristen said, exercise, walking, diet and trying to maintain some normalcy, so having all those things when you’re not in the hospital are much easier. So all those things tied together hopefully have made transplant much or accessible, safer and sort of less frightening to patients, too.

Jack Aiello:
And, Kristen, you probably get patients there at Arkansas asking why are you suggesting two transplant instead of you just a single transplant? How do you answer?

Kristen Carter:
What’s funny is you ask three transplanters what we think about transplant because we’re all for transplant and we’ve done, you know almost 12,000 transplants for myeloma. And we do do tandem transplants, and they have seen, like Dr. Richter said, the deeper the remission, we know the longer the progression‑free survival, and there have been clinical trials that show that tandem does lead to, I think, it’s a 15‑month progression‑free survival advantage.

However, tandem transplant is not for everyone for sure. I mean, we definitely have people that are in their 70s, late 70s, are we going to do a tandem transplant on that person? Probably not. If you have a 40‑year‑old then that’s where you’re thinking of tandem transplant and more aggressive therapies. And I will tell you we’re seeing patients younger and younger. The 30‑year‑olds that I see, come into our clinic it’s just heartbreaking. We usually think of myeloma in patients that are in 60s and 70s, and, you know, if you give those patients a 10‑year survival maybe that’s a success. But if you have a 40‑year or even a 30‑year‑old, 10 years is not a success.

So we’re trying to do what we can up front to give these patients the best long‑term progression‑free survival available. Yes, as we sit here today transplant today‑‑it may change tomorrow with newer therapies‑‑has shown the best benefit for these long‑term progression‑free survivals.

Jack Aiello:
We have patients in our support group and I know across the nation saying, well, should I consider trying to get one of these CAR‑T therapies instead of doing a transplant? Dr. Krishnan, let me ask you the question. Do you think one day that maybe CAR‑T might replace transplants? Or how do you answer patients that have that question?

Dr. Krishnan:
I don’t‑‑well, number one, I think that’s 15 steps forward. If you look at the progression‑free survival just from the Bluebird trial it was‑‑it’s not four years, which is what‑‑or three years even what you’d see. Again those are relapsed patients.

Jack Aiello:
Heavily pretreated, yeah.

Dr. Krishnan:
Exactly. We do know too is you first have to reduce the amount of myeloma in the body for CAR‑T to work well and also to reduce the toxicity of CAR‑T. So you can’t just take someone with newly diagnosed myeloma and give them CAR‑T cells.

What we are looking at is the trial that was going to open through the (?) VMD CPN is patients that have very, very high risk myeloma doing CAR‑T cells after an autologous transplant. So really in a way you’re trying to get the best of both worlds.

Jack Aiello:
And, Dr. Richter, you would probably agree with that?

Dr. Richter:
Absolutely. At the moment although CAR‑T technology is extremely exciting it is not FDA‑approved and as it’s on clinical trials spots are unfortunately very limited, so at the moment the standard of care is still to move towards an autologous transplant. Having an autologous transplant does not make you ineligible for many of the CAR‑T protocols.

The only type of transplant that limits options for CAR‑T is allogeneic stem cell transplant, makes you ineligible for many but not all of the CAR‑T protocols. But, again, the decision of which way to go now is going to change in the future and this is a conversation you should definitely have with your care team.

Jack Aiello:
Thank you. We need to start wrapping up, although I have a number of other questions I could ask you. And I do want to ask one question that was asked, and that was from Heather who asked the question about how‑‑can you discuss or how are any of the new treatments being used to treat amyloidosis that’s caused by myeloma? Dr. Krishnan, can you respond to that?

Dr. Krishnan:
I think we need to make a distinction because amyloidosis, what we call secondary amyloid that’s associated with myeloma and then there’s primary amyloidosis which tends to be much more of a different clinical symptoms, and those patients, quite frankly, often are sicker than myeloma patients because amyloid can involve the heart, the GI tract, kidneys and a lot of neuropathy. And certainly the heart, when amyloid involves the heart especially that can make patients quite fragile.

And so the drugs we use, we do use the myeloma drugs because amyloid is still a disorder from plasma cells, but we tend to use them at different doses. And, again, we monitor for different toxicities in that population. Having said that, you know, we’re very excited about daratumumab now and activity in amyloidosis. Again that’s primary amyloidosis.

But, as I say, amyloid is such a different bird for us. We have a director of amyloid here. It’s really grown into its own special niche. They have their own society too. I mean, we co‑mix, but again it tells you how unique that disease is.

Jack Aiello:
And, Dr. Richter, how do you treat secondary amyloid?

Dr. Richter:
Unfortunately, so far the drugs that we have for myeloma that we use in amyloidosis, they control the core problem which is the production of the light chains that tends to lead to amyloid but doesn’t get rid of the amyloid fibrils themselves. So we have patients that enter a hematologic remission where we get rid of the bad protein, but they still have significant organ dysfunction, either cardiac or renal most commonly from amyloidosis.

There are several drugs in clinical trials that are looking at targeting the amyloid fibrils themselves, and although it’s still somewhat controversial there’s some interesting data about doxycycline, which is an antibiotic a lot of us have used in the clinic, that there may be a component that doxycycline may destabilize some of the amyloid fibrils. Again, the data is still evolving, and we don’t know yet, unfortunately, how to treat many of these patients. Amyloid is one of those diseases which is often diagnosed after patients have had it for a very long time, and we often have a lot of ground to make up at diagnosis.

Jack Aiello:
Can I ask each of you to make closing remarks? The objective of this webinar was to provide insights to myeloma patients in terms of how to best move forward in getting the right treatment and cancer care for their myeloma. Dr. Krishnan?

Dr. Krishnan:
I guess I would bring it full cycle to echo what Dr. Richter said at the beginning. Myeloma is still is rare disease compared to breast cancer, lung cancer, so most community doctors don’t see a lot of myeloma. And we know from actually published articles now that the more myeloma patients you see the better the outcomes are for centers.

So we don’t expect everyone to travel to LA or the Bay Area. So it could be 20 miles, but it could take two hours, so we don’t have that expectation you’re going to come in every week to see a myeloma center, but at least have that conversation early in the course of diagnosis and at various stages along the way, if the myeloma comes back, for example. Again, good to have it at points where you’re thinking of changing therapy.

Jack Aiello:
Getting second opinions from myeloma experts like yourself to at least be part of your medical team and work with your community doctor is awfully important in my opinion.

Kristen, can you offer your summary comments?

Kristen Carter:
I think both Dr. Krishnan and Dr. Richter are absolutely correct. Get to an academic center I think is important if you have the means. Also be your own patient advocate. I do think that the more well informed you are the better. And also the big thing that I see a lot is we will see these new trials come out, and, oh, this is so exciting, but we don’t have long‑term follow‑up for these new treatments, and we’re going to try the tried and true with the long‑term follow‑up success in the treatment available. So getting to an academic center, getting the treatment that they recommend and being your own patient advocate I think are the biggest takeaways to our patients.

Jack Aiello:
And, Dr. Richter, you have about 30 seconds.

Dr. Richter:
So, to me, the biggest thing is don’t be quiet. I see a lot of patients being stoic. You don’t get extra points for being in pain, not sleeping at night, getting neuropathy. Our entire care team, our whole purpose is to help in any way that we can, and if we don’t know some of the symptoms are going on we can’t treat them, so I would rather hear 10 symptoms that are not worrisome signs than not hear one that is.

So please encourage you to reach out to your nurses, PA s, doctors, the whole care team especially when you’re in the visits. It’s all about you. Please speak up if you have any problems at all because we have a lot of ways to deal with them.

Jack Aiello:
Thank you all for the insights you provided for myeloma patients. My name is Jack Aiello, and I appreciate all that you do.


Please remember the opinions expressed on Patient Empowerment Network are not necessarily the views of our sponsors, contributors, partners or Patient Empowerment Network. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Clinical Trial Mythbusters: Does the Clinical Trial Process Need an Extreme Makeover?

Does the Clinical Trial Process Need an Extreme Makeover?

Clinical Trial Mythbusters: Does the Clinical Trial Process Need an Extreme Makeover? from Patient Empowerment Network on Vimeo.

Downloadable Program Guide

Many cancer patients feel that the clinical trial process is in need of a serious makeover. One of them is Jim Omel. Jim, a retired oncologist living with multiple myeloma, turned patient advocate, makes it his business to understand myeloma from the inside out. He joins this program to share his experience in clinical trials and how he learned about his vulnerabilities as a cancer patient.

Also joining the discussion is, Dr. Michael Thompson, medical director for the Early Phase Cancer Research Program at the Aurora Research Institute and an active clinical researcher developing new treatments, particularly early phase (Phase I and II) molecular biomarker-driven clinical trials.

Join us for a meeting of the minds on debunking myths around clinical trials. How are patients protected within a trial? Will I as a patient be lost in the clinical trial system? Can I select my own arm in a trial? The questions are endless and, left unanswered, contribute to the barriers to trial enrollment.


Transcript:

Andrew Schorr:

Welcome to this Patient Empowerment Network program. I’m Andrew Schorr from Patient Power. I’m joining you from near San Diego, Carlsbad, California, and I’m so excited about this program, Does the Clinical Trial Process Need an Extreme Makeover? Having been in a clinical trial, and I’ll talk about my experience in a little while. I am a big fan, but I know that people have concerns, and I know that the percentage of cancer patients who are in clinical trials among adults is very low. How does that affect drug development and having the chance to get closer to cures for us?

I want to thank the financial sponsors for this program who provided assistance to the Patient Empowerment Network. They are Celgene Corporation, Astellas and Novartis. They have no editorial control, so what happens in the next hour is what we say, the questions you ask, what we hear from our experts who are joining us.

If you have a question, send it in to questions@patientpower.info. Again, if you have a question, send it in to questions@patientpower.info, and our wonderful producer Tamara will take a look at it, forward it to me, and as we can over the next hour we’ll be discussing questions you have already sent in. And we’ll have a very inspiring, I think, and provocative dialogue between our experts.

So let’s meet them. I want to take you to Grand Island, Nebraska, where my dear friend Jim Omel is there. He’s a retired now family practice physician. And, Jim, for years you’ve been a myeloma patient. When were you diagnosed with myeloma, and what’s happened along the way? You’re taking regular treatment now, I think, some treatment for the bone complications. How are you doing, and when were you diagnosed?

