Tag Archive for: imaging

What Questions Should Patients Ask About Joining a Clinical Trial?

What Questions Should Patients Ask About Joining a Clinical Trial? from Patient Empowerment Network on Vimeo.

Before participating in a clinical trial, what questions should you ask? Dr. Pauline Funchain of Cleveland Clinic shares critical questions patients should ask their healthcare team when considering a clinical trial.

Dr. Pauline Funchain is a medical oncologist at the Cleveland Clinic. Dr. Funchain serves as Director of the Melanoma Oncology Program, co-Director of the Comprehensive Melanoma Program, and is also Director of the Genomics Program at the Taussig Cancer Institute of the Cleveland Clinic. Learn more about Dr. Funchain, here.

Katherine Banwell:

If a trial is recommended, what questions should a patient ask about the trial itself? 

Dr. Pauline Funchain:

Yeah. I mean, I think when it comes to that, I think that the important things to ask, really, are what are the drugs involved, and what your doc thinks about those drugs. 

I think, what is the alternative? So, again, we were talking about option A, B, and C. Is this option A of A, B, and C, or option C of A, B, and C? Are there ones like Cindi mentioned, where if you don’t do it at this point, you’re going to lose the opportunity, because you started on something else. Because a lot of trials require either that a person has never gone through therapy, and so this is sort of first line trial. But some trials are you have to be at the second thing that you’ve been on.  

So, these are the things that matter to know. Are you going to lose an opportunity if you didn’t do it now, or can you do it later, and what is the preference? And I think, practically speaking, a patient really wants to know what is the schedule? Can I handle this? How far away do I live from the place that is giving this trial? 

What are the locations available? Because if there’s a trial and you have to come in every two weeks, or come in four times in two weeks, and then once every month after that, that makes a big difference depending on where you live, what season it is, weather, that kind of stuff.  

And I think the question that you don’t really have to ask, but a lot of people ask, is about cost. So, medical care nowadays is complex, it costs money when you don’t expect it to, it doesn’t cost money when it’s – you just don’t know what will and what won’t. Financial toxicity is something that we really care about. Every center is really trying its best, but it’s hard to do in this type of environment. So, people then get concerned that clinical trials might be even more complex.  

I think clinical trials are much less complex in that way, because a lot more of it is covered by the sponsor, whatever that sponsor is, whether that sponsor is the National Institutes of Health, as a grant, or a pharmaceutical company.  

But, in general, a clinical trial really should cost the same or less than whatever the standard medical care is; that’s the way they’re built. So, many, many people ask us that question, but I think that is the question that probably is less important than what are the drugs, what does your doc think about this, are you going to lose an opportunity if there’s a different sequence, and does this fit into your life and your schedule, and people who can give you rides.  

Katherine Banwell:

Yeah, right.  Are there resources available to assist with the financial impact of a clinical trial? 

Dr. Pauline Funchain:

There are not specific resources for clinical trials; there are specific resources for patients in general, though. There are things like helping with utility bills sometimes, sometimes with rides, I think a lot of clinical trials do pay for things like parking. In general, many trials themselves have extra financial support in them. There was a trial I remember that paid for airfare and lodging, because there were only five centers in the country, and so we had people fly in, and the whole thing was covered. 

It depends on the trial. But in terms of outside of trials, there are always patient advocacy groups and things like that, where certain things can get covered. But often, the types of things that get covered by those groups are the same things that get covered with normal medical care. 

How to Find a Clinical Trial That’s Right for You

How to Find a Clinical Trial That’s Right for You from Patient Empowerment Network on Vimeo.

If you are interested in participating in a clinical trial, where do you start? Dr. Pauline Funchain of Cleveland Clinic shares resources for patients on where to find and access a clinical trial that’s right for them.

Dr. Pauline Funchain is a medical oncologist at the Cleveland Clinic. Dr. Funchain serves as Director of the Melanoma Oncology Program, co-Director of the Comprehensive Melanoma Program, and is also Director of the Genomics Program at the Taussig Cancer Institute of the Cleveland Clinic. Learn more about Dr. Funchain, here.

Katherine Banwell:

So, if a patient is interested in joining a clinical trial, where should they start? 

Dr. Pauline Funchain:

They can start anywhere. There are many places to start. I think their oncologist is a really, really good place to start. I would say an oncologist, depending on their specialties, will have a general grasp of trials, or a really specific grasp of trials. 

I would say that the folks who have the most specific grasp on trials – what is available, what isn’t available, what’s at their center versus the next state over center – are the academic medical centers; the ones that are sort of university centers, places like the Cleveland Clinic where the docs are specialized by the type of cancer. That group of folks will have the best grasp on what’s current, what’s available. 

And so, Cindi, your friend referred you. many people do say that. Just go to whatever your nearest university center is, just because there’s a lot more specialization in that sense. But I think it’s the age of the Internet, so people can look online. Clinicaltrials.gov is a fantastic place to look. It is not as up to date, I think, as something you can get directly from a person at a medical center, but it is a great place to start.  

There are many advocacy groups and websites that will point people to trials. I mean, there are Facebook groups and things, where people will chat about trials. But I think the detail is better at a site like clinicaltrials.gov, and even better with a cancer-specific oncologist at a academic medical center. 

You’ve Chosen to Participate In a Clinical Trial: What Are Next Steps?

You’ve Chosen to Participate in a Clinical Trial: What Are Next Steps? from Patient Empowerment Network on Vimeo.

What is it like to participate in a clinical trial? Dr. Pauline Funchain of Cleveland Clinic explains what to expect when joining a clinical trial and colorectal cancer survivor Cindi Terwoord shares her personal experience.

Dr. Pauline Funchain is a medical oncologist at the Cleveland Clinic. Dr. Funchain serves as Director of the Melanoma Oncology Program, co-Director of the Comprehensive Melanoma Program, and is also Director of the Genomics Program at the Taussig Cancer Institute of the Cleveland Clinic. Learn more about Dr. Funchain, here.
 
Cindi Terwoord is a colorectal cancer survivor and patient advocate. Learn more about Cindi, here.

Katherine Banwell:

Dr. Funchain, once a patient like Cindi decides to participate in a trial, what happens next? 

Dr. Pauline Funchain:

So, there is a lot, actually, that happens. So, there is a lead-in period to a trial. So, once you decide, it’s not like you can start tomorrow on a trial drug. What happens really, there’s a whole safety lead-in that we call an enrollment period, where there’s a long checklist of making sure that a person is healthy, and there’s nothing – no organ or anything in particular – where we would be worried about this particular drug. 

So, there’s a checklist, that way there are usually – sometimes there’s a new scan if the last scan is a little bit too old, just so that we know exactly what somebody looks like right when they walk into the trial and start the drug. There are usually some blood tests and procedures that come before, and some of the stuff – half of the blood is for the trial, and half of the blood is for scientist usually, so that they can work on some of the science behind what’s happening to someone on a trial, which is pretty cool.  

And sometimes there is a procedure – a biopsy or something like that – that’s involved.  

But, in general, the lead-in is somewhere usually between two and four weeks from the time somebody decides they’re willing to be on a trial. And there are some extra safety measures, like if you hear about a trial, you can’t go on the trial right away, there’s got to be sort of a thinking period that’s usually about 24 hours before you can literally sign your name on the line.  

But, yeah, I’d expect something about two to three weeks before going on a trial. And then once folks are on a trial, it’s kind of like treatment. It’s just getting the treatments when you get the treatments. Sometimes there’s extra checks, again for safety, on drug levels and things.  

Katherine Banwell:

Would you review the safety protocols in place for clinical trials?  

Pauline Funchain:

Yeah, sure. So, safety is number one when it comes to trials, really. There are guardrails on guardrails on guardrails. But in any clinical trial protocol, it actually starts even before the trial starts. So, whenever somebody wants to bring in a trial, or wants to start a trial – and this is true at any academic institution, or any institution that runs trials – the trial goes through something called an IRB, or an Institutional Review Board, and that board reviews it and says, “Look, is this safe, are we harming people, are we unnecessarily coercing people?” 

And they read through the whole thing. And usually there’s a protocol data monitoring committee that also looks at it, there’s usually two. And there’s a lot of checks that a trial has to go through to make sure it’s safe, and fair, for all participants. So, that happens first.  

And then once the trial opens, there is continual monitoring. Every visit, every number that’s drawn. Any visit, even if the visit isn’t at the hospital that’s running the trial, even if it’s at a local urgent care, all of those things end up getting reported back, and there’s a whole team of people besides.  

So, a patient will see the doc, or the nurse, or maybe sometimes a research coordinator, research assistant. But then there are all these research coordinators that sit in offices that review everything, put it in the computers, and then record everything that happens to someone on the trial.  

And all of that data actually goes to an external review organization, a clinical trial research organization. And what they do is, they look over all of the data also. So, it’s not just internal people checking, because internal people may be biased for the people that pay them, right? 

Katherine Banwell:

Right. 

Dr. Pauline Funchain:

All of that data goes to an external monitoring board also, to make sure that everything is going the way it’s supposed to go. 

Katherine Banwell:

Yeah. Cindi, in your experience, did you feel like safety was a priority? 

Cindi Terwoord:

Oh, definitely, definitely, yeah. They were very, very careful. Mine was a two-part; I had a vaccine along with this nivolumab (Opdivo).  

And so, they would have to give me the vaccine, sit there and stare at me, to make sure I didn’t faint or something, and that was a good half-hour.  

Then I got the immunotherapy, and I’d have to wait an hour after that before I started on the chemotherapy.  

Katherine Banwell:

Oh. 

Cindi Terwoord:

Yeah, they were in there watching me like a hawk, and I felt very safe, I really did. 

Katherine Banwell:

Dr. Funchain, what are a patient’s rights when they participate in a trial? 

Dr. Pauline Funchain:

So, the most important thing, I think, that Cindi mentioned before is, a patient can withdraw at any time. Any time. They can sign the paperwork, and the next second decide not to. They can be almost to the end of the trial and decide that they want to come off. The last word is always with the patient.  

I think the other thing, in terms of safety, you can see – so every patient before starting a trial gets an informed consent. It is multiple pages, there’s a lot of legalese in it.   

But they do try their best to make it as readable and understandable as possible, so that people can, even if they don’t have a medical background, kind of understand what they’ve gotten. The mechanism of what they’ve gotten, and what new drug they’re getting, and generally what are the risks and benefits.  

For instance, let’s say there’s genetic testing involved, there’s always clauses that tell you what that means, and how protected your genetic information is, that kind of stuff.  

So, it’s a very long thing. And again, once someone gets that, they have to have a certain amount of time before they can sign on the line. So, I think information education, and then the ability to come off if they find necessary. 

Katherine Banwell:

Yeah. What happens after a trial is completed? Is a patient monitored? And if so, how? 

Dr. Pauline Funchain:

So, that depends on the trial.  

Most trials do monitor after either the drug is complete, or the course is complete for a certain amount of time, and it depends on the trial. For some trials, it’s six months after; for some trials, it’s years afterwards. So, in melanoma, we have a trial that just reported out their 7-1/2-year follow-up. But it was actually the first immunotherapy combination of its kind that involved the drug that you had to need nivolumab.   

