What Are the Current Treatment Options for AML?
What Are the Current Treatment Options for AML? from Patient Empowerment Network on Vimeo.
An expert panel help viewers understand more about the evolving field of AML treatment. This includes identifying prognostic factors and determining patient subtypes to setting treatment goals and selecting a suitable course of treatment. The panel was made up Dr. Uma Borate of Oregon Health & Science University, Amanda Fowler of The Leukemia & Lymphoma Society, and patient advocate, Don Armstrong
Transcript:
Andrew Schorr:
And greetings from Los Angeles. I’m Andrew Schorr. Welcome to this Patient Empowerment Network program produced in association with the Leukemia & Lymphoma Society and with support from the following companies: Celgene, Daiichi Sankyo, Jazz Pharmaceuticals and Novartis. We thank them for their support, but be advised no outside party has any editorial control.
In this program we’re going to discuss the latest the treatments for acute myeloid leukemia. We have a wonderful panel, and over the next hour we will be discussing the options, but of course always discuss this with a healthcare team that you trust so you get the care that’s right for you or a loved one. Also, if you have a question send it in to aml@patientpower.info, aml@patientpower.info, and we’ll get to questions as we can.
Today with us with have with us a pretty long‑term survivor of AML joining us, and we also have an information specialist from the Leukemia & Lymphoma Society and a noted physician researcher from Oregon Health & Science University. So let’s go around the country and meet them. I’m in Los Angeles.
Let’s go to Fort Worth, Texas, and joining us now is Don Armstrong, who was treated for AML. Gee, Don, in 2005 your whole world turned upside down, right?
Don Armstrong:
It absolutely did. September of 2005 I had no idea there was anything wrong with me until I had a couple ladies tell me on a Friday afternoon I looked horrible. That ultimately led me to see my general practitioner, and a couple of days later I was diagnosed with acute myeloid leukemia, and from there right into the hospital, right into treatment.
Andrew Schorr:
Right. And you were in the hospital for a long time.
Don Armstrong:
I was. I was in the hospital the first time for 33 days.
Andrew Schorr:
Wow. And ultimately you had a transplant, which is one of the approaches still for AML, and you survived.
Don Armstrong:
I did. I did. Yeah, it was interesting. That first couple of days in the hospital there was so much activity going on around we and I couldn’t understand why I was getting so much attention until one of the nurses kind of pulled me aside and said, you understand you’ve got a 25 to 30 percent chance of surviving this type of leukemia, don’t you? And I said, now I do, yes. So it was quite a shock to the system, and it was something that‑‑you just have to kind of hang on and just go with the program as much as you can.
Andrew Schorr:
And a shock to the family. I mean, it’s a family affair.
Don Armstrong:
No question about it. Whenever you’re diagnosed with cancer, no matter what the cancer is, it’s just not you. The entire family is involved. And I had a great support system. My dad and my brother and my sister came from different parts of the country just to be with me and support me. I had an unbelievable group of family and friends that were there with me every single day.
Andrew Schorr:
And you had a transplant. Where did the donor cells comes from that sort of rebooted your immune system?
Don Armstrong:
That’s a great question. I’m actually currently still looking for my donor. My donor, he was happy. I know he’s a male. He was excited that he was able to help me, but I have never able to connect with him. I’m trying again. I just‑‑so through the Be the Match I got my stem cells, and fortunately there was someone there that was willing to give my life a second chance.
Andrew Schorr:
Now, Don is very active with the Leukemia & Lymphoma Society, which is a partner in this program. Don, you’ve spent many years now, your career has been in golf and you were a golf superintendent, and yes, you sprayed pesticides on the golf course. You’re devoted to giving back. Talk the a little bit about that and what you try to tell patients and families so they can get through this hopefully successfully, as you have.
Don Armstrong:
You know, like we had talked about, I had no idea there was anything wrong with me when I found out I had leukemia, and I was in for a pretty big fight of my life. After five rounds of chemo and a stem cell transplant, making it through that, I felt like I had been given a second chance, so I wanted to find a way to give back. And I found the Leukemia & Lymphoma Society. I found one of their campaigns, Team in Training. Started doing the endurance events and raising money for that organization. Just did my 2015 retraining event, but through it all I really wasn’t doing it just to run a marathon. I was doing it to raise money for research so that somebody else hopefully didn’t have to go through what I went through. So for me that was a big shift in my mindset. That eventually led me into being a part of the board of trustees, and I continued along that line. And I try to stay up on as much about AML as I can, and I talk to as many patients around the country as I can whenever I’m given the opportunity.