Jim Omel:

Andrew, I was diagnosed in 1997. It started off with a plasma cytoma at T10. I broke my back, I underwent a stem cell transplant in 2000 and had six years of remission. It came back in 2006, and I had radiation and lenalidomide (Revlimid), and it went away a while. Came back again in 2010, and I had radiation, bortezomib (Velcade), Revlimid, dex, and it went into remission. And since then, Andrew, I’ve been so fortunate that all I’ve been taking is bone-protective bisphosphonates.

Andrew Schorr:

Oh, good for you. Now, you were in a trial, but you decided not to continue, but yet you’re a believer in trials.

Jim Omel:

Oh, absolutely. Without trials our treatment wouldn’t change. When I had a full evaluation at Arkansas they suggested that I join their trial, and I did, and at the end of that trial was a tandem transplant. And I got to thinking and reading, and I didn’t really want to get that extent of treatment. I had a single transplant, and I dropped out of the trial. And that’s one of the things that I would certainly tell our listeners, that they can stop a trial at any time. They’re not bound to it. Ever since then, Andrew, I’ve had the good fortune of having fairly responsive myeloma, and when I had my treatments they responded to standard therapy. I certainly would have rejoined another trial if necessary, but I was fortunate that it responded the way it did.

Andrew Schorr:

Okay. And before we meet our next guest, I just wanted you to list some of the committees you’re on, because you’re very active locally and nationally on behalf of patients. So what are some of those activities you’re doing?

Jim Omel:

Well, I’ve been doing this since about 2000, so that involves a lot of activity. Peer review with the NCI was one of my main ways to get started.

Andrew Schorr:

National Cancer Institute.

Jim Omel:

Yes, and I progressed on to the Board of Scientific Advisors, which was a really good, important work with the director of the NCI. I’ve been an FDA patient representative for many years and was on the advisory board that brought Kyprolis or carfilzomib to us. I spend a lot of time each month for sure with the Alliance Cooperative Group working with Paul Richardson as we bring you new trials to patients. I’ve been with CINBR, Center for National Bone Marrow Transplant research for several years, several advisory boards. I’m on two pharma accompany advisory boards as they seek patient input.

Andrew Schorr:

Wow. All right. Well, the point of this, what I wanted our viewers to get, is that Jim is—trained as a physician, worked many years as a family physician, became a patient, eventually had to retire. He’s been through a lot of treatment and is very much an advocate for all of us, particularly in this process of trials. So we’re going to talk about the unvarnished truth about trials and see how we can make it better. Okay.

Let’s skip over to Milwaukee, Wisconsin, where we’re joined by Dr. Mike Thomson, who is very involved in research, and Mike has been very involved in all sorts of programs related to education. So, Mike, first of all, welcome to the program, and tell us a little bit about your involvement both locally in research and in education of other physicians nationwide and worldwide.

Dr. Thompson:

Sure. So not as impressive as Jim, but he’s one of my heroes who has really dedicated himself to improving the clinical trials process. I have an MD, PhD. My PhD is in pharmacology, and I was interested in pharmacogenetics and how individuals vary in their response to drugs, especially cancer drugs. I did my fellowship at MD Anderson and worked with a lot of myeloma doctors there and have worked in the community setting seven years in one place and about five years now where I’m located at and Aurora Healthcare in Milwaukee. I have been on the NCI Myeloma Steering Committee. I’m currently on the NCI lymphoma steering committee. I helped organize the ASCO 2016 meeting. I was the Chair of Education. As of June, I’m one of the editors for cancer.net around myeloma, so taking over from Paul Richardson who did that. So I’ll have about three years doing that and probably asking people like Jim for help to provide educational materials for people. And in the world of myeloma, I’ve created the MMSM or Multiple Myeloma Social Media hashtag to have Twitter chats, which I know some people don’t think are the optimal form of communication, but it is a way to get information out from experts and some opportunity for patients to ask questions. So I’ve been highly involved in social media, highly involved in the NCI and NCORP for increasing access to clinical trials in the community. And right now I am in the middle of an NCI designated clinical trial called EAA172 for multiple myeloma, which has gone through ECOG Executive Committee, the NCI Myeloma Steering Committee, and now we’re discussing with the companies and with Ctap how to bring that forward. And I think that’s—one of the things is how much effort it takes to bring some of these trials from concept to activation.

Andrew Schorr:

Okay. Now, we’ve mentioned this more rare cancer, multiple myeloma, not rare if you have it, but Jim has it, Mike specializes in it a lot. But what we’re talking about applies to the clinical trial process about broadly. So we may have people with us living with lung cancer and hoping to live longer and better, prostate cancer, chronic lymphocytic leukemia, like me, are also myelofibrosis. I’m a two-fer, if you will. There may be many different cancers among our audience, and the process applies to all. So we’re going to talk about that. So whatever it is, ask your questions, questions@patientpower.info. I’m just going to share a little personal story for a second, because I’m very passionate about it, and I wanted to mention it. And this is part of our Clinical Trials MythBusters series, and we have previous programs on Patient Power with lung cancer experts, experts in other conditions about the clinical trial process, so look that up on patientpower.info. There will be a replay of today’s program and also a downloadable guide with highlights that you can share, talk about it with your doctor, with other patients, with people you know and for your review. Okay.

So now my own story. I was diagnosed with chronic lymphocytic leukemia, the most common adult leukemia, in 1996—terrified, had no idea what it was. Didn’t know anything about what a trial was, didn’t know what the treatments were. Quite frankly, thought I’d be dead like within a week. I didn’t know. And so you start getting educated, and eventually that led to me connecting with academic medicine specialists and ultimately suggestion at the appropriate time of being in a Phase II clinical trial. I didn’t know what the phases were, we may talk about that along the way, and it was 2,000 miles from my house. So I traveled a number of times to be in that trial, and I had my local oncologist collaborating on that. And the end result was I had a 17-year remission. I had treatment again for chronic lymphocytic leukemia. It wasn’t until last year, 17 years. And I got the combination of medicines 10 years before that combination was approved. So I’m a believer.

The second thing I’d say about trials was I was in a second trial along the way, and I had deep vein thrombosis, blockages in the veins in my legs, for a blood thinner trial. And by being observed in that trial, that led to them discovering a second cancer which was at work related to those clots, myelofibrosis, and I was observed, so I liked the attention. It had nothing to do with what they were testing. It had to do with the observation you get. So, again, I love the attention of being in a trial. It may give you access to tomorrow’s medicine today, but there are things that may be broken. So, Jim, let’s start with that. Jim, what has been some of the frustration points for you the way the process has been today?

Jim Omel:

Well, I think one of the main things, Andrew, is that clinical trials tend to be designed to answer scientific questions. I think what they should do is be patient friendly. I think they should be designed to help patients. If you ask any researcher, what is the purpose of the scientific trial, clinical trial, they will say, to answer a question. If you ask a patient, they’ll think the purpose of the trial is to help patients. The—it may seem like a minor point, but it’s not. Patients need to be the center of them. We need to help patients understand what their contribution is to a trial. For instance, hardly ever does a patient hear how their outcome, what they did during a trial improved the final outcome of a trial. The patient needs to be centered. If we get the trial to a point where some of the questions are pretty obviously answered, rather than continuing to recruit patients just to be statistically valid, I think trials should close sooner. I think they should be more focused on getting patient care without necessarily the scientific question. I’m not a radical. I’m certainly a fan of trials. We wouldn’t be where we’re at without trials, but I think they should just become more patient-centered and patient-friendly.

Andrew Schorr:

Okay. Now, Mike, Dr. Thomson, so we know we can’t have new drugs approved by the FDA unless there are trials, Phase I, Phase II for sure, and often, typically, Phase III and sometimes even monitoring after a drug has been approved. I think you call those Phase IV trials. But from where you sit having been around this a long time what are some of your frustrations? What would you like to see be improved?

Dr. Thompson:

I agree a lot with Jim. I think another word to put on it is pragmatic trials. So I’ve been on a number of advisory committees, NCI investigator-initiated studies and pharma-directed studies. And when you have an advisory group with a bunch of academics they often think about the theories, and they think about what would be interesting to know. And increasingly both the NCI and others are getting not only patients but community physicians who will say I don’t really care about this question here. And we don’t think that it will fly and won’t accrue, and we know a lot of trials don’t complete accrual, so therefore patients are wasted, if you will, because we won’t have the information, we won’t be able to answer questions. So I agree. There are so many things get to involved it’s hard to break them all down, but part of the issue is answering a clinically meaningful question. I think the meaning should be patient-centered. Within those questions you can ask scientific questions that are imbedded in what are sometimes called secondary imports or co-relative studies. But I just last week was talking to some pharmaceutical leaders, and I said, you have to design a trial to answer a question people care about, and that’s patients and physicians. Because sometimes the trials are designed to get FDA approval, and they’re comparator arm if it’s a randomized study, is an arm that we don’t think is the current standard of care, and we have to do them in countries where they don’t have as many therapies and they don’t have as much access, so they’ll get them done. But then when they’re approved in the U.S. we don’t know what to do with the trial, because it’s not a question we’re asking. So that’s important. And I think if more studies are done not to get FDA approval but to go on pathways and to ask, what are the clinical branch points for decision-making, I think that’s when you’ll start getting good trials.

There are a number of other issues around the pragmatics. So there’s this NCI Match study, tons of people screened, very few people on the matched drugs, and they switched over to a strategy more like an ASCO TAPUR, where they waited for people that already had testing and then the people that had already kind of pre-screened couldn’t get evaluated for the study. And many, many more people went on study. The imaging and other things in the middle were not as rigorous as a usual clinical trial. It rolled quickly, and I think the point is you’re looking for big end points. Where you have to sort of go back to the classical, randomized, Phase III large study is when you’re trying to make incremental improvements, so, for instance, breast cancer where the cure rate or progression-free survival rate may be in the 90-something percentile rate, or even CML or other things where we’re doing so well you’d need a lot of patients and probably a standard design. But in many other areas you can do a variety of different techniques—Bayesian analysis, continuous reassessment models.