So, it is pretty cool. I mean, that combination changed the face of what patients with melanoma could come to expect from their treatment, so we’re all very interested to know what that kind of follow-up is. But, yeah, it depends on the trial.  

What Are the Risks and Benefits of Joining a Clinical Trial?

What Are the Risks and Benefits of Joining a Clinical Trial? from Patient Empowerment Network on Vimeo.

Why should a cancer patient consider a clinical trial? Dr. Pauline Funchain of the Cleveland Clinic explains the advantages of clinical trial participation.

Dr. Pauline Funchain is a medical oncologist at the Cleveland Clinic. Dr. Funchain serves as Director of the Melanoma Oncology Program, co-Director of the Comprehensive Melanoma Program, and is also Director of the Genomics Program at the Taussig Cancer Institute of the Cleveland Clinic. Learn more about Dr. Funchain, here.

See More from Clinical Trials 101

Related Resources:

You’ve Chosen to Participate In a Clinical Trial: What Are Next Steps?

Understanding Common Clinical Trial Terminology

How to Find A Clinical Trial That’s Right for You


Transcript:

Katherine Banwell:

Why would a cancer patient consider participating in a clinical trial? What are the benefits? 

Dr. Pauline Funchain:

So, I mean, the number one benefit, I think, for everyone, including the cancer patient, is really clinical trials help us help the patient, and help us help future patients, really.  

We learn more about what good practices are in the future, what better drugs there are for us, what better regimens there are for us, by doing these trials. And ideally, everyone would participate in a trial, but it’s a very personal decision, so we weigh all the risks and benefits. I think that is the main reason.  

I think a couple of other good reasons to consider a trial would be the chance to see a drug that a person might not otherwise have access to. So, a lot of the drugs in clinical trials are brand new, or the way they’re sequenced are brand new. And so, this is a chance to be able to have a body, or a cancer, see something else that wouldn’t otherwise be available.  

And I think the last thing – and this is sort of the thing we don’t talk about as much – but really, because clinical trials are designed to be as safe as possible, and because they are new procedures, there’s a lot of safety protocols that are involved with them, which means a lot of eyes are on somebody going through a clinical trial.  

Which actually to me means a little bit sort of more love and care from a lot more people. It’s not that the standard of care – there’s plenty of love and care and plenty of people, but this doubles or triples the amount of eyes on a person going through a trial. 

Katherine Banwell:

Yeah. When it comes to having a conversation with their doctor, how can a patient best weigh the risks and benefits to determine whether a trial is right for them? 

Dr. Pauline Funchain:

Right. So, I think that’s a very personal decision, and that’s something that a person with cancer would be talking to their physician about very carefully to really understand what the risks are for them, what the benefits are for them. Because for everybody, risks and benefits are totally different. So, I think it’s really important to sort of understand the general concept. It’s a new drug, we don’t always know whether it will or will not work. And there tend to be more visits, just because people are under more surveillance in a trial.  

So, sort of getting all the subtleties of what those risks and benefits are, I think, are really important. 

Katherine Banwell:

Mm-hmm. What are some key questions that patients should ask? 

Dr. Pauline Funchain:

Well, I think the first question that any patient should ask is, “Is there a trial for me?” I think that every patient needs to know is that an option. It isn’t an option for everyone. And if it is, I think it’s – everybody wants that Plan A, B, and C, right? You want to know what your Plan A, B, and C are. If one of them includes a trial, and what the order might be for the particular person, in terms of whether a trial is Plan A, B, or C. 

Participating in a Clinical Trial: What You Need to Know

Participating in a Clinical Trial: What You Need to Know from Patient Empowerment Network on Vimeo.

 Are you considering participating in a clinical trial? In this webinar, Dr. Pauline Funchain, cancer expert and researcher, discusses what to expect when joining a clinical trial, including patient safety and questions to ask your healthcare about trial participation. Patient advocate and colorectal cancer survivor Cindi Terwoord shares her experience and advice for other people with cancer considering joining a clinical trial.

Dr. Pauline Funchain is a medical oncologist at the Cleveland Clinic. Dr. Funchain serves as Director of the Melanoma Oncology Program, co-Director of the Comprehensive Melanoma Program, and is also Director of the Genomics Program at the Taussig Cancer Institute of the Cleveland Clinic. Learn more about Dr. Funchain, here.
 
Cindi Terwoord is a colorectal cancer survivor and patient advocate. Learn more about Cindi, here.
 

Katherine Banwell:    

Hello, and welcome. I’m Katherine Banwell, your host for today’s program. When faced with a cancer diagnosis, could a clinical trial be your best treatment option? Today, we’re going to learn all about clinical trial participation, what’s involved, and how you can work with your healthcare team to decide whether a trial is right for you.

Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you. All right let’s meet our guest today. Joining me is Dr. Pauline Funchain.

Dr. Funchain, welcome, would you please introduce yourself?

Dr. Pauline Funchain:      

Sure. Thank you for the invitation. So, I’m Pauline Funchain, I am a medical oncologist at the Cleveland Clinic. My specialty is melanoma and skin cancers. I also lead our genomics program here at Taussig Cancer Center.

Katherine Banwell:    

Excellent. Thank you so much for joining us today.

Dr. Pauline Funchain:       

Thank you.

Katherine Banwell:    

And here to share the patient perspective is Cindi, who is a colorectal cancer survivor. Cindi, we’re so pleased to have you with us today.

Cindi Terwoord:        

Thank you, nice to be here.

Katherine Banwell:    

Before we learn more about Cindi’s experience, I’d like to start with a basic question for you, Dr. Funchain. Why would a cancer patient consider participating in a clinical trial? What are the benefits?

Dr. Pauline Funchain:        

So, I mean, the number one benefit, I think, for everyone, including the cancer patient, is really clinical trials help us help the patient, and help us help future patients, really.

We learn more about what good practices are in the future, what better drugs there are for us, what better regimens there are for us, by doing these trials. And ideally, everyone would participate in a trial, but it’s a very personal decision, so we weigh all the risks and benefits. I think that is the main reason.

I think a couple of other good reasons to consider a trial would be the chance to see a drug that a person might not otherwise have access to. So, a lot of the drugs in clinical trials are brand new, or the way they’re sequenced are brand new. And so, this is a chance to be able to have a body, or a cancer, see something else that wouldn’t otherwise be available.

And I think the last thing – and this is sort of the thing we don’t talk about as much – but really, because clinical trials are designed to be as safe as possible, and because they are new procedures, there’s a lot of safety protocols that are involved with them, which means a lot of eyes are on somebody going through a clinical trial.

Which actually to me means a little bit sort of more love and care from a lot more people. It’s not that the standard of care – there’s plenty of love and care and plenty of people, but this doubles or triples the amount of eyes on a person going through a trial.

Katherine Banwell:    

Yeah. When it comes to having a conversation with their doctor, how can a patient best weigh the risks and benefits to determine whether a trial is right for them?

Dr. Pauline Funchain:       

Right. So, I think that’s a very personal decision, and that’s something that a person with cancer would be talking to their physician about very carefully to really understand what the risks are for them, what the benefits are for them. Because for everybody, risks and benefits are totally different. So, I think it’s really important to sort of understand the general concept. It’s a new drug, we don’t always know whether it will or will not work. And there tend to be more visits, just because people are under more surveillance in a trial.

So, sort of getting all the subtilties of what those risks and benefits are, I think, are really important.

Katherine Banwell:    

Mm-hmm. What are some key questions that patients should ask?

Dr. Pauline Funchain:      

Well, I think the first question that any patient should ask is, “Is there a trial for me?” I think that every patient needs to know is that an option. It isn’t an option for everyone. And if it is, I think it’s – everybody wants that Plan A, B, and C, right? You want to know what your Plan A, B, and C are. If one of them includes a trial, and what the order might be for the particular person, in terms of whether a trial is Plan A, B, or C.

Katherine Banwell:    

Mm-hmm. Let’s learn more about Cindi’s story. Cindi, you were diagnosed with stage IV colorectal cancer, and decided to participate in a clinic trial. Can you tell us about what it was like when you were diagnosed?

Cindi Terwoord:        

Yeah. That was in September of 2019, and I had had some problems; bloody diarrhea one evening, and then the next morning the same thing. So, I called my husband at work, I said, “Things aren’t looking right. I think I’d better go to the emergency room.”

And so, we went there, they took blood work – so I think they knew something was going on – and said, “We’re going to keep you for observation.” So, then I knew it must’ve been something bad. And so, two days later, then I had a colonoscopy, and that’s when they found the tumor, and so that was the beginning of my journey.

Katherine Banwell:    

Mm-hmm. Had you had a colonoscopy before, or was that your first one?

Cindi Terwoord:        

No, I had screenings, I would get screenings. I had heard a lot of bad things about colonoscopies, and complications and that, so I was always very leery of doing that. Shame on me. I go for my other screenings, but I didn’t like to do that one. I have those down pat now, I’m very good at those.

Katherine Banwell:    

Yeah, I’m sure you do. So, Cindi, what helped guide your decision to join a clinical trial?

Cindi Terwoord:        

Well, I have a friend – it was very interesting.

He was probably one of the first people we told, because he had all sorts of cancer, and he was, I believe, one of the first patients in the nation to take part in this trial. It’s nivolumab (Opdivo), and he’s been on it for about seven years. And he had had various cancers would crop up, but it was keeping him alive.

And so, frankly, I didn’t know I was going to have the option of a trial, but he told me run straight to Cleveland Clinic, it’s one of the best hospitals. So, I took his advice. And the first day the doctor walked in, and then all these people walked in, and I’m like, “Why do I have so many people in here?” Not just a doctor and a nurse. There was like a whole – this is interesting.

And so, then they said, “Well, we have something to offer you. And we have this immunotherapy trial, and you would be one of the first patients to try this.”

Now, when they said first patient, I’m not quite sure if they meant the first colon cancer patient, I’m not sure. But they told me the name of it, and I said, “I’m in. I’m in.” Because I knew my friend had survived all these years, and I thought, “Well, I’ve gotten the worst diagnosis I can have, what do I have to lose?” So, I said, “I’m on board, I’m on board.”

Katherine Banwell:    

Mm-hmm. Did you have any hesitations?

Cindi Terwoord:        

Nope. No, I’m an optimistic person, and what they assured me was that I could drop out at any time, which I liked that option.

Because I go, “Well, if I’m not feeling well, and it’s not working, I’ll get out.” So, I liked that part of it. I also liked, as Dr. Funchain had said, you go in for more visits. And I like being closely monitored, I felt that was very good.

I’ve always kept very good track of my health. I get my records, I get my office notes from my doctor. I’m one of those people. I probably know the results of blood tests before the doctor does because I’m looking them up. So, I felt very confident in their care. They watched me like a hawk. I kept a diary because they were asking me so many questions.

Katherine Banwell:    

Oh, good for you.

Cindi Terwoord:        

I’m a transcriptionist, so I just typed out all my notes, and I’d hand it to them.

Katherine Banwell:    

That’s a great idea.

Cindi Terwoord:        

Here’s how I’m feeling, here’s…And I was very lucky I didn’t have many side effects.

Katherine Banwell:    

In your conversations with your doctor, did you weigh the pros and cons about joining a trial? Or had you already made up your mind that yes, indeed, you were going for it?