Andrew Schorr:
Well, you’re going to hear a lot today, and research has paid off. Let’s go up to Colorado outside Denver where Amanda Fowler is an information specialist with the Leukemia & Lymphoma Society. And that means if you call, and I urge you to, whether a patient or a family member, they can help because based on research the world of AML has changed significantly and gives people a great deal more help and is changing those statistics that Don talked about. Amanda, welcome to the program.
Amanda Fowler:
Thank you it so much for having me.
Andrew Schorr:
So I’m right. Research has been paying off just in the last year or two and even with more research coming our way, right?
Amanda Fowler:
It’s incredible. There have been drug approvals in the last two years. There are numerous clinical trials. We are really seeing AML treatment change for the better at a pretty rapid pace right now.
Andrew Schorr:
Okay. So just to be clear, if someone who is watching now calls the Leukemia Society like the national number, and maybe you can tell us again, how do they get to you so you can help them sort this out?
Amanda Fowler:
Absolutely. It’s really easy to reach an information specialist. We are open 9 a.m. to 9 p.m. eastern time and our number, which we can repeat at any time, is 1‑800‑955‑4572, and that will take you straight to an information specialist. And once you reach us we’re really there to talk and figure out exactly what you need.
So if you’re not sure if you need help or not, give us a call anyway, and we can go over a lot of the resources and services from psychosocial support to financial resources, disease education. We have a clinical trial support center, so a whole variety of resources, and we encourage people when in doubt to reach out to us.
Andrew Schorr:
Okay. All right. Let’s hear about the research. So joining us from Portland, Oregon, at Oregon Health & Science University is Dr. Uma Borate, who is a hematologist‑oncologist. Dr. Borate, thanks for being with us.
Dr. Borate:
Absolutely. Thank you for having me.
Andrew Schorr:
Okay. So we’ve alluded to changes in AML, payoffs in research. You’re still in the lab moving research forward with your peers around the world. You all are making progress, am I right? The world and the options, the combinations, things you’re researching, that’s all changing incredibly fast.
Dr. Borate:
It absolutely is. And I just wanted to first just appreciate Don and his journey because we see patients like him every day, and we deliver these very shocking and stressful diagnoses to patients and kind of see the journey of absorbing what this means for them. The family rallying around them. And then the treatment and the eventual, you know, the role that takes them hopefully to a cure. And I just‑‑every day when I talk to my patients I just applaud their courage. So, Don, I just wanted to put that out there. You guys are awesome.
With that said, I think in the last I would say four to five years, and Amanda can attest to this, we’ve had over nine FDA‑approved therapies for AML after about four decades of no progress. And a lot of this has come with discoveries in the lab where we have identified specific genes that have had genetic changes, what we call genetic mutations, that lead to a patient developing AML.
And now we have what we call targeted therapies where we can target that specific genetic mutation and therefor destroy the AML cells in a way that doesn’t expose the patient to a lot of additional toxicities. However, for a certain subset of the AML patients, like Don, we know that these targeted therapies can work for a while, but if you are going for what we call a curative active therapy, for a lot of patients transplant is still right now one of the most, I think, advocated and proven curative therapies out there.
Andrew Schorr:
Okay. So let’s talk about testing. So somebody is diagnosed with AML. What should happen now so that you as a specialist, the doctor, the team that they see, they know what version of AML you have and whether it matches up with either one of these approved therapies or maybe something you at an academic medical center are researching that could be the drug of tomorrow?
Dr. Borate:
So I think that’s a great question, and for patients or family members or anybody else listening out there I think what you a alluded to as the most crucial step in the diagnosis of AML and the subtype of AML is the testing. So as soon as we identify a patient, like Don described, typically it’s, you know, you don’t feel well. You go to somebody, they say, oh, my god, your blood work doesn’t look right. You go to another doctor.
The first thing that we ask that happens is when a bone marrow biopsy, which is the diagnostic procedure that gives you the diagnosis of AML is done, that it be subjected to adequate genetic testing. And by this I mean there are many, many laboratories out there that do what we call expanded genetic mutational panels, and they test for all the different genes that could have a mutation that potentially could be targeted or make the patient a candidate for a clinical trial in the future.
Leukemia & Lymphoma Society is sponsoring an extremely revolutionary trial called BDML, which we are a part of, which does this testing and returns the results back to the clinical provider or the physician in seven days, which is really unheard of in terms of a timeline. This used to take about two weeks on average.