And one thing Jim mentioned was stopping for futility or if there’s an obvious benefit, and that is done but probably not as often as it should be. And the designs using what are called interim analyses or futility analysis with data safety monitoring boards or DSMBs, probably could be more robust. There could be more of them. I think people are afraid to do them, because they do slow the trial down, they slow accrual, and that has to do with stuff both within the trial as well as extrinsic to it. So there are a number of barriers and issues, but I think Jim’s pinpointed them as well.

Andrew Schorr:

Okay. Well, folks, you can tell that Dr. Thomson is a scientist. We’re going to unpack this and get down to the nitty-gritty. So, okay. So, Jim, so first of all, we mentioned this term “randomization.” So people wonder in cancer am I going to get the good stuff? I know that I’m sick, maybe like in your area, multiple myeloma, there have been lots of new medicines, but in some other areas not, like pancreatic cancer, for example.

So, say, I understand the standard therapy, and you’re testing it maybe against that, but I want to get the good stuff, because I’m really hopeful. I want to be a believer. So could you just describe where we are with randomization, because that’s a concern people have?

Dr. Thompson:

Absolutely, Andrew, and thanks for asking that question. That’s a real red, red hot button item for me. I maintain that if the patient has gone through the effort of studying their cancer, studying the possible treatments, and they’ve learned of a trial that’s opened that they would qualify for, they’re excited, they go talk to the principal investigator, and they say I want to be in this trial. And the PI turns to them and they say, well, we’ll flip a coin. You may get the medicine we’re going to be using, or you may get standard therapy. Just imagine how disappointing that would be. And when it comes to randomization, Andrew, there’s many, there are many trials that absolutely lack equipoise. And I’m afraid that scientists often use equipoise.

Andrew Schorr:

Now, tell us what that means. You’ve got to define that for us.

Jim Omel:

Equipoise basically means equal, equal balance within the arms. In other words, technically, officially the principal investigator doesn’t know which arm is best. And yet look at it from the patient’s standpoint. Let me give you an example. There was a trial in which patients had the choice of three oral drugs in one arm versus a stem cell transplant in another arm. Now, think about that. Think of the insurance ramifications. Think of the fact that it takes almost a year to really totally recover from a stem cell transplant, versus taking three oral drugs. How can anyone say that there’s equipoise in a trial like that? So how can you pattern your life with the flip of the coin or a computer randomizing you into one of those arms?

Andrew Schorr:

Wow. That’s, that’s an important issue. Another one is, Mike, you know, people are—one of the ladies wrote in on Facebook I posted about this program, and she said, well, the trials are not really accessible to me because I live in a rural area, and they’re only in the big cities. You’re in one, Milwaukee. But Jim’s in Grand Island, Nebraska, and some people if you set requirements for the trial, well, you’ve got to come see me, you’ve got to come to the clinic for a variety of tests with some frequency and somebody has to drive four or five hours and take off work and get babysitters and all that, it just makes it impractical. Where are we with more trials being available or having an aspect of it, like testing, closer to home?

Dr. Thompson:

So I work at a community setting. I’m at our kind of flagship hospital but we cover most of the population centers of Wisconsin, so I think we cover about 70 or 80 percent of the population. So that’s a huge issue for our site is that we—when I talk to sponsors including as recently as last week I say if we can’t do it at all our sites I’m not really interested in doing your trial.

There are exceptions of course. We’re doing a surgical trial or a radiation trial that has to be at one site or sometimes a Phase I trial with just a lot of blood monitoring, very intensive, they can only be done at a few sites. But in general I completely agree that we should try to have the drugs available to people in the community they live in, because that’s where their social networks are, right? So that’s where their family is. They can stay at home. They don’t have to just go into a hotel. They don’t have to pay for travel, and I think it’s better for everyone. And for companies, I’ve been trying to tell them that it’s more generalizable to the reality of where cancer patients are. So

85 percent of cancer patients are in the community setting and are treated there, and drugs should be accessible to them there. So, you know, both the using the CCOP mechanism or NCCCP, and now we have the NCI Community and College Research Program or NCORP. The whole idea is to increase that access to community sites. So this has been going on a long time. I think there were budget cuts, and so the U.S. and the way we’ve established our cancer budgets has been to decrease access at least NCI trials and usually need some of those NCI trials to support the research infrastructure to do other studies. So I think part of that, you know, a lot of these things you follow the money. And if there was more money for community research sites, you could hire more research staff to get these things done.

But I think we need to get them done in the community, because we know if you do early phase studies and they look promising in highly selective patients, then when you expand them and put them in the community you go from efficacy to effectiveness, and the effectiveness isn’t there because the patients are different. So there are all these things with real-world data and comparative effectiveness research at ASCO’s cancer link trying to get at some of that not on study to just try to get the data.

But we need to have access to people, and the way to make drugs cheaper, make them develop faster and answer more questions, both scientific and patient-oriented, is to get more people on trial. There’s a big example for immunotherapy drugs where there are so many immunotherapy drugs and trials there are not enough patients to get it done. So we’re going to enrolling in trials which don’t complete, or we’re not going to be able to answer these questions, so it’s going to stall and move it out the process of moving faster. In myeloma, we move very fast, but we need to do this in other areas too.

Andrew Schorr:

Right. So let’s talk about that. So, Jim, you know, the president had a big kick-off, HHS Secretary Azar I think just yesterday as we do this program, was before Congress and part of it was the discussion of can we lower the cost of drugs ultimately? And one aspect of it is can we speed drug development. So instead of all these trials languishing at the cost of millions of dollars, hundreds of millions of dollars, how do we speed it up?

So one is participation, certainly, but can the process be simplified as well, Jim? What work is going on there, so we can try to get these answers and get to the FDA and present the data quicker, and hopefully there’s been lower cost in getting to that point?

Jim Omel:

Well, as we’re learning more and more about each individual patient, personalized medicine and targeted therapy, we certainly should start relying more on biomarkers. Biomarkers can be a way to select patients that would particularly fit a given treatment.

We need to lower costs. We need to make trials slicker and faster. Single-arm trials are those in which a patient just get—all the patients get the therapy. They all get the same treatment. And FDA has actually approved drugs based on single-arm trials, a much faster and efficient way to get an answer.

The problem is that the costs are going to be there. When I think about Mike and all the work that he does in developing his venetoclax (Venclexta) trial that he mentioned, Mike has put in months or years, and it’s all above and beyond his normal time. I mean his day job is to take care of patients, so all of the work that he does to develop a trial is just remarkable in the extra hours it takes and the consistency that Mike gives to doing his work. We need to make the trials more efficient.

We need to use biomarkers. We need to make them shorter. We need biostatisticians to come up with ways to give us an answer without having to approve so many hundreds or thousands of patients to all these potential new treatments.

Andrew Schorr:

So, Mike, let’s talk about that. And, Mike, first of all, I want to thank you for your—well, both of you, but, Mike, certainly in the clinic, thanks for your devotion to this.

But continuing on that, so this was brought up by Jim, biomarkers, and I know in some of the blood cancers now we’re talking about more and more minimal residual disease testing, and we’re doing genomic testing to see what genes have gone awry, what’s our version of lung cancer or a breast cancer or a myelofibrosis or whatever it is.

And then do we qualify for a trial? What’s our specific situation? Do you feel that that sort of precision medicine testing and analysis can help refine this, so we know which trial is right for which person at which time and also some analysis along the way of how is it going?

Dr. Thompson:

Yeah, so at my site I’m the director for precision medicine, and I gave a talk at ASCO on precision medicine and barriers in the community setting, so I’m very passionate about that. And I think that is one of the ways you can try to get things done with smaller numbers of patients and things done faster. And part of this is alignment, right? So there’s different perspectives, a patient perspective, a payer perspective, a pharma sponsor perspective, the physician. There’s all these different perspectives, and I think it’s trying to get them all aligned and trying to get things done faster.

So, you know, there are some areas where we don’t know enough, and we can’t use biomarkers. But there are other areas where we have a biomarker, and there’s feasibility, and we can test that quickly. And if we are looking for a large effect size—here I am in jargon mode—but if you’re looking for a big, big hit, a home run, is to look for an alteration that is very specific and we think is—a drug can target. So-called targeted therapy—it’s a little bit of a misnomer.

So—and lung cancer has been one of the hottest places for this. So there’s ALK inhibitors, ROS1 inhibitors, EGFR inhibitors, and now BRAF inhibitors, HER2 targets. So lung cancer has exploded with precision medicine therapy, and the same with melanoma and BRAF. So, you know, I think even skeptics will say you don’t really need statistics if the prior therapies, nothing worked, and you give something, and 80 percent of people respond.

There are issues with precision medicines, but the main thing is not response rate but durability. And I think that’s going to be the next iteration of the NCI Match study, which is a large precision medicine study, is stop doing just these small groups of people who are showing activity, but then they relapse quickly. And I think it’s going to look at systems analysis, and how do we overcome resistance.

But one way to get at this and another different take on it is inclusion and exclusion criteria. So this has to do with access and individualizing and being patient-centric. Many of the inclusion and exclusion criteria, when somebody says, oh, I have lung cancer, oh, here’s a lung cancer trial, and they say, oh, you can’t go on the trial. And much of that is because there’s language that’s been cut and pasted from a previous trial which is not really pertinent.

So if the new drug is metabolized by the kidney, you don’t necessarily need to look at the liver studies. And we did a small study or I was aware of a small study done by Kaiser where if we improve the inclusion-exclusion criteria, accrual rate can go up 30 percent—so no cost to that.

Andrew Schorr:

Wow.

Dr. Thompson:

And Ed Kim led a publication about six journal articles in JCO about different aspects of inclusion-exclusion criteria including function, HIV status, age, etc.

Andrew Schorr:

Well, yeah. We had Ed Kim on the program just a week ago, as a matter of fact.

So, Jim, inclusion, exclusion, so first of all, we’re in this age where electronic medical records, it would seem that at your fingertips there could be some analysis of your record and some matching or offering of trials that could come out of an analysis of your results, genomic results. Do you have ALK or ROS or whatever, if it’s lung cancer, whatever it may be maybe JAK2 positive in myelofibrosis, what is various status for us?

And also broader inclusion criteria, and Mike was getting at that, saying some was just—excluding was just cut and pasted. And a lot of us patients would feel, well, that’s just unfair. So what’s your comment on all that, about inclusion and exclusion and analysis so we can be matched with trials more easily, can be offered to us?