Cindi Terwoord:        

Yeah, I already said, “I’m in, I’m in.” Like I said, it had kept my friend alive for these many years, he’s still on it, and I had no hesitation whatsoever.

I wish more people – I wanted to get out there and talk to every patient in the waiting room and say, “Do it, do it.”

I mean, you can’t start chemotherapy then get in the trial. And if I ever hear of someone that has cancer, I ask them, “Well, were you given the option to get into a trial?” Well, and then some of them had started the chemo before they even thought of that.

Katherine Banwell:    

Mm-hmm. So, how are you doing now, Cindi? How are you feeling?

Cindi Terwoord:        

Good, good, I’m doing fantastic, thank goodness, and staying healthy. I’m big into herbal supplements, always was, so I keep those up, and I’m exercising. I’m pretty much back to normal –

Katherine Banwell:    

Oh, good for you.

Cindi Terwoord:        

– as far as my strength. I like to lift weights, and I run, so I’m pretty much back to normal.

Katherine Banwell:    

Good for you. Thanks so much for sharing your story with us.

Cindi Terwoord:        

You’re welcome.

Katherine Banwell:    

Dr. Funchain, once a patient like Cindi decides to participate in a trial, what happens next?

Dr. Pauline Funchain:    

So, there is a lot, actually, that happens. So, there is a lead-in period to a trial. So, once you decide, it’s not like you can start tomorrow on a trial drug. What happens really, there’s a whole safety lead-in that we call an enrollment period, where there’s a long checklist of making sure that a person is healthy, and there’s nothing – no organ or anything in particular – where we would be worried about this particular drug.

So, there’s a check list, that way there are usually – sometimes there’s a new scan if the last scan is a little bit too old, just so that we know exactly what somebody looks like right when they walk into the trial and start the drug. There are usually some blood tests and procedures that come before, and some of the stuff – half of the blood is for the trial, and half of the blood is for scientist usually, so that they can work on some of the science behind what’s happening to someone on a trial, which is pretty cool.

And sometimes there is a procedure – a biopsy or something like that – that’s involved.

But in general, the lead-in is somewhere usually between two and four weeks from the time somebody decides they’re willing to be on a trial. And there are some extra safety measures, like if you hear about a trial, you can’t go on the trial right away, there’s got to be sort of a thinking period that’s usually about 24 hours before you can literally sign your name on the line.

But, yeah, I’d expect something about two to three weeks before going on a trial. And then once folks are on a trial, it’s kind of like treatment. It’s just getting the treatments when you get the treatments. Sometimes there’s extra checks, again for safety, on drug levels and things.

Katherine Banwell:    

Would you review the safety protocols in place for clinical trials?

Dr. Pauline Funchain:    

Yeah, sure. So, safety is number 1 when it comes to trials, really. There are guardrails on guardrails on guardrails. But in any clinical trial protocol, it actually starts even before the trial starts. So, whenever somebody wants to bring in a trial, or wants to start a trial – and this is true at any academic institution, or any institution that runs trials – the trial goes through something called an IRB, or an Institutional Review Board, and that board reviews it and says, “Look, is this safe, are we harming people, are we unnecessarily coercing people?”

And they read through the whole thing. And usually there’s a protocol data monitoring committee that also looks at it, there’s usually two. And there’s a lot of checks that a trial has to go through to make sure it’s safe, and fair, for all participants. So, that happens first.

And then once the trial opens, there is continual monitoring. Every visit, ever number that’s drawn. Any visit, even if the visit isn’t at the hospital that’s running the trial, even if it’s at a local urgent care, all of those things end up getting reported back, and there’s a whole team of people besides.

So, a patient will see the doc, or the nurse, or maybe sometimes a research coordinator, research assistant. But then there are all these research coordinators that sit in offices that review everything, put it in the computers, and then record everything that happens to someone on the trial.

And all of that data actually goes to an external review organization, a clinical trial research organization. And what they do is, they look over all of the data also. So, it’s not just internal people checking, because internal people may be biased for the people that pay them, right?

Katherine Banwell:    

Right.

Dr. Pauline Funchain:    

All of that data goes to an external monitoring board also, to make sure that everything is going the way it’s supposed to go.

Katherine Banwell:    

Yeah. Cindi, in your experience, did you feel like safety was a priority?

Cindi Terwoord:        

Oh, definitely, definitely, yeah. They were very, very careful. Mine was a two-part; I had a vaccine along with this nivolumab.

And so, they would have to give me the vaccine, sit there and stare at me, to make sure I didn’t faint or something, and that was a good half-hour.

Then I got the immunotherapy, and I’d have to wait an hour after that before I started on the chemotherapy.

Katherine Banwell:    

Oh.

Cindi Terwoord:        

Yeah, they were in there watching me like a hawk, and I felt very safe, I really did.

Katherine Banwell:     

Dr. Funchain, what are a patient’s rights when they participate in a trial?

Dr. Pauline Funchain:    

So, the most important thing, I think, that Cindi mentioned before is, a patient can withdraw at any time. Any time. They can sign the paperwork, and the next second decide not to. They can be almost to the end of the trial and decide that they want to come off. The last word is always with the patient.

I think the other thing, in terms of safety, you can see – so every patient before starting a trial gets an informed consent. It is multiple pages, there’s a lot of legalees in it.

But they do try their best to make it as readable and understandable as possible, so that people can, even if they don’t have a medical background, kind of understand what they’ve gotten. The mechanism of what they’ve gotten, and what new drug they’re getting, and generally what are the risks and benefits.

For instance, let’s say there’s genetic testing involved, there’s always clauses that tell you what that means, and how protected your genetic information is, that kind of stuff.

So, it’s a very long thing. And again, once someone gets that, they have to have a certain amount of time before they can sign on the line. So, I think information education, and then the ability to come off if they find necessary.

Katherine Banwell:    

Yeah. What happens after a trial is completed? Is a patient monitored? And if so, how?

Dr. Pauline Funchain:    

So, that depends on the trial.

Most trials do monitor after either the drug is complete, or the course is complete for a certain amount of time, and it depends on the trial. For some trials, it’s six months after; for some trials, it’s years afterwards. So, in melanoma, we have a trial that just reported out their 7-1/2-year follow-up. But it was actually the first immunotherapy combination of its kind that involved the drug that you had Cindi, nivolumab.

So, it is pretty cool. I mean, that combination changed the face of what patients with melanoma could come to expect from their treatment, so we’re all very interested to know what that kind of follow-up is. But, yeah, it depends on the trial.

Katherine Banwell:    

Dr. Funchain, are there common clinical trial terms that patients should know?

Dr. Pauline Funchain:    

Yeah, there are trial terms that people hear all the time, and probably should know a little bit about. But I think the most common thing people will hear with trials are the type of trial it is, so Phase I, Phase II, Phase III. The important things to know about that are essentially, Phase I is it’s a brand-new drug, and all we’re trying to do is look for toxicity. Although we’ll always on the side be looking for efficacy for whether that drug actually works, we’re really looking to see if the drug is safe.

A Phase II trial is a trial where we’re starting to look at efficacy to some degree, and we are still looking at toxicity. And then in Phase III is, we totally understand the toxicity, and we are seeing promise, and what we really want to do is see if this should become a new standard. So, that would be the Phase I, II, and III.

Another couple of terms that people hear a lot about are eligibility criteria, or inclusion criteria. So, those are usually some set of 10 to 30 things that people can and can’t be. So, usually trials only allow certain types of cancer, and so that would be an inclusion criteria, but it will exclude other types of cancers. Most trials, unfortunately, exclude pregnant women. That would be an exclusion criteria.

So, these are things that, at the very beginning of a trial, will allow someone to enter, or say, “You’re not in the safe category, we should not put you on a trial.” Many trials are randomized, so people will hear this a lot. Randomization.

So, a lot of times, there is already a standard of care. When there’s already a standard of care, and you want to see if this drug is at least the same or better, then on that trial, there will be two different arms; a standard of care arm and experimental arm.

And then in order to be fair, a randomized trial is a flip of a coin. Based on a electronic flip of a coin – nobody gets to choose; not the doc, not the patient. On that type of trial, you’ll either get what you would normally get, standard of care, or something new. So, that’s a randomized trial. Not all trials are randomized, but some are. And those are the things that people will run into often.

Katherine Banwell:    

So, if a patient is interested in joining a clinical trial, where should they start?

Dr. Pauline Funchain:    

They can start anywhere. There are many places to start. I think their oncologist is a really, really good place to start. I would say a oncologist, depending on their specialties, will have a general grasp of trials, or a really specific grasp of trials.

I would say that the folks who have the most specific grasp on trials– what is available, what isn’t available, what’s at their center versus the next state over center – are the academic medical centers; the ones that are sort of university centers, places like the Cleveland Clinic where the docs are specialized by the type of cancer. That group of folks will have the best grasp on what’s current, what’s available.

And so, Cindi, your friend referred you. many people do say that. Just go to whatever your nearest university center is, just because there’s a lot more specialization in that sense. But I think it’s the age of the internet, so people can look online. Clinicaltrials.gov is a fantastic place to look. It is not as up to date, I think, as something you can get directly from a person at a medical center, but it is a great place to start.

There are many advocacy groups and websites that will point people to trials. I mean, there are Facebook groups and things, where people will chat about trials. But I think the detail is better at a site like clinicaltrials.gov, and even better with a cancer-specific oncologist at a academic medical center.

Katherine Banwell:    

If a trial is recommended, what questions should a patient ask about the trial itself?

Dr. Pauline Funchain:    

I mean, there’s so many questions to ask.

Katherine Banwell:    

Safety is definitely one of them, right?

Dr. Pauline Funchain:    

Yeah. I mean, I think when it comes to that, I think that the important things to ask, really, are what are the drugs involved, and what your doc thinks about those drugs.

I think, what is the alternative? So, again, we were talking about option A, B, and C. Is this option A of A, B, and C, or option C of A, B, and C? Are there ones like Cindi mentioned, where if you don’t do it at this point, you’re going to lose the opportunity, because you started on something else. Because a lot of trials require either that a person has never gone through therapy, and so this is sort of first line trial. But some trials are you have to be at the second thing that you’ve been on.

So, these are the things that matter to know. Are you going to lose an opportunity if you didn’t do it now, or can you do it later, and what is the preference? And I think, practically speaking, a patient really wants to know what is the schedule? Can I handle this? How far away do I live from the place that is giving this trial?

What are the locations available? Because if there’s a trial and you have to come in every two weeks, or come in four times in two weeks, and then once every month after that, that makes a big difference depending on where you live, what season it is, weather, that kind of stuff.

And I think the question that you don’t really have to ask, but a lot of people ask, is about cost. So, medical care nowadays is complex, it costs money when you don’t expect it to, it doesn’t cost money when it’s – you just don’t know what will and what won’t. Financial toxicity is something that we really care about. Every center is really trying its best, but it’s hard to do in this type of environment. So, people then get concerned that clinical trials might be even more complex.

I think clinical trials are much less complex in that way, because a lot more of it is covered by the sponsor, whatever that sponsor is, whether that sponsor is the National Institutes of Health, as a grant, or a pharmaceutical company.