Jim Omel:

Inclusion and exclusion criteria are really important parts of trials. They’re what get people into trials, they’re what keep people from being in trials. And, unfortunately, Andrew, many times the criteria are very defined, very narrowed, and drug companies especially want to do it this way to get the best effective appearance of their drug. They want to get approval. And yet in the real world, in fact most times, patients who would not even need inclusion criteria are the very patients that are going to be taking these drugs.

And Mike’s right. There’s too much cut and paste. If a trial takes a thousand patients to write a proposal or protocol, too many times researchers will just take the exclusion criteria that might have been from a previous trial and, like Mike said, cut and paste it when perhaps it’s not even necessary to have creatinine values or kidney values measured so precisely on this particular drug compared to the other one.

So those are the criteria that let people in or keep people out of trials, and they absolutely need to be widened. To make a drug more applicable to the general population we need to reflect the general population more in trials.

Andrew Schorr:

Right. Right. It’s sort of a Catch-22. So if somebody is at a drug company and they’re investing hundreds of millions of dollars maybe to develop a drug, and then that trial is languishing or taking longer to get there, somebody ought to go back and say, well, can I loosen up this criteria, get the big answer and do benefit to patients who may be very willing to be into a trial that doesn’t have all of these requirements that are not really necessary? And we get the answer and get it quicker, and help people along the way. I mean, it’s pretty obvious to me, and I hope they’re watching, folks.

So, Mike, here’s a question for you, though, and you work with people in the community setting. So we have patients who have written in and said, you know what, where I go to the cancer clinic they never mentioned trials to me, and Jim alluded to the extra time it may take for physicians and their teams is when there are trials. You have just treating people with current therapies, and then you’ve got research layered on top of that. It’s very time consuming.

But what about just awareness at the community level? What can we do about that so that wherever I go into a clinic they have a clear picture of what I’m dealing with, and if there is important research going on that relates to me I hear about it? Now, maybe they say, you’ve got to go to a university center, you’ve got to go to Milwaukee, wherever you have to go, but there’s that discussion.

Dr. Thompson:

Yeah, so with all of these, you know this has been analyzed in multiple different papers. We were on one looking at a trial log, trying to look at some of these issues, and what seems to be clear is when people are offered trials they tend to go on them at about the same rate, and that has to do—seems to be somewhat independent of socioeconomic status, race, etc., or geographic area.

So one of my colleagues, Dr. Verani, told us about—about this, about rural settings how do you get people on trial. So there are different barriers. So one is the trial, and like Jim said, if you can only do some therapy that you have to come in quite a bit for that limits the geographic area you can accrue to for most people.

There are site issues where if you don’t have enough research staff to be there enough the doctor doesn’t feel supported to spend time on it. There are physician issues where they may not care about trials, or they have too much people scheduled in clinic, they’re an hour behind, and they can’t stop to spend time on it.

Also in the community setting you may be seeing every type of cancer, and you can’t remember everything, versus at many academic settings you may only see one or a cluster of types of cancer. So if you’re seeing lung cancer all day and you have 10 trials open, you probably know those trials very well for lung cancer, because you don’t care about the CLL or myeloma trials, you only care about lung.

And then there are patient factors. So patients that are in rural Wisconsin may have different characteristics, and the reason they’re in rural areas, you know, the motivations is about, you know, going in for things and stuff like that may be different than people who have the capabilities to fly to Boston or Houston or New York, and they can do that. So all of those areas are important.

Now, one potential way to help mitigate some of those things is we have got a clinical decisions support tool, which is an IT product, which our physicians have to enter in what they’re going to do with the patient. So it could be observe, no treatment, hospice or various therapies. And when they put in the cancer and the stage it pops up with the clinical trials, the first thing that pops up. And so the physician doesn’t have to do the trial, but they have to say why they’re not doing it. And so we can track over time. It doesn’t necessarily help that individual patient, but that doctor has been aware of the trial, and we kind of get an idea of why people are not going on studies, and so that’s one way to do it.

Something we just did the last week is we had a different IT product where the NCI-matched precision study opened up five new arms with different targets for different drugs. So we looked back at the number of patients that had those targets identified within our entire system, and then we screened those to see how many people were still alive, and were their organ functions still good enough to go on these trials because of the inclusion-exclusion criteria, and we found several. So we’re now able to contact the physicians and the research staff to go back for these patients that had screened for molecular testing and now they have new options.

So I think there are IT issues that you can do systemically to try to take some of those barriers away, and then each of those points does have barriers which probably have different solutions and different ways of tackling. But one reason, you know, the accrual rate hasn’t gone up a lot is it’s not easy. It’s a complex problem, so there’s not going to be one single thing you do. There’s going to be many different ways to try to improve things, including patient education.

Andrew Schorr:

Yes, well, okay. To let’s flip that over. Jim, you and I are patients. So what do we want to say, and from your perspective?

So back at the clinic and from group has, so Mike is working on IT to identify trials and have it pop up on the screen for the doctor. Okay. Great. But we’re the ones living with the condition. What can we do so that promising research that we may learn about is available to us? We can see whether it matches up with us. Maybe we have to go down the road. Maybe we have to have a discussion with our doctor to even encourage them to have you us be in a trial. How do we make it happen, okay?

Jim Omel:

Well, of course, we all need to educate ourselves about our cancer. When I was in medicine school I had heard about myeloma, but I certainly wasn’t any expert in it. I had two patients in my practice that had myeloma. I knew sort of how to take care of them. But since I developed my myeloma, I have become my own expert. And as I lead my support group, Andrew, I make them experts. I teach this cancer to them so that they can make educated decisions.

Patients are very likely to go on the Internet, watching Patient Power. In my particular cancer, they’re going to go to the IMF and MMRF to look at myeloma trials and see what’s available. And they will take that information to their doctors, many times making their doctor aware of trials that perhaps they aren’t each advocating or aware of.

So, Mike’s right. There are many factors that keep patients from trials, but one of the things that patients really do themselves is educate themselves and perhaps even to the extent of bringing or educating their doctor about what can be available for their treatment.

Andrew Schorr:

Mike, I want to ask you about cost. So you mentioned different inclusion, exclusion, or what’s your liver function or this or that. So there is a problem where maybe certain drugs or certain aspects of a trial are covered, but then your insurance company, you know, that you have or Medicare or whatever, they say, oh, no, we don’t pay for that, but yet it’s part of the trial or it goes along.

So people have a concern about cost. I want to ask you about two aspects of cost related to testing sometimes. And then also are there programs that can assist with the logistical costs for patients as well?

Dr. Thompson:

So when I trained at Mayo Clinic and MD Anderson, and when I got—first went into practice I prided myself in not caring about cost. And then I realized you have to think about these things because you can bank—you know, we bankrupt, about 40 percent of people with cancer get bankrupted. So these are huge issues for people who want to keep their houses, that want to hand something down to their kids, and cost is huge, right? So that can either be throughout the whole course of standard treatment, or it can be trying to meet the cost of going places, trying to find clinical trials.

So the Affordable Care Act and various other national and state legislative initiatives have tried to make insurance companies pay for the standard costs in clinical trials. There are some carve-outs for smaller companies and things like that, and so this is, you know, not perfect, but in general insurance companies should pay for the standard cost of clinical trials. They should pay for standard imaging stuff too, and they try to get out of that. So it’s not a perfect world, but that should be covered. And any research-associated costs should be covered by the company. Even in some NCI trials some people disagree with what should be covered and isn’t, and it’s complicated. But in general, a patient, the research cost should be covered.

Now, that does not include travel, lodging and a lot of incidentals. So there are a variety of foundations, that could be The Leukemia & Lymphoma Society, that could be other organizations which could help with that. Individual hospitals or health systems might have ways of approaching that. And sometimes there are things you can do within the various companies. So there’s a new target called Entrek, and the company Loxo, I’ve heard will fly people who wherever there’s a site and pay for them to go on the study, which I think is amazing. That’s not true for every company and every drug being developed. But that’s one way to do it.

One of the issues that comes up with IRBs if you’re giving people money, are you coercing them? And, you know, if you’re just recovering the cost to travel, I don’t think you are, right? But those are one of the things that come up. But certainly there are lots of disparities. And just like in different countries, they don’t have access to the drugs we have as standard drugs here, and not all of these disparities are going to be fixed because we have—outside of cancer we have lots of disparities in the United States, but cost is a big issue.

And then value, which we’ve been increasingly talking about in the oncology community, which is utility over cost. And that’s more for once we’ve done the trial figuring out even if shows like it works, how do we figure out how to use it based on those characteristics?

Andrew Schorr:

Thanks. And also I wanted to mention that Mike Snyder is sending that question, answering why it cost so much. I hope that answers it.

We have—you know, some people wrote in as we were preparing for this program and they were bitter because they thought they had a spouse, let’s say, that had died in a clinical trial. And that relates to a couple of things. One is transparency. Is the data from a trial and any dangers that show up, is that reported and analyzed in public, Jim? And also what are the risks being in a trial, and what is the monitoring to try to have trials be at safe as possible. So, Jim, maybe you could talk about that from a patient perspective.

I want to make sure I know what I’m getting, I know what the risks are, and if any have come up along the way I want it to be reported, and I want to know that there’s a team looking out for me.

Jim Omel:

You have every right to expect that, Andrew. If you’re in a trial you have the right to get that knowledge if there’s new things that come up that we’ve learned about. And part of every trial as it’s being written, there has to be a data safety monitoring board. These are the experts who will do what you’ve asked be done. They will monitor the trial as it goes along. They will look for any safety issues. If there are patients who are developing liver toxicities, they will find this. They will point this out and perhaps see if the trial needs to continue or if something needs to be revised.

The presence of institutional review boards review whether trials should go forward or not. Patients who are in trials actually get very, very good medical care and medical coverage. In fact, I would maintain, Andrew, that they get better care than just standard care. They have experts that are watching them even more carefully than would be in a general routine care setting because they’re looking for these concerns and problems.

The person who mentioned the bad outcome, we can’t ever say that every trial is going to be perfect. There are going to be concerns. That’s why trials are done. But they’re relatively rare, and we do have boards and review organizations during the trial, not afterwards, but during the trial to be looking out for your benefit, Andrew, so that you’re not hurt by the trial.

Andrew Schorr:

All right. But let’s say this—and, Mike, for you. So, first of all, admittedly a lot of these trial start, and people are sick people, and they’re feeling maybe the trial is their last hope. We had a friend, Lisa Minkove, who died in the CAR-T trial for CLL not long ago. She had been very sick with CLL, so we’d hoped that it would work. It didn’t work for her, whether CLL won. And we know other people whereas the learning is going on about often powerful new medicines they didn’t benefit. Or in one case, there was a drug, venetoclax we know about, there were some deaths early on when the drug was far more powerful than was originally understood. So what do we do? I mean that’s the real world I guess of scientific study, but that’s a concern, you know, Mike, of people saying, oh, my God, I’m worried about being a guinea pig the unknowns on the subject of dangers.

Dr. Thompson:

So there are a couple of things. So whenever people say—it doesn’t come up as much recently about being a guinea pig, I say, well, guinea pigs don’t have choices, so. And so like Jim has said you can drop off a trial if you want to drop off it. But—so I think for adverse events and things that can happen, one reason to randomize people is that you do understand then if you treat someone with one thing and then another and the death rate the same in both, the drug is not causing it. That’s just the disease. And a couple years ago, there was a presentation from the group at Dana-Farber on the precision medicine program, and the issue was they were taking so long to get people evaluated that their performance status or how well they felt was good, and by the time they got through the evaluation many of them had died. Because the disease, you know, when you get to fifth, sixth, seventh-line therapy it can often progress very rapidly.

And so I think that’s one of the issues, that people can feel the drug did it, and it’s hard to know. And we get these—doctors get these things called adverse events reporting forms, and we have to try to come up with is this probably related, possibly related, and we also get these forms that say you have a patient on the study. The study is open in three countries, thousands of people on it. One person died of a heart attack, and you have no idea as the physician, well, is that the same rate as—you know they’re 70 years old. Is that the same rate as this other 70-year-old. So you need the enumerator and the denominator, and that’s what the DSMB or the Data Safety Monitoring Board is supposed to do, which is look at the data and say, is this beyond what we would expect? And they can stop the trial. They can do expanded cohorts. They can do things to try and figure that out. Now, we know from like even car companies lying about their exhaust systems that if the Data Safety Monitoring Board gets false data, well, you can’t fix that. But that’s pretty nefarious. Like that I think is not something that’s commonly happening and would be a very serious thing to happen.

Now, one thing for transparency is that almost all studies I’m aware of get registered on clinicaltrials.gov or maybe some other sites but usually that site, and they’re supposed to report out the outcomes. It’s not also a perfect process, but you should be able to see how long the study has been open, are there any complications related to it and those types of things. So this whole process is not perfect, but I would say in general the people at the companies are trying to develop something they think is going to work. They’re trying to do it safely, both to help develop their drug well as well as to avoid a bunch of regulatory issues, and the people on the Data Safety Monitoring Board are trying to do their best to answer these questions. But the smaller the number of patients which increasingly will take the trials we are doing and almost are aiming for, it’s harder to be definitive about when these things happen and what caused it.

Andrew Schorr:

Right. Right. It’s imperfect, as we said. So, Jim, Mike Thompson mentioned earlier, gave lung cancer as an example and, of course, across immunotherapy, there are so many companies endeavoring to move this research along. So let’s say you had lung cancer or one of these others where this is big, although it’s going on in the hematology area too, so a patient says, oh, my god, there are all these trials, and I might qualify for one, two, three, four. How do I prioritize? What do I bet on? And maybe my own doctor is doing more than one. So what do you say to patients if they become receptive to being in a trial and there’s more than one trial that they qualify for?

Jim Omel:

That’s a very good question, and it’s a nice kind of problem to have, to have choices of trials. I think, Andrew, the best answer is the patient needs to look at what they are looking for. Are they looking for longevity? Are they looking for something that’s going to expend their life? Are they looking for a trial that maybe will greatly improve their quality of life? Perhaps they’re looking for a trial that gives them one pill per week versus two injections a week. So there are certainly effectiveness end points. There are different things that patients find of value.

But to answer your question it really comes down to each patient needs to ask themselves, what is it I’m looking for in a trial? Do I want something that makes my burden lighter? Do I want something that’s going to extend my life? How much am I willing it accept as far as potential problems versus the standard of care that I know what the problems exist with if I don’t go on a trial?

Andrew Schorr:

Right. So that’s a question we got in, is they’re trying to assess that. One was about how do I prioritize? The other is, by being in a trial, Mike, is it going to make me sicker? Like, to do I have to go through the valley of the shadow of death to get, hopefully, to a better place, and how do you discuss that with your doctor when not everything is known?

Dr. Thompson:

Yeah, maybe I’ll kind of step back and say for phases of trials, Phase I, the intent—both ASCO and NCI say the intent of a Phase I trial is therapeutic. But the statistical design is to evaluate safety. A Phase II is to look at initial efficacy or how well it works, and Phase III is to compare versus standard of care the efficacy. So there’s other types of designs, phase 0, Phase IV and other things, but it used to be, I think, you know, I—we would say don’t go on a Phase I unless that’s the last option because you’ve already gone through the safety initial efficacy if it’s a Phase III trial. It costs a lot of money to do Phase III trials so fewer are being done now, and we’re kind of finding that in this era of precision medicine people are going on trials, and there’s no one rule, but I look at it as if it’s a study involving a lot of different groups of patients, a lot of—you know, it’s not individualized to you, I don’t know, but I think it will have less of a benefit probably than if it’s something like a study designed for BRAF melanoma back when that was a study and you have BRAF. Well, it’s targeted for you. It doesn’t mean it will work, but even if it’s an early phase, a Phase I or II trial, it’s really aimed at your disease.

And we’re finding this with venetoclax, with T1114, and there’s other markers, FLT3 in AML, all these things, and sometimes we find that the drug doesn’t work like we think it’s going to work. The ALK and ROS story in lung cancer, it may benefit other people that we didn’t recognize before, and that’s part of–we’re trying to find people besides T1114 that respond to venetoclax in myeloma because it looks like some people will. But I think as we’re getting more targeted therapy it doesn’t mean there’s no toxicity, but it at least has the suggestion that we’re targeted more at your specific cancer. And some of these pills can have as much toxicity as IV chemo s, but our aim is to decrease toxicity and increase efficacy. And I think, like Jim said, you’ve got to look at different trials and hopefully with a physician who has time to sit down and run through several scenarios. And some people will take the most aggressive therapy because that’s what they’re after, and some people will try something that’s easier and closer to home. So everyone’s values are a little bit different, and you have to try to individualize as a patients.

Andrew Schorr:

Right.

Dr. Thompson:

One thing about trial matching is besides clinicaltrials.gov, there’s myeloma and other groups that are doing these matching, so you can put in characteristics of your cancer and you can try to filter out and get a closer approximation, including at clinicaltrials.gov you can click on the states in the surrounding area or how many miles you’re willing to travel.

Andrew Schorr:

Right. I would mention, put in a plug for our advocacy group friends, whether it’s Lung Cancer Alliance, Bonnie Addaria Lung Cancer or the International Myeloma Foundation with The Leukemia & Lymphoma Society, you can be in contact with them directly and talk about your situation, and they will often be very aware of trials and how it’s starting to line up with these sub groups, subtypes of illness. Here’s a question we got it in with Jack. I just want to get in a couple more before we have to go. This relates to what you were talking about the National Cancer Institute’s Match trial, as I understand it, Mike. He said, regarding precision medicine I thought I heard that initial results have been disappointing for the NCI trial which treats patients with a specific mutation with a specific drug for that mutation. How does this impact precision medicine? You want to talk on that? Mike?

Dr. Thompson:

Yeah, so the people who are opponents of precision medicine would say that the SHIVA trial in Europe and the NCI Match trials were failures. I think you need to look at it a little more carefully. And if you do a huge screening and you don’t have many drugs you don’t have many matches and not many people are going to benefit. So there are some arms in match that match the accrued the number they wanted, and the drugs didn’t work well. So those were truly we think negative studies. But I think the things about Match are there is a huge interest in the community, and they had thousands or several hundred people screened when they only had a few arms opened, and those people weren’t matches, and it basically overwhelmed the system. And then they had to rejigger it to open up more arms. So I think we could—you know, pick holes in the design of the initial study, but I think it took everyone by surprise how much interest there was in trying to personalize these molecular therapies. And other iterations such as ASCO TAPUR, there’s company versions of it like Novartis Signature, and I think the new design of Match do allow for better match rates, and we’ll see how after they’ve adjusted how well they can hit their targets.

Andrew Schorr:

Okay. So that’s an example, where we’re going through a makeover there. Before we go, Jim, we have people watching from all over the world, and Mike alluded to sometimes trials done in other countries. Certainly they are. So we have somebody from New Zealand, we have people from other countries now. How do I access trials? Does it have to be in my country? Or what would you say to an international audience as far as finding out what’s available to them?

Jim Omel:

That’s a difficult question because every country has their own standards. Each country has their own boards that review. What is allowed in some countries are not even allowed. Observational trials can have more importance in some countries than others. Again, it’s a tough question. I think perhaps the person who asked it really needs to be again their own advocate and go online, go with their physician, go to their local support groups, go to their national groups, because they’re the ones that can give that local person their answer. There’s no one set answer for every country because there are some many variances.

Andrew Schorr:

Right. I do want to tell one of my favorite stories. I had a friend Jan Rin in Dublin, Ireland. She had a tremendous problem with more advanced chronic lymphocytic leukemia, one of the conditions I have, no trial for her there. She heard about Imbruvica being studied in Leeds, England, different health system, national health system. She was in Ireland, didn’t have it. She got permission from the Irish government to go over to Leeds and be in Dr. Hellmann’s trial there, and I think it saved her life. She would tell you that. So she had to be pushy. There were newspaper articles. She had to do lots of things to make it happen. It’s going to be varied by country but it starts with…

Jim Omel:

…drug like the one you mention, and it’s not available in the country, and there’s so much of that in myeloma. We have many, many drugs in the US that they don’t have in other parts of the world, and it would be so sad to be a patient in those countries, know that a treatment like that is available but not have access to it. So we all need to work to get these drugs available to patients wherever they’re at.

Andrew Schorr:

Right. Amen. I want to just get some final comments from you. We may just go a couple minutes over. So, Mike, the process is improving, I hope, you’re working on it. Can we feel confident that these gaps, if you will, improving it for prevision medicine, more awareness among the doctors wherever we may go, financial assistance, working with the insurance companies, are you working on it so that this process, we can have some improvement and hopefully have higher levels of enrollment and can get drugs approved quicker?