But, in general, a clinical trial really should cost the same or less than whatever the standard medical care is; that’s the way they’re built. So, many, many people ask us that question, but I think that is the question that probably is less important than what are the drugs, what does your doc think about this, are you going to lose an opportunity if there’s a different sequence, and does this fit into your life and your schedule, and people who can give you rides.

Katherine Banwell:    

Yeah, right. Are there resources available to assist with the financial impact of a clinical trial?

Dr. Pauline Funchain:    

There are not specific resources for clinical trials; there are specific resources for patients in general, though. There are things like helping with utility bills sometimes, sometimes with rides, I think a lot of clinical trials do pay for things like parking. In general, many trials themselves have extra financial support in them. There was a trial I remember that paid for airfare and lodging, because there were only five centers in the country, and so we had people fly in, and the whole thing was covered.

It depends on the trial. But in terms of outside of trials, there are always patient advocacy groups and things like that, where certain things can get covered. But often, the types of things that get covered by those groups are the same things that get covered with normal medical care.

Katherine Banwell:    

Okay. Before we wrap up the program, Cindi, what advice do you have for patients who may be considering participating in a trial?

Cindi Terwoord:        

Do it. Like I said, I don’t see any downside to it. You want to get better as quickly as possible, and this could help accelerate your recovery. And everything Dr. Funchain mentioned, as far as – I really never brought up any questions about whether it would be covered.

And then somewhere along the line, one of the research people said, “Well, anything the trial research group needs done – like the blood draws – that’s not charged to your insurance.” So, that was nice, that was very encouraging, because I think everybody’s afraid your insurance is going to drop you or something.

And then the first day I was in there for treatment, a social worker came in, and they talked to you. “Do you need financial help? We also have art therapy, music therapy,” so that was very helpful. I mean, she came in and said, “I’m a social worker,” and I’m like, “Oh, okay. I didn’t know somebody was coming in here to talk to me.”

But, yeah, like I said, I’m a big advocate for it, because you hear so many positive outcomes from immunotherapy trials, and boy, I’d say if you’re a candidate, do it.

Katherine Banwell:    

Dr. Funchain, do you have any final thoughts that you’d like to leave the audience with?

Dr. Pauline Funchain:    

First, Cindi, I have to say thank you. I say thank you to every clinical trial participant, everybody who participates in the science. Because honestly, whether you give blood, or you try a new drug, I think people don’t understand how many other lives they touch when they do that.

It’s really incredible. Coming into clinic day in and day out, we get to see – I mean, really, even within a year or two years, there are people that we’ve seen on clinical trial that we’re now treating normally, standardly, insurance is paying for it, it’s all standard of care. And those are even the people we can see, and there are so many people we can’t see in other centers all over the world, and people who will go on after us, right?

So, it’s an amazing – I wouldn’t even consider most of the time that it’s a personal sacrifice. There are a couple more visits and things like that, but it is an incredible gift that people do, in terms of getting trials. And then for some of those trials, people have some amazing results.

And so, just the opportunity to have patients get an outcome that wouldn’t have existed without that trial, like Cindi, is incredible, incredible.

Katherine Banwell:    

Yeah. Dr. Funchain and Cindi, thank you both so much for joining us today.

Cindi Terwoord:        

You’re welcome, thanks for having me.

Dr. Pauline Funchain:    

Thank you.

Katherine Banwell:    

And thank you to all of our collaborators. To access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell, thanks for being with us today.

Are Clinical Trials Safe?

Are Clinical Trials Safe?  from Patient Empowerment Network on Vimeo.

Clinical researcher Dr. Seth Pollack explains the safety protocols in place for clinical trials, including how data is reported and protected. Patient advocate Sujata Dutta goes on to share her experience in a clinical trial.

Dr. Seth Pollack is Medical Director of the Sarcoma Program at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University and is the Steven T. Rosen, MD, Professor of Cancer Biology and associate professor of Medicine in the Division of Hematology and Oncology at the Feinberg School of Medicine. Learn more about Dr. Pollack, here.

Sujuta Dutta is a myeloma survivor and empowered patient advocate, and serves a Patient Empowerment Network (PEN) board member. Learn more about Sujuta, here.

See More from Clinical Trials 101

Related Resources:

Is a Clinical Trial a Last-Resort Option?

A Patient Shares Her Clinical Trial Experience

If I Participate in a Clinical Trial, Will I Be a Guinea Pig?


Transcript:

Katherine Banwell:

Some patients feel that clinical trials aren’t safe, is that the case, Dr. Pollack?  

Dr. Seth Pollack:

No. I mean, we go through, as I was saying before, these clinical trials are extensively vetted. So, the safety is, of course, one of the things that we look most carefully about. But as I was saying before, like with any treatment’s cancer treatments have toxicity, that’s a common problem. So, and when you’re dealing with something brand new sometimes there is a little bit more risk. So, when you’re talking about these very early-stage Phase I trials you probably want to talk to your doctor about what sorts of toxicities you can expect and where they are in the Phase I trial. Are you the first ever to receive this new drug? And if you are nobody’s making you go in the clinical trial, so it can only help to get more information. Right? So, you should ask your team about it, you should find out. 

Most of the time there’s going to be a lot of patients that have been treated already, I mean, they can’t give you definitive data about how things are going but they can maybe say, “Hey. I’ve already treated a few patients on it, and they seem to be doing great.” 

Katherine Banwell:

So, you need to weigh the pros and cons of the trial. 

Dr. Seth Pollack:

You do need to weigh the pros and cons. Now, when you’re talking about these Phase IIs and Phase IIIs, I mean, these are drugs now that have really been vetted for their safety and we have a lot of data about it. And even the Phase Is, it’s not like these things are coming out of nowhere, they’ve been scrutinized, we really expect that they’re going to be safe but we’re doing the trial to prove it. So, it’s a good thing to ask about. 

Katherine Banwell:

Yeah, yeah. 

Sujata Dutta:

Yeah. I would also add that it’s so closely monitored that safety is a top priority, it’s front and center. So, the advantage, I think, with being on a trial is the close monitoring of the patient exactly for this reason. 

If something is amiss it’s going to be picked up as quickly as possible and you’re any issues are going to be addressed as soon as. So, I think, safety does get addressed pretty quickly.  

Katherine Banwell:

Good. 

Can data from trials even be trusted? Dr. Pollack, is that the case? 

Dr. Seth Pollack:

Well, of course, I mean, it can be trusted. Because the thing with the clinical trial data is that you really see the data and there’s all kinds of scrutiny making sure that the data is reported accurately. Now, there’s a whole other conversation we could have as to whether we could interpret the data differently. And sometimes that is an issue that comes up, but the data is reported very accurately. 

So, and there are statistics that are very well understood, and the bar is actually pretty high to say one arm of the trial was better than the other arm of the trial. So, if patients have better survival on one arm, if we say that, usually it means they did considerably better. Enough better that it wasn’t a random chance that one extra patient did better on the treatment arm. No. There were enough patients that did better that the statisticians can go through it with a fine-toothed comb. And they can be absolutely sure up to exactly how many percent sure they can tell you, 0.05 percent or less chance of error that this was a real difference between the study arm and the standard of care arm. 

Sujata Dutta:

I think you mentioned too that one is trust, and one is data. So, Dr. Pollack mentioned a lot about the data, I think the trust is also a very important thing. I like to go with positive intent because I do not have a reason to believe my doctor has some ulterior motive to suggest a clinical trial. And so, I trust them wholeheartedly. The first hurdle is you have to trust the system or what is being proposed to you because, as Dr. Pollack said, it’s gone through a lot of vetting. A recommendation to be part of a trial itself is vetted by your doctor when they make the recommendation. So, have faith, trust, that they are making a good recommendation. And then, of course, the data, I don’t know much about that, but as I said, I trust it. So, I would trust the data too. 

Katherine Banwell:

Of course. Of course. Some patients feel like they’re going to lose their privacy. Sujata, did you feel that at all? 

Sujata Dutta:

No. Not at all. 

I mean, with everything else that is also taken care of, my information, or whatever, is not made available to anybody. And so, obviously there’s a lot of people will get those, and I had a huge pile of paperwork to go through, but I think that’s a good thing. For my peace of mind that I knew that my information was not going to be shared outside of the study, the trial, etc., and things. So, no, I don’t think that’s a problem. 

Katherine Banwell:

Beyond these misconceptions is there anything else you hear? Dr. Pollack?  

Dr. Seth Pollack:

No. I mean, look, in our crazy modern world there’s concerns everywhere, but the clinical trial is very, very careful. Whenever possible we use the medical chart.  

And then, we have a very stringently protected database that’s storing people’s information, but it’s deidentified. So, I mean, we have a separate key to figure out who the patients are and then we try to limit the use of the patient’s name or any identifying information about them beyond that. So, and your information is not shared. For example, if there’s a drug company involved in the trial, your information is not shared with the drug company, you have a new identifier that is unique and not traceable back to you that is provided to whoever, if there’s outside groups working on the trial with you. So, your information is very carefully protected, and everyone is very conscious about issues regarding privacy.  

Katherine Banwell:

That’s great to know.  

Could a Clinical Trial Be Your Best Cancer Treatment Option?

Could a Clinical Trial Be Your Best Cancer Treatment Option? from Patient Empowerment Network on Vimeo.

Is a clinical trial right for you? Cancer expert and researcher Dr. Seth Pollack is joined by PEN board member and empowered patient, Sujata Dutta, to discuss key information about clinical trials. The guests review clinical trial terminology, debunk common misconceptions about trials, and Sujuta shares her own story of participation in a clinical trial.

Dr. Seth Pollack is Medical Director of the Sarcoma Program at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University and is the Steven T. Rosen, MD, Professor of Cancer Biology and associate professor of Medicine in the Division of Hematology and Oncology at the Feinberg School of Medicine. Learn more about Dr. Pollack, here.

Sujuta Dutta is a myeloma survivor and empowered patient advocate, and serves a Patient Empowerment Network (PEN) board member. Learn more about Sujuta, here.

Download Guide

See More from Clinical Trials 101

Related Resources:

Could a Clinical Trial Be Your Best Cancer Treatment Option? Resource Guide

Understanding Clinical Trial Phases

How Could Clinical Trials Fit Into Your Myeloma Treatment Plan?


Transcript:

Katherine Banwell:    

Hello, and welcome. I’m Katherine Banwell, your host for today’s program.

Today we’re going to discuss clinical trials, what they are and how they work, and debunk some misconceptions along the way. Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you. All right. Let’s meet our guests today. Joining me is Dr. Seth Pollack. Dr. Pollack, welcome. Would you please introduce yourself?

Dr. Seth Pollack:

Yeah. Thanks so much. It’s a pleasure to be here, my name is Seth Pollack. I’m a medical oncologist here at Northwestern University Medical Center.

And I specialize in treating patients with cancer, and I have a specific interest in patients with a type of cancer called sarcomas.

Katherine Banwell:    

Excellent. Thank you for taking the time to join us today. And here to share the patient perspective is Sujata Dutta, who is on the board of the Patient Empowerment Network and is currently participating in a clinical trial. Sujata, it’s a pleasure to have you with us.

Sujata Dutta:

Pleasure to be here Katherine. Hello, Dr. Pollack. And hi everyone, my name is Sujata Dutta, and I was diagnosed with a cancer called multiple myeloma in December of 2019. And I’ve been on a clinical trial since September of 2020.