Dr. Thompson:

Yeah, I think we’re all very concerned about it. We should all be aligned in having more patients on trial, moving things faster and getting it done more cheaply. And I think we’re making progress. It’s not as fast as any of us want, but we’re all trying to move the ball forward.

Andrew Schorr:

Okay. So, Mike, it comes—excuse me. Jim, it comes down to us then as patients. We have to push, right? We have to see what’s within ourselves, what are we willing to do, understand our clinical situation and what’s going on for our cancer, and we’ve got to push, right?

Jim Omel:

And one of the things we need to push for are more interesting trials. We need to make pharma companies put up their drug against another pharma company’s drug. I think it’s so troubling when they’re afraid to take big steps. They just take little, incremental steps with their trials. If we can put drug A of one company versus drug A of another company—pharma companies are really reluctant to do those kinds went trials, and yet those are the kind that would be exciting to patients. I could give certain names of myeloma drugs, but we won’t get into that. It just needs—we need to get better, more interesting trials, and that will attract patients.

Andrew Schorr:

Okay. So I want to just put in a plug for something. We started something at Patient Power called the Patient Power Ambassador Program, and you can see it listed on our site, where you can share your voice. So we can all work with Jim, work with Dr. Thompson, and we cannot just be getting what’s right for us, but we can push on this process. So please consider doing that. Because I want to thank you, Jim Omel, for not just getting what’s right for you as a myeloma patient, but working on these government panels and with advocacy groups to try to advance it for all of us. Jim Omel, thank you for doing this.

Jim Omel:

Thank you, Andrew. It’s a pleasure to do this, and I’ll keep doing it.

Andrew Schorr:

Yes. And long life, Jim. Thank you.

Jim Omel:

Thank you.

Andrew Schorr:

And, Dr. Mike Thompson, thank you, Mike, for your leadership too and those extra hours put in, not just for programs like this but all the clinical research speaking to industry and the government to try to improve this process. Thanks for being with us, Dr. Mike Thompson.

Dr. Thompson:

Thanks for having me on, and I think this is the some of the most powerful patient educational material that people can get, this type of program.

Andrew Schorr:

All right. Thank you so much. So, folks, we’re all in this together. So you have your own issues about whether you know about trials, whether you want to be in a trial, that’s right for you or a loved one, whether it’s close to home, not close to home, so—but we have these discussions. So please look ongoing at the clinical trials mythbuster series. The let us know how we did today. You can always write to me, andrew@patientpower. Our producer, Tamara, T-A-M-A-R-A, at patientpower.info. And talk to your own doctor and your own healthcare team about clinical trials and where they line up, what are the obstacles, for you participating. And let’s see if we can improve this process and ultimately have more medicine that can lead to a cure for us be available sooner. Thank you for watching. We’ve done our best today, but this is an ongoing discussion. In Carlsbad, California, I’m Andrew Schorr. Jim joined us from Nebraska, Dr. Mike Thompson joined us from Wisconsin. Worldwide, we’re here for you. Remember, knowledge can be the best medicine of all. Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.


Please remember the opinions expressed on Patient Empowerment Network are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Myeloma Patient Cafe® June 2018 – Participating in a Clinical Trial

Host and Multiple Myeloma patient, Cheri Rineker, leads a panel of Multiple Myeloma patients who have all participated in a clinical trial. The panel discusses what it’s like to join a clinical trial, how they got into a clinical trial, and what it takes to be in a clinical trial.


Transcript:

Cherie Rineker:

My name is Cherie Rineker, and I will be your host today.  Today we will be discussing what it’s like to join a trial, how we got in one, and what it takes to be part of a trial.  We have a lot to cover, and we have four guests that were kind enough to take time out of their busy days to share their experiences with us.  So having said that, I would like to start by asking you, Brian, where are you from, when were you diagnosed and what were some of the trials you were part of?

Brian Helstein:

I am from Southern California I’m one of the five people you will ever meet who was actually born in Hollywood.  I was formally diagnosed in February 2011.  In retrospect, symptomatic throughout most of 2010, but that’s with 20/20 hindsight.

At the time that I was considering undergoing a stem cell transplant I was offered one clinical trial through City of Hope which formally is identified as BNTCTN0702.  It was a three‑arm stem cell transplant trial where one arm would get two stem cell transplants, one arm would get a single stem cell transplant and go directly to maintenance, and a third arm would get the stem cell transplant, go through consolidation therapy and then go to maintenance.  And I was in that third arm at the time that that was offered to me‑‑I work in higher ed, and at the time that that was offered to me I said, certainly.  If I’m going to be sick somebody besides me needs to learn from this.  So I signed up for that.

And when I was going through the consolidation therapy they asked me if I wanted to participate in another trial that was‑‑that was attempting to monitor my maintenance and so that they wanted me on a specific maintenance regimen, and I signed up for that one.  So I was on maintenance from that from spring of 2012 through September of 2017.

Cherie Rineker:

Excellent.  Thank you, Brian.  What about you Matt?  Where are you from and what year were you diagnosed?  What studies have you participated in?

Matt William:

Hi, Cherie.  I grew up in Santa Cruz, California, but I was diagnosed in 2011 in Anchorage, Alaska, and then eventually moved to here in Kauai.  I live in Kauai now.

And I have been in four different trials.  The first one I don’t remember.  It was pretty minor, just a different combination of drugs that were common drugs.  And then my second one was my cells with radioactive antibody isotope injection and then followed by an allotransplant.  And I had a stem cell transplant before that, so that was my second one within a year.  And then I wasn’t given much time left, and I was sent to City of Hope for a study on an agent to help with deletion 17p, and that was followed‑‑let me look at my notes here.  I dasanutlin and (?) ixazomib with dexamethasone, and that helped bridge the gap.  I wouldn’t have made it to my CAR‑T trial without that.  And then eventually just recently finished up a CAR‑T cell trial in Seattle.

Cherie Rineker:

Excellent.  Thank you.  Eric, can you tell us where you’re from?

Eric Wolf:

Yeah, thank you.  I’m from Southern California also, grew up in Pasadena, California.  Was diagnosed in 2012 with a vertebra collapse.  That’s how my disease presented.  I have been on four different trials.  Post transplant in 2012, I was part of a shingles vaccine trial.  Don’t know if I got the placebo or the vaccine.  It was one of those types of trials.

Since then I was on a drug trial that did not work.  And then most recently I am currently on a trial.  It’s an antibody‑drug conjugate trial through City of Hope, and as part of that I did a gene sequencing trial.  So four different trials, and currently on an antibody‑drug conjugate trial.

Cherie Rineker:

Excellent.  Thank you.  Thank you.  And last but not least, Barb, could you tell us a little about yourself?

Barb Hansen:

Hi.  Thanks, Cherie.  This is a great opportunity, I think.  I am from Morrison, Colorado, which is a small town outside of Denver.  I was diagnose in 2006, December 6.  This was after breaking a rib back in March, so it took quite a while to get my diagnosis.  And I had been in a clinical trial, the CAL GB100104, which was the trial that helped set the protocol for stem cell transplants now.  That happened back in October of 2007.

And my doctor had talked about a stem cell transplant for me, and then later I found out, like two weeks later I found out it was going to be part of this trial and didn’t want to participate.  And when they said it was going to be a 15‑year trial I said, yeah, sign me up.  So I had the stem cell transplant October of (?) 2017, and then was given the maintenance drug the following February, and then took that maintenance drug for five years and have been in remission ever since then.

Cherie Rineker:

Excellent.

Barb Hansen:

Thank you.

Cherie Rineker:

Thank you, Barb and everybody.  Well, many of you know me.  I was diagnosed in November of 2012 after six months of much back pain and severe fatigue and was diagnosed with multiple myeloma while in the ICU.  And they found three tumors on my spine.  One had gone into my spinal cord, and they were surprised I was still standing.  And then I did nine induction therapies which only brought my counts down to 80 percent and my bone marrow.

But they went ahead and did a stem cell transplant followed by another, and then I went through a total of 13 lines of therapy.  And when the 13th wasn’t successful, relapsed again in December of last year, I told my oncologist that‑‑who wants to put me on four chemo drugs instead of the usually three, I said I want to tryout the CAR‑T.

So very, very sick, I started making the trips to Sarah Cannon, which is about a 14‑hour drive from our house.  And was accepted into the trial and received my CAR‑Ts on the 12th of March, and a few weeks later I showed no myeloma at all in my blood and then also none on the PET scan and none in my bone marrow.  So what 13 lines of treatment were not able to do over 65 months, CAR‑T basically gave me my life back within weeks.  And I’ve become a big proponent of trials ever since.

So what’s a myeloma clinical trial like?  The experiences are probably as vast as the amount of patients that are in it.  Matt, allow me to start with you.  What made you decide to join a trial, and how did your experiences compare to the actual expectations that you had?

Matt William:

Gosh, with my most recent trial I was at the end of the road.  It was the last house on the block, so I was eager to get in and did everything I could to do that.  I was turned down all over the place.  I was on the list in several places including China, and I was told that I only had a couple of months to live and that I probably wouldn’t‑‑there was a‑‑I had an allotransplant so I was being turned down because of that.

And then Seattle Cancer Care Alliance came up with a study that I heard about just through word of mouth.  Although I was a patient there I was not aware of it.  Somebody told me on social media about it, and I got my name on the list.  And my doctor told me I wouldn’t make it, that it wasn’t going to open in time for me, and he was kind enough on his own time to do some searching for me and found the City of Hope trial that targeted my 17p deletion problem.

And so I was willing and eager and trying to get in everywhere and was continually disappointed being turned down, and I thought it was going to happen again there for sure.  And I was lucky, there was a doctor, the Dr. Green there was‑‑opened it up a little bit.  There was many reasons for him to exclude me, but somehow I just squeaked in there, and my story is very similar to yours.  Shortly after, in 28 days, I had no sign of myeloma in my marrow or my blood.

There was a little bit left in my PET scan, but I just got back last week from Seattle and there’s zero sign of myeloma now after my 90‑day test.  So I’m just so grateful for clinical trials and to be able to finally get into the CAR‑T trial.