Katherine Banwell:    

Thank you, for that information. And we’re going to go into that further in just a few moments. Let’s start with a basic question, Dr. Pollack. What is a clinical trial?

Dr. Seth Pollack:       

Yeah. It’s a basic question, but actually, sometimes, it can be harder to answer than you might think.

I think everybody has an idea in their mind about what a clinical trial is, you’re going to test a new approach. But actually, there’s a whole variety of different things that can be a clinical trial, right? A clinical trial a lot of the times is testing a new drug, could be testing something for the very first time, could be testing something in combination with other drugs for the very first time. It could be testing a standard approach but doing it in a new way. It could even be giving less treatments than we usually do. For example, if there’s a very intense, harsh, standard of care treatment we might even have a clinical trial where we try a little bit less and see that patients do just as well. So, all of those things are clinical trials, but really the clinical trial in its heart is a very organized and careful approach to testing a new treatment strategy for patients.

Katherine Banwell:    

Okay. What are the phases of a clinical trial?

Dr. Seth Pollack:       

Yup. So, the Phase I clinical trial is usually when we’re testing something for the first time in however we’re doing it. So, it could be the first time we’re testing a new drug, or the first time we’re testing a drug in combination with other drugs. And the real thing about a Phase I trial is that the main goal of the trial is to look at the safety and tolerability of the regiment. That doesn’t mean that we’re not really trying to figure out if the regiment works, I mean, that’s also one of the most important things. But the most important thing for a Phase I trial is making sure that it’s safe and tolerable. A Phase II trial is where we, sort of, shift and we’re still making sure, and double checking, that the drug is, but now our main focus becomes on the efficacy of the strategy.

So, now we’re trying to really figure out if this is a strategy that seems affective enough to go to a Phase III. And a Phase III is a big multi-center trial. Frequently those will be placebo controlled where a lot of the times there’ll be randomized trial where we really try to absolutely prove, beyond a shadow of a doubt, that, that strategy is affective. And those are most of the types of trials that patients will encounter.

Katherine Banwell:    

Okay. Thank you for providing clarity around the phases. Before we move onto safety and benefits of clinical trials, let’s hear from Sujata. Sujata, I understand that you went through a series of treatments for your multiple myeloma, which is a type of blood cancer, including a stem cell transplant.

At what point did you and your doctor consider a clinical trial might be best for you?

Sujata Dutta:  

Yes, you’re right. I was diagnosed with multiple myeloma in December, and so the line of treatment or the standard protocol is that you go through what is called an induction therapy. Which is like a few cycles of chemotherapy which get you ready for a transplant. And the transplant, the hope is that it kind of washes away, or cleans off all the cancer cells for you, or at least brings the cancer to a very, very minimal level. And I did go through six rounds of chemo which got me ready for the transplant, and I went through the transplant in June of 2020. However, I’m amongst the very few, small percentage of people that just did not respond with the transplant. So, I was at the same point as where I started. So, it was a little bit disappointing, but my doctors were there to help me understand the situation. It was a hard pill to swallow.

But anyways, there were options. And that’s what I feel very hopeful about with multiple myeloma is that there are so many options available today through treat, or to at least bring the disease under control to a very large extent. And I expressed a desire to be in a trial very earlier on, so my doctor did know that I would lend a year or two listening to what the trials were. And it just so happened that there was a trial that was very apt in my situation, somebody who had gone through a transplant. They have some criteria, and I was able to meet that criteria. And so, for me, it seemed to be the right decision to make. And so, that’s how I agreed to be part of the trial.

Katherine Banwell:    

Can you go into some detail about why you thought a clinical trial was a best thing for you?

Sujata Dutta:  

Yeah. So, initially before knowing much about the strain that I’m a part of, I just had the desire to be part of a trial because I was always in awe of patients who had been in trials before me.

And because of whom I was benefiting. But whatever regiments, medications, combos, whatever was happening. And so, from that perspective I always wanted to give back in some way. Unfortunately, we are having more people being diagnosed with cancers, with multiple myeloma, and so I was very motivated to do something for the community that I was now part of. And so, I had my transplant at Mayo, and I knew that they had a whole bunch of trials and had access to different types of trials. So, that was my first motivation and it just so happened that, as I said, my experience with transplant didn’t go the desired way. And so, when I heard that there was a possibility that I could be part of a trial, I kind of leaned into actually agreeing to be part of that.

Katherine Banwell:    

Yeah. It sounds like that was the next step for you.

Sujata Dutta:  

Yup.

Katherine Banwell:    

Yeah. Well, I’d like to address a list of common concerns about clinical trials that we’ve heard from various audience members prior to this program.

And this is probably the most common; I will be a guinea pig. Dr. Pollack, how do you respond to that?

Dr. Seth Pollack:       

Yeah. I know that is a common concern. I mean, I think the thing that people have to understand about clinical trials is there is just so much oversight that happens for these clinical trials. Every document, every procedure, is scrutinized by multiple committees. There’s a scientific review committee, there’s a review board, IRB, that reviews these. Many of these trials are reviewed by the FDA and they’re reviewed by your doctor and your doctor’s colleagues that are also participating in the trial. So, every detail is discussed at length.

In fact, a lot of the times there’s a lot more structure to being on the clinical trial than just routine clinical care because they’ve thought so thoroughly about when everything needs to be done and what the right timing of is for the various procedures.

Katherine Banwell:    

Right. And another concern that people have is; clinical trials are my last resort treatment option. What do you say to that Dr. Pollack?

Dr. Seth Pollack:       

Yeah, no. That’s a common misconception. So, we like to have clinical trials for every phase of the patient’s cancer journey because we’re trying to make every single part of the cancer journey better. So, I think a lot of people think that, okay, when they hit their last resort that’s kind of the time to try something new. Even in the very earliest parts of the cancer journey, even in the diagnosis phase sometimes we’ll have clinical trials where we’ve tried different images, modalities, or look at things in a different way in terms of the biopsies.

But then, in terms of the cured-of treatments, when somebody is in the cured-of setting we don’t usually try something very brand new. But a lot of the times we’ll try something that is very affective for patients at the end, and we want to try and make the cured-of strategy even better. So, a lot of the times for those patients we’ll have new therapies that are very safe and established that we’re trying to incorporate earlier into patients’ treatments because we know they work really well, right? And then, even in patients who have incurable cancer a lot of times it’s better to try a clinical trial earlier on just because sometimes the clinical trials have the most exciting new therapies that are bringing people a lot of hope.

And a lot of the times you want to try that when you’re really fit and when you’re in good shape. So, that’s why I think that you really want to think about doing a clinical trial when the opportunity arises.

Katherine Banwell:    

Yeah. Beause it could be beneficial to you and it’s certainly going to be beneficial to other people. Is this fact or fiction; it will be expensive? Dr. Pollack?

Dr. Seth Pollack:       

That’s fiction because the way the clinical trials work is we go through everything very carefully to figure out what things are standard and what things are unique to the clinical trials. So, if you are getting chemotherapy, you’re going to need blood work, you’re going to need the chemotherapy drugs, you’re going to need some sort of imaging, CT scan, or whatever your doctor would do.

And all those sorts of things are considered standard, so your insurance company is built for those. Then there’s a bunch of things that are considered research. For example, there’s special research bloodwork, maybe there’s an investigational agent that’s being added to standard chemotherapy. Those things are billed to the study, so you don’t actually have to pay anything extra, it’s just like you’re getting the normal treatment as far as you’re concerned. I mean, that’s the way it always is, and I haven’t had any of my patients ever get into real problems in terms of the finances of these things. It always works very straight forward like standard therapy.

Katherine Banwell:    

Okay. That’s good to know. The logistics will be a nightmare and I don’t live close to a research hospital. Sujata, did you have that issue?

Sujata Dutta:  

Yeah. That’s a very interesting one, and actually I’ll share my experience. I did have this concern about logistics, because I got my transplant at Mayo Rochester, which is a two-hour drive from where I live. And so, when I got to know about it literally me and my husband were like, “Oh, my gosh. What are we going to do?” It’s not just me, my husband is my caregiver, he has to take the day off to drive me to Mayo, wait through my treatment, and drive me back. Then we have boys who were distance learning at the time, and so what do we do with them? Do we drop off a friends or take a favor from a friend? And so on and so forth.

So, the logistics was an issue and we literally said, “Thanks but no thanks” and we walked out of the room. And we came downstairs, and my husband was like, “What the heck?” My team understands everything, and I fortunately work for a very good employer, and they understand everything, people first. And so, he was like, “I can figure this out. Let’s do it if this is what’s going to help you, then let’s just figure this out.” And at that time, it was so good, and I have total respect for Dr. Pollack.

You and everybody in this medical community. My doctor who leads the trial at Mayo, she actually said, “Why don’t you check with your local cancer center? Maybe they are also approved by FDA, and they may be able to administer this treatment to you.” Unfortunately, at that time they weren’t but we were like, “We’re going to go ahead with the trial. It doesn’t matter.” My husband was like, “I’ll take the day off, you don’t worry about it.” And then, four months later my institute did get approved by FDA, and so I was able to transfer from Mayo to my local cancer center, Abramson Cancer Center, which is 20 minutes from home. And so, there are options, I know that it can be an issue and it can be overwhelming at the time which was the case with me. But I was able to overcome that, so maybe there are options available that the patients can consider.

Katherine Banwell:    

Yeah. Dr. Pollack, do you have anything to add?

Dr. Seth Pollack:       

No. I think the logistics and the location are real concerns with clinical trials.

Clinical trials do sometimes require you to have an extra visit, sometimes they’re a little bit less flexible in terms of when you can get your medication. If you’re getting a standard treatment your doctor may say, “It’s probably okay for you to wait an extra week.” Whereas sometimes on a clinical trial, not always, but sometimes they could be a little bit more strict about when you’re supposed to get certain things. And likewise, with the travel for some people that can be an issue. I mean, the clinical trial is not available everywhere. I mean, Sujata was very lucky that she was able to do the clinical trial she was doing close to home, but that doesn’t always happen. So, I think that’s an important thing to talk to your clinical team about.

Katherine Banwell:    

Yeah. Some patients feel that clinical trials aren’t safe, is that the case, Dr. Pollack?

Dr. Seth Pollack:       

No. I mean, we go through, as I was saying before, these clinical trials are extensively vetted. So, the safety is, of course, one of the things that we look most carefully about. But as I was saying before, like with any treatment’s cancer treatments have toxicity, that’s a common problem. So, and when you’re dealing with something brand new sometimes there is a little bit more risk. So, when you’re talking about these very early-stage Phase I trials you probably want to talk to your doctor about what sorts of toxicities you can expect and where they are in the Phase I trial. Are you the first ever to receive this new drug? And if you are nobody’s making you go in the clinical trial, so it can only help to get more information. Right? So, you should ask your team about it, you should find out.

Most of the time there’s going to be a lot of patients that have been treated already, I mean, they can’t give you definitive data about how things are going but they can maybe say, “Hey. I’ve already treated a few patients on it, and they seem to be doing great.”

Katherine Banwell:    

So, you need to weigh the pros and cons of the trial.

Dr. Seth Pollack:       

You do need to weigh the pros and cons. Now, when you’re talking about these Phase IIs and Phase IIIs, I mean, these are drugs now that have really been vetted for their safety and we have a lot of data about it. And even the Phase Is, it’s not like these things are coming out of nowhere, they’ve been scrutinized, we really expect that they’re going to be safe but we’re doing the trial to prove it. So, it’s a good thing to ask about.

Katherine Banwell:    

Yeah, yeah.

Sujata Dutta:  

Yeah. I would also add that it’s so closely monitored that safety is a top priority, it’s front and center. So, the advantage, I think, with being on a trial is the close monitoring of the patient exactly for this reason.

If something is amiss it’s going to be picked up as quickly as possible and you’re any issues are going to be addressed as soon as. So, I think, safety does get addressed pretty quickly.

Katherine Banwell:    

Good, good. Okay. That’s good to know. Another concern is; I’ll get a placebo. Dr. Pollack, what is a placebo first of all? And is that true in a clinical trial setting?

Dr. Seth Pollack:       

So, there are clinical trials with placebos, it’s a real thing. And what a placebo is, it’s a pill and it’s made to look just like the real pill, but it doesn’t have any active drug in it. Sometimes people say it’s a sugar pill, but it may or may not be sugar, but it’ll probably be something without a taste. But it’s an inert substance that is not going to affect you at all.

And your doctor won’t know whether you’re getting a placebo or not, so a lot of the times they’ll call these things double-blind because your doctor doesn’t know, your pharmacist doesn’t know. And to unblind you they have to go through special procedures to find out whether you’re on the studied drug or not.

Katherine Banwell:    

Would a placebo be given solely? Or would it be given in addition to this new drug that’s being tested?

Dr. Seth Pollack:       

Yeah. So, it’s unusual for a placebo to be given solely. Usually there’ll be a clinical trial where you’re getting the standard treatments plus the new drug or standard treatment plus the placebo, so no matter what you’re getting the standard treatments. There are still some trials where, and these are usually for patients with very advanced cancer, who there’s not really any treatment options that are good. Where they will randomize people to just be on the standard drug versus the placebo.

Sometimes what they’ll do is if they want to do a trial that’s the standard drug versus a placebo, they’ll do the imaging very frequently and they’ll have a crossover. So, a crossover means that everybody gets to be on the new drug, but some people will have to go on the placebo first. So, and then they watch you very closely. So, if you get randomized to go on the placebo and your cancer starts to grow, they figure it out very quickly and then they give you the opportunity to go on the new drug.

Katherine Banwell:    

I see, okay. I’ll be stuck in the trial forever and I can’t change my mind. Sujata, did that happen to you?

Sujata Dutta:  

No. I mean, when I finally agreed and signed the dotted line it was made very clear to me that it was voluntary, I was volunteering to be part of the trial and I could get out of the trial at any point of time. So, in my case I’m in Phase III of a trial, the first commitment was for two years and then the next was five years.

So, again, it sounds daunting to me right now, two years is coming to an end in July of this year. I’m like, “Wow! Two years are over already?” And then five years, I’m not thinking about that, but again, it was at any point I could just say that I’ve had enough, or whatever be the reason, I could get out of the trial. So, no. Yes. There’s an option.

Katherine Banwell:    

Can data from trials even be trusted? Dr. Pollack, is that the case?

Dr. Seth Pollack:       

Well, of course, I mean, it can be trusted. Because the thing with the clinical trial data is that you really see the data and there’s all kinds of scrutiny making sure that the data is reported accurately. Now, there’s a whole other conversation we could have as to whether we could interpret the data differently. And sometimes that is an issue that comes up, but the data is reported very accurately.

So, and there are statistics that are very well understood, and the bar is actually pretty high to say one arm of the trial was better than the other arm of the trial. So, if patients have better survival on one arm, if we say that, usually it means they did considerably better. Enough better that it wasn’t a random chance that one extra patient did better on the treatment arm. No. There were enough patients that did better that the statisticians can go through it with a fine-toothed comb. And they can be absolutely sure up to exactly how many percent sure they can tell you, 0.05 percent or less chance of error that this was a real difference between the study arm and the standard of care arm.

Sujata Dutta:  

I think you mentioned too that one is trust, and one is data. So, Dr. Pollack mentioned a lot about the data, I think the trust is also a very important thing. I like to go with positive intent because I do not have a reason to believe my doctor has some ulterior motive to suggest a clinical trial. And so, I trust them wholeheartedly. The first hurdle is you have to trust the system or what is being proposed to you because, as Dr. Pollack said, it’s gone through a lot of vetting. A recommendation to be part of a trial itself is vetted by your doctor when they make the recommendation. So, have faith, trust, that they are making a good recommendation. And then, of course, the data, I don’t know much about that, but as I said, I trust it. So, I would trust the data too.

Katherine Banwell:    

Of course. Of course. Some patients feel like they’re going to lose their privacy. Sujata, did you feel that at all?

Sujata Dutta:  

No. Not at all.

I mean, with everything else that is also taken care of, my information, or whatever, is not made available to anybody. And so, obviously there’s a lot of people will get those, and I had a huge pile of paperwork to go through, but I think that’s a good thing. For my peace of mind that I knew that my information was not going to be shared outside of the study, the trial, etc., and things. So, no, I don’t think that’s a problem.

Katherine Banwell:    

Beyond these misconceptions is there anything else you hear? Dr. Pollack?

Dr. Seth Pollack:       

Well, I hear a lot of people really interested in clinical trials. I mean especially, I treat some patients with rare cancers or with unusual presentations and I think people are very excited to be a part of something that could be new, that could be the next wave. A lot of times the clinical trials have new things with the most exciting science that could be the future of treatment.

So, I think a lot of people are excited about clinical trials. And I also hear some of the reservations that you’re expressing. I think usually when patients ask their questions are very straightforward and easy to address so that people can make their own decisions.

Katherine Banwell:    

Dr. Pollack, I’d like to go back to you and ask you the same question about privacy. Do patients need to be worried about that?

Dr. Seth Pollack:       

No. I mean, look, in our crazy modern world there’s concerns everywhere, but the clinical trial is very, very careful. Whenever possible we use the medical chart.

And then, we have a very stringently protected database that’s storing people’s information, but it’s deidentified. So, I mean, we have a separate key to figure out who the patients are and then we try to limit the use of the patient’s name or any identifying information about them beyond that. So, and your information is not shared. For example, if there’s a drug company involved in the trial, your information is not shared with the drug company, you have a new identifier that is unique and not traceable back to you that is provided to whoever, if there’s outside groups working on the trial with you. So, your information is very carefully protected, and everyone is very conscious about issues regarding privacy.

Katherine Banwell:    

That’s great to know. Sujata, there’s clearly a lot of hesitation and misconceptions out there. What would you say to someone who’s considering a trial but is hesitant?

Sujata Dutta:  

I would say speak to your provider, speak to your doctor, and get all these myths kind of busted to say, “it’s going to be expensive” or whatever those questions are. And then, through that process also try and understand what is it that the study is trying to achieve? How is that going to be beneficial to you? So, in my instance, it wasn’t the last line of defense, it was just one of the processes or combos that would help me. And so, that was important for me to understand and then a little bit of education as well. So, I was asking, I have questions on my phone every time I meet my provider, and I did the same thing. So, I think that one of the good practices is keep your note of your questions and have those questions ready. And no question is silly, all questions are important. So, ask as many questions as you can and use that opportunity to educate yourself about it.

And maybe you realize, “No. I don’t think it’s working for me” or “I don’t think this trial is good for me.” But it’s good, important, to have that conversation with your provider, that’s what I would recommend highly.

Katherine Banwell:    

Excellent. Thank you, Dr. Pollack, if someone is interested in participating, how can they find out about what trials are even available for them?

Dr. Seth Pollack:       

Yeah. I mean, the best thing to do is to start just by asking your doctor if they know about any clinical trials. And a lot of the times the clinical trials are run at the big medical centers that may be closer to you, so you could ask your doctor if there’s any clinical trials at the big medical center even. Or I always think it’s good to get a second opinion, you could go get a second opinion at the big medical center that’s close to you and ask them what clinical trials are at your center.

And sometimes they’ll be conscious about some of the clinical trials that may be even run around the country. And you can ask about that as well.

Katherine Banwell:    

Would specialists have more information about clinical trials than say a general practitioner?

Dr. Seth Pollack:       

So, I specialize in rare cancers, so a lot of the times the general practitioners they’ve got my cell phone number, and they text me, and they say, “Hey, do you have a clinical trial going on right now?” And that happens all the time, but yeah, the specialists will usually because frankly there’s so much to know. And the general practitioners really have a lot to keep track of with all the different types of diseases that are out there. Whereas at the big centers, the specialists, part of their job is really to keep their tabs on what’s going on with the clinical trials.

So, they’re good people to ask, either your local doctor could reach out to them, or you could go get a second opinion and ask.

Sujata Dutta:  

There’s also a lot of information, Katherine, on sites such as LLS, or PEN, or American Cancer Society that they also publish a lot of information. Of course, I would recommend once you have that information then vet it by your specialist, or whatever. But if you’re interested in knowing more about clinical trials in general and some that would work for you, then those are also some places to get information from.

Katherine Banwell:    

That’s great information. Thank you, I was going to ask you about that Sujata. Well, before we end the program, Dr. Pollack, I’d like to get your final thoughts. What message do you want to leave the audience with related to clinical trial participation?

Dr. Seth Pollack:       

Yeah. I think clinical trials it can be a very rewarding thing for a lot of patients to do, I think patients really like learning about the new treatments. And I think a lot of patients really like being a part of pushing the therapies forward in addition to feeling like sometimes they’re getting a little bit of an extra layer of scrutiny, because there’s a whole extra team of research coordinators that are going through everything.

And getting access to something that isn’t available yet to the general population. So, I think there’s a whole host of advantages of going on clinical trials, but you need to figure out whether or not a clinical trial is right for you.

Katherine Banwell:    

Yeah. Sujata, what would you like to add?

Sujata Dutta:  

Absolutely, I second everything that Dr. Pollack is saying. And in my personal experience I wouldn’t say everything is hunky-dory, everything is fine. I’m going through treatment, I have chemo every four weeks, I started with chemo every week. That’s when the logistics pace was really difficult because going to Mayo every week was not easy. But anyways, as the trial progress itself every four weeks, but as I said the benefits are huge because I have labs every four weeks. I meet my provider every four weeks.

So, we go through the labs and anything amiss, I’ve had some changes to my dosage because I’ve had some changes in the labs. And so, there’s a lot of scrutiny which I like, but the flip side, for maybe some maybe like, “I have to have chemo every four weeks. Do I want to do that or not?” Or whatever. In my case, I knew it, and I signed up for it, and I’m committed to doing that for two years. And so, I’m fine with that. So, I would say all in all, I’d see more benefits of being in a clinical trial. One, you’re motivated to give back to the community. Two, you are being monitored and so your health is important to your provider just as it is to you. And so, I highly recommend being part of a trial if it works for you and if you’re eligible for one.

Katherine Banwell:    

Yeah. Sujata Dutta, and Dr. Pollack, thank you both for taking the time to join us today.

Sujata Dutta:   

Thank you.

Dr. Seth Pollack:       

Thank you.