Cherie Rineker:

Yes, Matt.  We’ve gone this journey together, and I’m so, so thrilled to hear about your results from last week.  Brian, you can you tell us about why you decided to join the trial?

Brian Helstein:

Seriously, I really have spent my entire working life‑‑I’m getting ready to retire, and I’ve spent 50 years in higher ed.  I really, really had no clue at the time of diagnosis life expectancy or anything along those lines, and I figured, you know, that I was going to learn how to deal with this, that or the other infirmity the treatment was going to cause, and other people needed to know how to do that and do it better.  They needed to learn from my experience.  It wasn’t‑‑it just wasn’t something I was prepared to take with me.

And I must say I’m somewhat rebellious about things.  I have a dermatologist at this point who does not like me to go running out in the sunlight without a hat, without a long‑sleeved shirt.  If he had his way, I would also have ski mask and tights on.  No exposure to sunlight, and I have been known to go for a long run in my running shorts and shoes.

This was not going into a clinical trial where I was going to be told you’re going to take these drugs on this day, you’re going to show up at such and such a time on that day.  I knew that I was going to have to be disciplined, and I was going to have to follow exactly the protocol of the trial if it was going to be of any benefit to other people.  So I made that compromise.  I’ll go running without a shirt once a month or whatever, and I will be at the doctor’s office at, you know, 10:15 if that’s the time I’ve been summoned for.

I don’t know any other way to put it.  It was a matter of being disciplined so I could share so that others could benefit from this.

Cherie Rineker:

Very good reason.  Very good reason, Brian.  Thank you for sharing.  Eric, why did you decide to join a trial?

Eric Wolf:

Yeah, I think in some respects they are easy, right?  So the one that I did having to do with‑‑what is it, the shingles trial, it was there, it was offered to me.  It didn’t require much of me but calling in and reporting once a month on what‑‑if I experienced anything.  It was really easy, and so in some sense there’s those types of clinical trials that are just sort of tag‑ones to what we’re already doing.  Same thing with the extra marrow that was collected to do my gene sequencing.  That was not really a big deal.

But like yourself and Matt, there are other clinical trials that we seek out on our own part because we need those to manage our disease, and that was the case with the other two trials I’ve been on.  You know, the one I’m on now is because, as much as we don’t like to admit it, our options are limited, and so this is a trial that’s there and available and the timing lines up, and so you do it.

Cherie Rineker:

Excellent.  So anybody else besides Matt and I has had to travel long distances for their trials?  And then Barb, I’d like to ask you the same question as well, so maybe you can answer that.

Barb Hansen:

Well, I certainly didn’t have to travel.  I’m right here in the Denver area.  I think what really helped me make the decision, and my family helped also, is that I really felt confident with this new doctor I was seeing.  I had seen someone for five months, a hematologist‑oncologist who was not a specialist in multiple myeloma, and then after attending a stem cell seminar and this doc answering five questions in like five minutes I just felt very confident with him.  So I started, I transferred my records and just felt very confident with my healthcare team, and it was his recommendation.

Also, I have kind of a science background and my son does cancer research, and I know that the new science isn’t going to happen without clinical trials.  I’m a real advocate for clinical trials and did‑‑I volunteered for Colorado Cancer Research Program, which coordinates all the trials.  So it’s been a rewarding experience.

Cherie Rineker:

Thank you, Barb.  I see you wrote an article at one point about to trial or not to trial, that’s the question.  And I was very surprised in my research how few people actually participate, grownups versus children.  And I found it wasn’t just the grownups fault, or not wanting to do it, but it’s actually not as easy as Matt and I found with the CAR‑T, and maybe some of you as well, to get into a trial because there are so many requirements in order to get into that.

Did any of you have that issue or an issue of travel, money, or health that made getting into a trial challenging?  Matt, start with you.  I know you had to travel very far.

Matt William:

Yes.  When was I was diagnosed I was in Alaska.  There wasn’t really a myeloma specialist, and we got online right away and found the Seattle Cancer Care Alliance, and we’re really happy there and found a great doctor there.  But we had‑‑for my first transplant we relocated down there for 10 months.  Luckily, they had a little school and some‑‑for our kids and some housing.  But it was expensive.  It wasn’t free.  They had a social worker that helped us a lot, and we did some fund‑raising.  Lots of really great friends that helped out and just really streamlined our finances and our bills and sold a lot of stuff to fund it.

And, yeah, it was very expensive, and yeah, over time it’s really taken a toll on all of our savings and investments and all of that stuff.  But it’s worth it, you know.  Money can be remade and we can live simpler.  And so it was very much worth it.  If I wasn’t flexible with that type of stuff I wouldn’t be alive, so I had to do it.  And I would go to any lengths to find help and an answer to my problem.

Cherie Rineker:

Right.  Right.  Thank you.  Thank you.  I totally understand what you mean, having a young child in my family myself.  We have four beautiful daughters.  So thank you.  We’re glad you chose to hang in there.

Can anyone tell me about how they found out about the trial they joined?  Eric, can we start with you?  Today, online offers a tremendous amount of resources.  Which one, if any, did you use, and how do you stay informed about the latest trials?

Eric Wolf:

A little bit like Matt.  I made the decision to move to an area where I knew there would be good support when retired from the military, so I moved up into the LA area near City of Hope knowing that they had fantastic care and availability of trials and other things like that.  So I get most of my information through them.

Also, of course, read blogs and read information from the IMF and other things that are coming out.  And that all leads to trying to make the best decision.  So in the case of my current trial it was a matter of talking with my doctor and look at actually three different trials that were available, any of which could have been a good fit for me.

And then it’s a matter of which one‑‑then it’s kind of a matter of timing, right?  It’s just are you sick enough, ironically, to meet all the qualifications for this or that particular trial.  And so that’s kind of how I made the decision in concert with my doctor looking at the options available.

Cherie Rineker:

Right.  Thank you.  Thank you for sharing that.  Brian, what about you?

Brian Helstein:

I was on the fence about having a stem cell transplant, not on the fence about joining a trial.  And the‑‑my caregiver, my darling wife, basically pushed me off the fence and said, you will have this.  I’ve heard your doctor say that you will have a much better opportunity for long‑term survival if you go through with this.

So at City of Hope, as I was being interviewed and prepped for the stem cell transplant, they gave me a list of options which included amongst other things the participation in a trial.  And I was impressed with what they were looking at.  I was impressed with what the options were.  Unlike Eric’s comment about the shingles trial where he might have gotten a placebo, there was no placebo involved here.  There was standard of care treatment, there was standard of care plus and standard of care plus plus, which was what was going to be offered.

So it was at that point a fairly easy decision saying, okay, I have made this commitment to go ahead with the transplant, so let’s see about going ahead with the trial and, as I say, being disciplined enough for follow directions.  But it wasn’t‑‑there was no hesitation about it.  It was not something where there was a specific start date, again, like Eric, where I needed to fit in or I needed to be so sick or so healthy.  It was very much you’re going to do a stem cell transplant, and then beyond that we’re going to put you into one of these three arms and we will monitor you from that point.

Cherie Rineker:

Right.  Right.  Thank you, Brian.  Barb, I’m not sure.  Did I already asked you this question, or do you have anything to add?

Barb Hansen:

Well, my stem cell transplant was part of a clinical trial, and when my doctor said to me with a stem cell transplant you might be able to take a drug holiday.  That was appealing to me, so that combined, you know, being part of the clinical trial then was a bit of a driving force.  I hated being on dexamethasone.  I did not sleep well for, well, a long time.

And then, you know, I just can’t encourage people enough to find a multiple myeloma specialist who really knows this complicated disease and treats people individually and just knows what’s best for the patient.  And I was very glad that I joined the clinical trial, and I certainly advocate for them whenever I get a chance to.  Thank you.

Cherie Rineker:

Excellent.  So what advice do any of you‑‑do we all have for those myeloma patients that feel overwhelmed now and through the entire process, what they can do when they feel they’re running out of time or options?  What is it that you would like to tell them?  What has helped you on the internet?  I know Brian at SparkCure really helped me to find the trial that I got into, BB2121, a Celgene CAR‑T trial that ended up giving me my life back.  Just like Matt, I only had a couple months left.  What would you tell others?  Eric, do you maybe care to answer that first?

Eric Wolf:

Yeah.  So this disease of course is real science‑e, and we’re always thinking one step ahead, what’s the next thing, what’s the next thing?  So part of that calculation should be clinical trials, and so you have to keep up with what’s going on with those.  There’s a lot of information out there in different blogs on Cancer Care Network, on Sparks, and those types of things.

Of course, if you’re fortunate enough to be associated with a Cancer Research Center like City of Hope, then that’s a great opportunity.  They have posters throughout the campus about different trials that are going on, and of course I can reach out to my doctor at any time and look at those things.  But I think that all goes into our calculation of how we’re going to manage our disease and what’s the next step for us.

It’s‑‑different people have‑‑and I’ve ebbed and flowed over the years about how much I want to be involved, and sometimes you just want to take a break.  You just want to just do whatever my doctor says, and I don’t want to think about this disease for a while.  I just want a couple of months off.  We have that option, but it always comes back and comes to the forefront.

So I think as‑‑the advice is to look ahead, think ahead, keep up with what’s out there, but don’t let it overwhelm you.  At some point you have to live your life and just not‑‑you can’t live for the disease, live every day thinking about the disease.

Cherie Rineker:

Right.  I think we all agree with that, Eric.  Matt, what would be any of the advice you would give?

Matt William:

Just going back to how I accessed some of the trials.  It started with me, just through my doctors.  I had three trials that were just recommended by my specialist, and then it led to‑‑once I got to the CAR‑T cell therapy it was a little harder to find.  And I started with the Leukemia and Lymphoma Society, and they were very helpful, and they actually taught me how to do a little searching myself.

And then I got into some Facebook chat room type stuff where I was getting more information, and that’s where I met you.  And you recommended SparkCure, so it kind of led to that.  And I was doing my own stuff, and I met couple other people that kind of were like Brian, helping out.

But ironically, it turned out to be, you know, I like sending little messages, private messages to people and making acquaintances, and I became friends with this guy, Grant, from South Africa.  And he’s the one who told me about the trial that I finally got into, and it was at the very hospital that I was at, but I was unaware of it.  And he told me so early I got my name in there.