Katherine Banwell:    

And thank you to all of our partners. To access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell, thanks for joining us.  

Understanding Waldenström Macroglobulinemia and How It Progresses

Understanding Waldenström Macroglobulinemia and How It Progresses from Patient Empowerment Network on Vimeo.

Dr. Jorge Castillo of Dana-Farber Cancer Institute provides an overview of Waldenström macroglobulinemia (WM) and how the condition presents and progresses.

Dr. Jorge Castillo is Clinical Director at the Bing Center for Waldenström Macroglobulinemia Dana-Farber Cancer Institute and Assistant Professor of Medicine at Harvard Medical School. Learn more about Dr. Castillo, here.

See More From The Pro-Active Waldenström Macroglobulinemia Patient Toolkit

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Current Waldenström Macroglobulinemia Treatment Approaches


Transcript:

Katherine:                  

Let’s start with the very basic. What is Waldenstrom macroglobulinemia?

Dr. Castillo:               

Yeah, Waldenstrom’s macro – it’s a mouthful.

Katherine:                  

It is.

Dr. Castillo:               

I can just call it WM for ease.

It is a blood cancer, and in this blood cancer, the malignant cells are nesting in the bone marrow. And not only that. These malignant cells kind of secrete, produce, a protein called IgM.

IgM is an antibody that should be protecting us from infections, and in a normal state, we all have a little bit of IgM, and that’s a good thing. But in these patients, with these malignant cells, as these cells accumulate in the marrow, they actually increase the levels of IgM in our patients, and that can translate into a number of different symptoms, which we will probably talk about later.

Katherine:                  

Yes. How is it staged?

Dr. Castillo:               

So, the staging is a very interesting aspect. So, when we think about cancer, we think about stage I is in one spot, stage II in another spot, stage III, right, and it gets more extensive as we go along. That doesn’t really apply to Waldenstrom’s. Waldenstrom’s is a whole-body disease right from the start. The main reason for that is because it’s a disease of the bone marrow, and we all have bone marrow in all our bones, from our skull all the way to the great toe, so if you were to get a sample from each bone space, we would find the malignant cells there. So, this is a disease that is a whole-body disease right from the start, so therefore, there’s no stage I, II, or III. That is just the way we envision this.

Katherine:                  

How does the condition progress?

Dr. Castillo:               

So, it’s interesting because a number of the patients that we see in my clinic are actually asymptomatic at the time of the presentation. I would say maybe about a third of the patients I see in my clinic that were diagnosed with this disease for other reasons. They either had an abnormal laboratory value or an abnormal imaging study or some other reason. And when they come, they are worked up. Initially, they are found to have these malignant cells and these IgM elevation, but they have no other problems whatsoever.

So, I would say most patients will be asymptomatic at the beginning of the disease, and probably they will be asymptomatic for years before the symptoms actually do start. So, what happens is the malignant cells start taking over the bone marrow space, and it reaches a point in which the bone marrow, the healthy bone marrow, doesn’t have space to produce the normal cells that they should produce.

So, the first things that we tend to see in these patients is anemia, so the hemoglobin level starts dropping.

The red cells are the first ones that are being affected by this process so that the anemia is being seen first. If we leave that for a long time, then the other blood cells will decrease also, the white blood cells and the platelets over time. But the first one is almost always the anemia. And obviously, that, patients feel tired. They feel short of breath. They feel fatigued and all of that.

Now, the IgM itself can cause other problems on their own. If they have there’s too much IgM, they can actually make the blood a little thick, and that can cause a little bit of problems with the circulation, specifically in the eyes, for example. Some patients have blurred vision. Some patients have nosebleeds or headaches, right, with all that hyperviscosity, which means the blood is too thick. In some other patients, we have nerve damage. You know, they can have numbness in their toes, and then that increases into the – progresses, extends into the feet, into the shins, into the knees and then the fingers.

And so, that happens over years sometimes. Some patients can have enlargement of lymph nodes in their necks and in the axillary areas or in the inguinal areas, or even enlargement of organs, the spleen and liver and things like that. So, when we think about the clinical manifestations of Waldenstrom’s, it varies, very diverse. But I would say most patients would have anemia. I think that’s probably the most important aspect of it.

How Does Essential Testing Affect Myeloma Care and Treatment?

How Does Essential Testing Affect Myeloma Care and Treatment? from Patient Empowerment Network on Vimeo.

 Why is it important to ask about essential testing for your myeloma? Find out how test results could reveal more about your myeloma and may help determine the most effective care for your individual disease.

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What Should You Ask Your Doctor About Myeloma Testing?


Transcript:

Why should you ask your doctor about essential myeloma testing?

When a patient is diagnosed with myeloma, they typically undergo a series of tests that aid in diagnosing and staging their individual disease. The standard tests include:

  • Blood Test
  • Urine Test
  • Bone Marrow Biopsy, and
  • Imaging

As research in the field evolves, genetic profiling via more in-depth cytogenetic testing is increasingly common to further classify your myeloma. This testing often identifies unique biomarkers of the myeloma, such as translocations or changes in chromosomes.

So why do the results of these tests matter?

  • The presence of certain biomarkers can indicate a patient is low-risk, which can suggest a more positive prognosis.
  • There are certain biomarkers that indicate high-risk myeloma, meaning an aggressive treatment approach may be more effective.

Knowing your risk in myeloma is useful to your healthcare team when choosing a treatment approach or may help in determining if a clinical trial might be right for you.

How can you Insist on the best care for YOUR myeloma?

  • First, always speak up and ask questions. Remember, you have a voice in YOUR myeloma care. Your doctor is expecting you to ask questions and should be able to answer them.
  • Ask your doctor if you have had or will receive genetic testing for risk stratification and how the results may impact your care and treatment plan. Be sure to ask for paper or electronic copies of your important test results.
  • And finally, bring a friend or a loved one to your appointments to help you process information and to take notes.

To learn more about your myeloma and access tools for self-advocacy, visit powerfulpatients.org/myeloma 

How Are Cytogenetics Used in Myeloma Care?

How Are Cytogenetics Used in Myeloma Care? from Patient Empowerment Network on Vimeo.

Myeloma expert, Dr. Nina Shah, explains cytogenetics and how results of these tests affect care and treatment for myeloma patients.

Dr. Nina Shah is Associate Professor of Medicine in the Fepartment of Medicine at the University of California San Francisco (UCSF) and treats patients at the Hematology and Blood and Marrow Transplant Clinic at UCSF Helen Diller Family Comprehensive Cancer Center. Learn more about Dr. Shah, here.

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What Standard Testing Follows a Myeloma Diagnosis_ (1)

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Transcript:

Katherine Banwell:

What is cytogenetics, and how is it used in myeloma patient care?

Dr. Shah:

We use the term cytogenetics and FISH sort of interchangeably, and really what it is, is the DNA characteristics of the bad plasma cells. So, the myeloma cells, and a lot of them may have changes in their DNA that are what we call clonal, meaning that they’re in a significant percentage of those cancer cells, or they might be non-clonal, which are less significant. But it’s the way the DNA is put together or maybe cut and pasted so that it changes the characteristics and maybe the aggressiveness of the disease.

Katherine Banwell:

What is the goal of this in-depth testing? Are there specific markers you’re looking for?

Dr. Shah:

When we look for things like cytogenetics and send FISH testing, we look to see if patients have changes that might make their disease may be more aggressive.

For example, it may cause their plasma cells, the myeloma cells, to grow faster or more aggressively. So, we look for changes that might, for example, have a deletion of a certain chain that puts the brakes on tumors, or it may have a translocation, which is when the chains sort of do-si-do together and that makes the cells grow faster. 

Understanding Your Role in Myeloma Treatment Decisions

Understanding Your Role in Myeloma Treatment Decisions from Patient Empowerment Network on Vimeo.

Many factors are considered when choosing a myeloma treatment. Dr. Nina Shah, a myeloma expert, reviews how treatment decisions are made and the patient’s role in deciding on an approach.

Dr. Nina Shah is Associate Professor of Medicine in the Department of Medicine at the University of California San Francisco (UCSF) and treats patients at the Hematology and Blood and Marrow Transplant Clinic at UCSF Helen Diller Family Comprehensive Cancer Center. Learn more about Dr. Shah, here.

See More From Engage Myeloma


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An Expert Reflects on Hopeful Advances in Myeloma Treatment 


Transcript:

Katherine Banwell:

What are the main factors that you take into consideration before a treatment approach is decided on?

Dr. Shah:

We always have to remember that treating a patient is also treating a person. So, it’s not just about what the disease the patient has but who the patient is. And so, we take into consideration goals that the patient as well as other health factors that may take – be taken into consideration. For example, the patient may have high blood pressure or a heart condition. But regarding the disease, we really also take into consideration what the profile of the disease is, maybe how much disease burden the patient has and some genetic factors that may impact our decision-making.

Katherine Banwell:

What is the patient’s role in treatment decisions?

Dr. Shah:

The patient should always be the center of the decision-making. I think that’s a really important thing for us to remember because ultimately, it’s the patient who has to make the decision and has to withstand the treatment. Alongside of that there may be some caregivers as well, but the patient has to, 1.) understand the disease, and 2.) understand the treatment options. So, it’s best if the patient has as much information as possible.

Katherine Banwell:

Are treatment considerations different for patients with relapsed disease?

Dr. Shah:

For patients with relapsed disease, there’s a lot of things to consider that may not have been true when the patient was first diagnosed. For example, you always have to think of what maybe the patient had as a prior – excuse me, as a prior treatment, and also how the patient tolerated it. 

What You Should Know About Myeloma Clinical Trial Participation

What You Should Know About Myeloma Clinical Trial Participation from Patient Empowerment Network on Vimeo.

Myeloma expert Dr. Nina Shah shares her view on why patients should consider a myeloma clinical trial and provides advice for finding and participating in a trial.

Dr. Nina Shah is Associate Professor of Medicine in the Department of Medicine at the University of California San Francisco (UCSF) and treats patients at the Hematology and Blood and Marrow Transplant Clinic at UCSF Helen Diller Family Comprehensive Cancer Center. Learn more about Dr. Shah, here.

See More From Engage Myeloma


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An Expert Reflects on Hopeful Advances in Myeloma Treatment 


Transcript:

Katherine Banwell:

Why should a patient consider participating in a clinical trial?

Dr. Shah:

I am a huge fan of clinical trials, as you probably figured out. And the reason for that is that it’s the only way we know how to do things. And for everything we figured out about myeloma, it’s because patients participated beforehand in clinical trials. So, of course, it’s a way to pay it forward. But aside from that, there’s an experience that a patient can have on a clinical trial that is really unlike other experiences that patients may have. For example, they will be given the opportunity to understand a lot about their disease that maybe they may not have understood before, and they may have the opportunity to try a treatment that might be beneficial.

There are no guarantees in a clinical trial, and that informed consent procedure where the doctor tells you about the risks, benefits, and alternatives, should be very comprehensive and clear. But it does allow for patients to get access to something they may not have had before. And I think one of the other things that’s important is that it’s sort of a concierge service, I would say, with clinical trials, because you have to be monitored very closely. So, of course, all your symptoms have to be known. And you get a little bit more time, I would say, when you participate in a clinical trial because we really want to know the pluses and minuses of these treatments.

Katherine Banwell:

How can patients participate in research? Where do they start?

Dr. Shah:

Participating in research is a great opportunity for patients and something that we’re grateful for as myeloma physicians. There are many ways to look on various websites. There are things like SparkCures. There’s ClinicalTrials.gov. You can look at any academic website. Almost all advocacy groups also have opportunities for you to look at clinical trials.

And any time you get the opportunity to look at patient education sites, they may have a link for you to look for other clinical trials that might be relevant to your particular stage in disease or the particular kind of myeloma that you have. When in doubt, please, if you have a chance, talk to your local oncologist perhaps to maybe refer you to a myeloma specialist. We can do this by Zoom now, so there should be no reason that we can’t be a part of your care team at least for a consultation. 

Which Tests Do You Need Following an MPN Diagnosis?

Which Tests Do You Need Following an MPN Diagnosis? from Patient Empowerment Network on Vimeo.

After a diagnosis of essential thrombocythemia (ET), polycythemia vera (PV), or myelofibrosis (MF), what testing should take place? Dr. John Mascarenhas shares an overview of essential and in-depth testing for patients with myeloproliferative neoplasms (MPNs).

Dr. John Mascarenhas is Associate Professor of Medicine at the Icahn School of Medicine at Mount Sinai (ISMMS) and the Director of the Adult Leukemia Program and Leader of Clinical Investigation within the Myeloproliferative Disorders Program at Mount Sinai. Learn more about Dr. Mascarenhas, here.

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Transcript

Katherine Banwell: 

What tests are necessary to help understand a patient’s specific disease at diagnosis?

Dr. Mascarenhas:

Usually, the blood counts are the first opening door test that allows some understanding of, again, either an abnormal production of red blood cells, platelets or under production of these cells. And, that’s really where often the evaluation begins. And then, there are further blood tests that often are done.

And I would say almost indefinitely or almost definitely one should have a bone marrow biopsy that helps categorize the type of myeloproliferative neoplasm because there can be overlap in how the blood counts can look from one disease to the next and overlap in the mutations like the JAK2 mutation. So, sometimes, the blood counts and the molecular testing are not enough, and a bone marrow biopsy looking under the microscope at the different types of cells, the proportion of cells, whether there’s fibrosis where there’s others other types of cells that shouldn’t be there and they’re looking at the chromosomes and the flow cytometry, these are associated tests. As well as almost probably anywhere anyone goes at this point, they’re going to get next-generation sequencing, which is looking at multiple genes and mutations, and that gives a more broader, deeper sense of the disease.

So, those really become the integral parts. In some cases, patients will end up getting imaging of their abdomen to see if they have an enlarged spleen or enlarged liver.

Although that’s not always necessary, that is often part of the workup. So, it’s bloodwork, it’s bone marrow biopsy, sometimes imaging is usually the cornerstone.

Katherine Banwell: 

And, what is molecular or biomarker testing?

Dr. Mascarenhas:    

So, molecular testing today really means – at one point, it really meant looking at PCR for specific gene mutations.

So, for example, we would look at the JAK2 and we would say, “In a given person, is this gene mutated?” We all have JAK2 gene, but in patients with these diseases, they’re more commonly mutated which means altered in the blood cells. And, it’s very important for a patient to understand not in every cell in their bodies, but in their blood cell compartment. And, that helps us understand and start characterizing their disease, and sometimes that mutation can be measured. It can be at a low level. It could be a high level. And, that’s all put together in trying to understand the molecular basis of these diseases.

Today, next-generation sequencing has really taken over and that’s looking at more than just one gene.

Its sequencing could be 40 genes, it could be 200 genes, to get a sense of the complexity of the disease and looking for certain mutations which are considered biomarkers that can portend prognosis or I think increasingly, we’ll see may inform treatment decisions and may even be targets themselves of therapies.

Katherine Banwell:              

Right. Should all patients diagnosed with ET, PV, or MF undergo biomarker testing? Is that necessary?

Dr. Mascarenhas:       

I would say it’s part of the modern evaluation and management of patients today. I don’t think that that was true 10 years ago. But, I think the field has matured. I will say I’m the first person to acknowledge to patients that we get a lot of information back, and the truth is we don’t often know what to do with all of that information. So, sometimes we get information back that can cause anxiety because you can see mutations in genes. But they don’t always inform us on how to educate the patient about their disease or tell us what to do with the treatment.

So, there is still a lag as there normally would be between the testing of the results that we get, and then the actual knowledge of what to do with that. And, that’s still a process that’s in evolution.

How Does Myeloma Testing Affect Care and Treatment?

How Does Myeloma Testing Affect Care and Treatment? from Patient Empowerment Network on Vimeo.

What is cytogenetic testing in myeloma? Donna Catamero, a nurse practitioner specializing in myeloma, describes this in-depth testing, including the FISH test, and how the results impact the care of patients.

Donna Catamero is Associate Director of Myeloma Translational Research at Icahn School of Medicine at Mount Sinai Hospital in New York City.

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Transcript:

Katherine:

Blood and urine tests, bone marrow biopsy and imaging tests are all standard following a myeloma diagnosis, but what about more in-depth testing?

Because the terminology around biomarker testing varies, can you help break this down for patients, and how this in-depth testing is referred to in myeloma?

Donna:

So, biomarkers is a term that is commonly tossed around in many different cancer diagnoses and it means different things. But in general, it’s characteristics that can inform us about a diagnosis, about a patient’s prognosis and about their response to treatment. So, this can include things that we measure in the bloodwork, in the urine, even imaging. These are all things or markers that we look at to determine a patient’s either, like I said, response or risk stratification.

Katherine:

What about cytogenetics? What is that exactly and does that fit under the umbrella of biomarker testing?

Donna:

Yeah, so cytogenetics is a genetic snapshot of a patient’s cancer. So, it will give us a sense of how the disease will – the characteristics of how it will behave. But again, it’s just a snapshot and it’s not a precise science but certain mutations or certain genes will kind of inform us like “This might be maybe a more aggressive form and we need to do X, Y and Z.”

Katherine:

Which of these more in-depth tests are necessary in myeloma? Let’s start with the FISH test.

Donna:

So, FISH is a cytogenetic technique. So, what we do is, when we do the bone marrow, we send that off and we look at the genetics. Like I said, it’s a snapshot. And certain mutations will put patients in different risk stratifications, so we normally do this at the time of diagnosis and then with each relapse.

Katherine:

It seems that all of the test results can aid in determining outpatient’s risk. So, why is risk stratification so important?

Donna:

So, risk stratification is important.

It will give us a sense of how a patient might respond to certain treatments. Maybe a patient won’t respond as well to a stem cell transplant as someone with standard risk. So, we take this into account, but in this current time, in 2021, we don’t typically change our treatments according to risk. That’s why clinical research is very important because we’re studying right now patients with high-risk cytogenetics, do they do they better on certain therapies.

Katherine:

How do the results of these tests affect treatment choice and prognosis?

Donna:

So, someone who might have high-risk cytogenetics, we might want to be maybe more aggressive with our therapy. So, we might change how we want to maintain a patient. Usually, after a stem cell transplant, we give patients maintenance therapies. So, patients who have high-risk disease, we might change our strategy and have a more aggressive regimen in that maintenance setting. And with patients with higher risk, we probably will monitor them very, very closely in case – looking for signs for relapse. 

What Standard Testing Follows a Myeloma Diagnosis?

What Standard Testing Follows a Myeloma Diagnosis? from Patient Empowerment Network on Vimeo

What tests will you have following a myeloma diagnosis? Are there additional tests you should request? Dr. Joshua Richter provides an overview of key testing for myeloma and why each test is necessary.

Dr. Joshua Richter is director of Multiple Myeloma at the Blavatnik Family – Chelsea Medical Center at Mount Sinai. He also serves as Assistant Professor of Medicine in The Tisch Cancer Institute, Division of Hematology and Medical Oncology. Learn more about Dr. Richter, here.

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Transcript:

Katherine:

What standard testing follows a myeloma diagnosis?

Dr. Richter:

So, the standard testing that follows a myeloma diagnosis is multifaceted. So, the first one is blood work. And we draw a lot of blood tests to look at the bad protein that the cancer cells make. So, we send tests like a protein electrophoresis which tells us how high that bad protein is. We send immunofixation. That test tells us what type of bad protein it is. You’ll hear names like IgG kappa and IgA lambda.

These are the different types of bad proteins made by myeloma cells. Oftentimes, we’ll send urine tests to find out how much of that bad protein that was in the blood is coming out in the urine. We will, typically, do a bone marrow biopsy. It’s a test where we put a needle into the back of the hip bone to look at the marrow itself. And we’ll use that marrow to figure out how much myeloma there is, any other characteristics like the genetic changes in those cells.

The other big thing is imaging. So, the classic imaging that we do with myeloma is something called a skeletal survey. It’s, basically, a listing of X-rays from head to toe. But nowadays, we have newer techniques, things like whole body low-dose CAT scans, something called a PET-CT scan, and MRI scans. And your care team may have to figure out which one is right for you at what given time.

Katherine:

Mm-hmm. Are there additional tests that patients should ask for?

Dr. Richter:

Absolutely. One of the most important things from myeloma has to do with the genetic risk stratification.

So, for almost all cancers, the staging has a very big impact. And people will often think of cancer in stages I, II, III, and IV, and they’re managed very differently depending upon what stage it is. Myeloma has three stages, stage I, II, and III. But the most important thing is, actually, beyond the staging is what’s called the cytogenetics risk stratification. So, it’s really important when the bone marrow is sent to be sure that it is sent for, kind of, advanced techniques. Because you really want that snapshot of exactly what the genetic profile is, because that gives us information of A) how to treat, and B) prognostic, you know, who will tend to do better or worse based on this information. And even though that may not tell us which drugs to use, specifically, it may say, should we do something like a transplant or not? Should we consider a clinical trial early or not?

Katherine:

I see. How do test results affect treatment choices?

Dr. Richter:

So, test results can affect treatment choices in a number of ways. Probably, the most common one is thinking about the routine blood tests like your CBC or complete blood count and your chemistry, which looks at things like your kidney function. Some drugs tend to have more toxicity to the blood counts. So, if your blood counts are very low, we may choose drugs that don’t lower the blood counts very much.

Kidney function which we, usually, measure by something called the creatinine. Creatinine is made by the muscles and cleared out by the kidneys. So, if your kidneys aren’t working very well, you don’t pee out creatinine, and that creatinine level will rise in the blood. If your creatinine level is high, we may choose certain drugs that don’t affect the kidneys or not metabolized or broken down by the kidneys.

The genetic studies that we use – we’re not quite at this base yet where we can say, if you have this genetic abnormality in your myeloma, we should use this drug except there’s some really great data on the cutting edge about a drug called venetoclax.

Venetoclax is a pill that’s used to treat other diseases like lymphoma and leukemia. And it turns out that people who have what’s called a translocation (11:14) which means part of the 11th chromosome and part of the 14th chromosome in the cancer cells swap material.

Those people respond amazingly well to venetoclax. So, we’re starting to have what we would call precision medicine where we find your genetic abnormalities, not that you got from your parents or passed to your kids, but the genetics inside the tumor cells to tell us which treatments will work best for you.