So I think, leading up to your question, persistence, you know, and don’t give up.  And just take it one day at a time.  And I like the advice of don’t get overwhelmed with it and just keep a good attitude.  And then ultimately be flexible.  There’s a lot of help out there and I’m continuing to be helped with my air fare and stuff like that, I forgot to mention before.

And so there’s a lot of‑‑don’t get overwhelmed by the money.  There’s some help out there for that, too.  And just one day at time and don’t give up and just try to reach out to other people and get‑‑the personal information, one patient to another online probably ended up being the most beneficial to me.

Cherie Rineker:

Thank you, Matt.  And I completely agree with you.  I’m pretty busy on Facebook myself, and I had people pushing me when my body and my mind could not handle any more and I wanted to give up.  And there was one lady in particular who just kept nagging me about it, and just to quiet her up I started following her advice and stuff.  And then one thing led to another, and I’m sitting here today because of these personal experiences.

And just the other day there was a gentleman who just basically said, I’m at the end of the line, can’t do no more, and I’ve been working really hard today and yesterday to write letters and talk to my doctors and to try and get him, because I know when we’re that sick sometimes it’s really hard for us to do it ourselves.  So absolutely there’s support you can get online‑‑

Matt William:

One more thing, Cherie.  I forgot to mention, it’s so important, Patient Power has been amazing with their videos and these interviews, and it really helped get me pointed in the right direction as well and some hope about CAR‑T cell and a little extra information and got me excited and added some hope to my journey.

Cherie Rineker:

Absolutely.  Absolutely.  We owe a lot to Patient Power.  What about you, Barb?

Barb Hansen:

I’m very pro clinical trials, and I do have a couple of venues where I’m able to encourage people to check out that option.  One is our multiple myeloma journey partner program, and because I tell my story there having had a stem cell transplant I also include the clinical the trial stories.  And so when I’m doing that I encourage people to check out the possibility, the option of going through a clinical trial.  And here in the United States we really need to encourage people to do that.

And then the other avenue I have is through the Leukemia and Lymphoma Society first connection program.  The Patti Robinson first connection program where I get calls from the society asking me if I’m available to talk to a person who is in another part of the country or here in Colorado.  And so it’s a person who just wants a call.  They’re either newly diagnosed or they’re going to go through a stem cell transplant or they’re considering a clinical trial, and so we chat and talk and I, you know, point out what I’ve been through and what has worked for me.

Always encourage them to talk to their doctor, and I don’t give medical advice by any means, and it’s encouraging.  Very rewarding to talk to people like that.

Cherie Rineker:

Thank you, Barb.  Yeah, you just taught me about two things I’d never heard of, so that is wonderful.  And I really think when patients talk to each other we can tell other things that the pretty pamphlets that are sent along with our Revlimids or our Velcades doesn’t always talk about all the things that we really experience.  So it’s wonderful to hear a person who’s been there explain things to us.  You, Brian?

Brian Helstein:

What I would tell somebody, first of all, is take a deep breath.  It ain’t going to kill you today.  And then the second thing, as we move forward with this, after you’ve had that deep breath, start evaluating what’s important to you, how hard are you prepared to fight this.  This goes to what Matt was talking about, the kind of thing that Eric was talking about.  What drives you?  What motivates you to keep going, and to keep those things in your mind?  It will make a tremendous difference in how you approach your various treatment options, the people you work with.

Barb was just talking about talking with, working with her doctors, and one of the things that I think all of us will agree on is you have to feel comfortable with your doctor, and if you don’t, it’s time to find a different doctor, a different treatment facility, whatever.  You need to be comfortable as you’re working with these people.

And, again, I think it was you, Eric, said sometimes you want to turn your mind off and stop worrying about this thing for a little bit.  I find that that’s fairly easy to do as long as I keep in front of me why I’m prepared to keep fighting, to keep going.  At that point, having made that decision, having put that focus on, it makes it easier for me not to focus on being sick.  And that’s something I would tell somebody, is why are you here?  What do you want to do with the time you’ve got left?

None of us are getting out of here alive.  Seriously.  All a diagnosis of multiple myeloma does is say, okay, you’ve got something that can kill you, and now you can put a name on it.  That’s bringing home in a very visceral way something that we probably intellectually knew but were not emotionally prepared to deal with.  And so focusing in on what’s important becomes very important part of moving forward.

Cherie Rineker:

Having a purpose in life is so very important.  I always tell people that even when you have cancer you can beat this disease if you keep in your mind you can, like you said, know what’s important, why you’re fighting to stay here, and then just do what you have to do.  Absolutely.

I would like to ask one final question of all you, all my guests here today.  How is life treating you today?  How are you feeling?  How are the drugs doing?  How are the side effects?  How are you sleeping?  And what is motivating you?  I know, for you, Brian, it’s your running, correct?

Brian Helstein:

Well, I’m working on the retirement actually.  Running is just like you brush your teeth in the morning.  That’s just a normal activity that I do.  Yeah, it’s just something that is part of my daily life, but, no.

I’ve been running a program for the University of Southern California, for example, that enables access to all of our licensed electronic resources, books, journals, databases, for the last 13, 14 years.  And I’m trying to clean up my sloppy programming, document my work, and train the people who will be my successors.  So that’s my real daily operational motivation at this point.

I’m not doing anything other than looking forward to some silly things in retirement.  I want to be on the Champs‑Élysées one day when the Tour de France ends.  Okay?  It always ends on the Champs‑Élysées.  That’s one of my goals in life.  It’s not a major driving force.  I have five adult children.  I would love to see some grandchildren.  That’s something you can’t control.

Cherie Rineker:

Right.  Eric, what about you?  How are you doing these days?  You look really healthy.  All of you, by the way.  Looks like we’ve taken on the beast and we’re winning.

Eric Wolf:

Like Brian, I’m trying to retire again.  After one retirement from the military I’m trying to retire again and just kind of working on some other things.  The clinical trial I’m on now, it’s been very rough.  It’s pushed my blood counts down, so I’ve had trouble with bruising, had trouble with shortness of breath and just getting enough energy.  So working through that, and who knows where this will lead, maybe CAR‑T or something else.

But, ultimately, the things that I like to do, I like to ride my motorcycle.  I like to backpack and camp, although the backpacking has been kind of cut short these days.  Just have done a lot of that over the years.  And looking forward to spending more time with the grandbaby.  We have a two‑year‑old granddaughter now, so enjoying that time.  I will say though, ultimately, my hope is in eternity.  And that’s from my Christian faith.  I’m enjoying life.  I’m enjoying fighting the disease, I actually am, and I’m positive about that.  But, like Brian mentioned, we’re all‑‑we’re all going to die eventually, so my hope is ultimately in eternity and the joy of that.  So that’s kind of where I am.

Cherie Rineker:

Thank you for sharing.  Very nice.  How about you, Matt?  Girls keeping you busy?

Matt William:

Gosh, yes.  I have so much to live for.  And I just turned 50.  We didn’t think that was going to happen.  We’re expecting a grandchild in December, I didn’t think that was going to happen.  We just found out about that.  And, you know, I really like what you just said.  You know, cancer hasn’t been all that bad to us.  It’s been‑‑we’ve had our share of struggles.  But I’ll tell you what.  Our quality of life has actually gotten better.  It’s brought us closer together, closer to our god, and we just really don’t take things for granted so much anymore.

And there’s a lot more to look forward to in the future.  I’m an avid surfer, or was.  I haven’t been out in the surf in over a year, and‑‑because of some phone problems, but I’m thinking that’s not too far away, that I’ll be able to start slowly back at that.  And just continuing to raise these kids and enjoying life, you know.  And just one day at a time, not worrying about what’s coming next all the time, you know.  I feel like I have that little break right now.

Cherie Rineker:

Absolutely.  Enjoy it, enjoy it, my friend.  Thank you.  What about you, Barb.  What are your aspirations?

Barb Hansen:

Well, I’m just so thankful to be here.  When I started Googling back in 2006 and even talking to my dear doctor, Dr. Jeff (?) Mathes at Colorado Blood Cancer Institute, Richards Rocky Mountain Cancer Center, you know, back then the average life expectancy was three, four years, and now it’s upward of 10, and I’ve beat that so a far.  So I am just so thankful to my healthcare team and just grateful to be here enjoying my two grandchildren, who I did not think I would have either.  Forest and Estelle, they are the joy of our lives.  Really enjoy them.

I golf a little, nine holes.  I do get tired because.  Of all of back issues, the bone fractures that I had, I do tire.  I love doing the volunteer work that has come my way, and now there’s more work they’ve asked me to do primarily through our church.  And I’m going to have to start saying no.  You know, I’m just taking a break and really enjoying life.

Tomorrow I’m going to Boise to talk to a support group about my journey, and I look forward to those times.  It’s really neat getting to be with other people who have this crazy disease and just showing them I’m still here.  I went through a stem cell transplant, a clinical trial, and it’s encouraging to help give other people‑‑help them with their journey and be hopeful.  Faith, family, and just enjoying the outdoors, creation.  That’s what it’s all about.  Thank you.

Cherie Rineker:

Yes, thank you all.  For me too it’s my family.  It’s also I became a huge advocate just for myeloma patients.  Because it took so long for them to diagnose me I always thought if my story and my symptoms are out there and somebody is seeing a YouTube of mine and that makes them go to a doctor and find out and say, hey, I want an Kappa light chain test or a Bence Jones 24‑hour urine test or anything.

Or even a doctor that would become more aware because we are putting ourselves out there with our stories, and if even just one person won’t be diagnosed with stage III but maybe as stage I and have a better chance of long‑term survival then I’m extremely grateful for putting myself out there.

So besides enjoying my family I really helping Patient Power and anybody else who comes knocking on my door, whether it’s through Facebook or companies giving talks, giving interviews, anything like that.

So I want to thank you panel for sharing your stories, giving your advice with us today.  As we all know, myeloma is a very difficult, painful disease to control, and I know without online support and things like Patient Cafe and SparkCure it would be a lot harder for me and I likely wouldn’t even be here today.

Thanks also to our listeners for tuning in.  We hope we were able to answer some of your questions about trials and how to get into them.  Reach out to Patient Power with any questions, please.  And we hope you’ll tune soon in again for our next show.


Please remember the opinions expressed on Patient Empowerment Network are not necessarily the views of our sponsors, contributors, partners or Patient Empowerment Network. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.