And so in seven days I know all the genetic changes in my patient’s leukemia and I can determine, hey, is this the right therapy for them? Should they go on a clinical trial that we have? And BDML offers several of what we call (?) Inaudible that are used to match them to the appropriate clinical trial or the appropriate drug.
Andrew Schorr:
Okay. So, Amanda, let’s talk about this for a second. So, oh, my god, a patient is diagnosed with this acute condition. Don found himself in the hospital right away, and I understand there can be different versions of AML. Some like do not pass go, boom, you’re going to the emergency room at the hospital right away, or some there’s a little more time. But a call comes to you, and people want to feel confident that where they are or where‑‑do they go to this hospital or that hospital, this clinic or that one. They get someone who is knowledgeable when the whole world of AML has been changing.
So how do you counsel people so they get the right testing and just all the range of options are considered for them?
Amanda Fowler:
Sure. Absolutely. So I should say a lot of the calls, particularly for the acute leukemias, actually come not from the patient but from the caregiver. The patient is often sick and overwhelmed, and it’s a family member who is making the calls. Of course we are having to talk to both the patient or the caregiver, but it is really important to be at a center of excellence for this diagnosis.
Not all hospitals are equipped to handle AML. If you’re lucky, a local hospital will see that and send you on to the bigger center, but sometimes that work does fall on the family member. We recommend that people go to university‑type settings or National Cancer Institute comprehensive cancer centers. These are going to be the larger hospitals that understand what tests are involved. They will have clinical trials as options, and they will be knowledgeable on the latest treatment options.
Andrew Schorr:
Right. So, Don, just to you. You talk to patients, and you speak around the country. It takes a lot of courage for a patient or family member to say, thank you, Doctor, but I think we’re going to go, take mom or dad, we’re going to go over there because, you know, I mean, hospitals are competitive, right?
Don Armstrong:
Absolutely.
Andrew Schorr:
But you and the family want to make sure that your loved one gets state‑of‑the‑art care. What would you say to them to be a good consumer?
Don Armstrong:
Well, I agree with what Amanda said. I think it’s important that you’re in the right hospital, the right setting because not all the hospitals can actually handle the diagnosis of AML. So I think it’s really important that you’re in a center that can treat that, that’s got experience treating it. And so I always tell patients call the Leukemia & Lymphoma Society’s IRC and find out where is the best place to go.
Andrew Schorr:
And here’s Amanda. And, Amanda, you have a medical advisory board. You keep lists of AML specialists, right? So you can actually say where in where you live, in Kansas, in California, in Texas, etc., here are centers of excellence, right?
Amanda Fowler:
Yes, absolutely. We will talk to patients about where to go, wherever it is that they live. And then occasionally if it’s later on in the treatment and they’re considering transplant they may even look to travel, and we’ll help discuss any option that they want to get them to the center of excellence.
Andrew Schorr:
Okay. All right. Dr. Borate, let’s go back to the basics just for a second because we have people who are trying to understand what went wrong. I’m a leukemia patients too, but with chronic lymphocytic leukemia, but I know that our bone marrow often in our hips and bones is the blood factory. What went wrong in that blood factory, and how does it show up in AML?
Dr. Borate:
So, thank you, Andrew. I think that’s the so‑called million dollar questions is we know that there’s a combination of factors that can cause what we call these genetic mutations that then go on to lead to the actual disease.
So whether it be CLL, which you alluded to, or AML, age is a big factor. So as all of us grow older the unfortunate reality is as our cells divide they accumulate genetic changes that (?) Inaudible repair, so that’s one thing that happens to all of us. Environmental and genetic factors play a big role, and I think the new emerging field in this is what patients would ask us, why did I get this? We would say, well, you were unlucky. You had this mutation. Something happened.
But now we know about 10 to 15 percent of leukemias actually have a genetic or what we call an inherited component. So if you talk to patients they would have‑‑some patients have a very strong family history, not just of leukemias or lymphomas, which are blood cancers, but other cancers. And I think it’s really important to nail that down and explore the inherited aspects because for patients like you or Don, if you have kids and grandchildren, you know, those have far‑reaching implications down the road.
However, 85 percent of these leukemias are what we call sporadic, meaning they just came about because of environmental and genetic factors that sort of played a role in one or two cells developing the mutation and then there is a competitive advantage for these cells. They start growing, you know, without any checks and balances, and once that happens they start crowding out the healthy cells in your marrow and they sort of replace, as you said, the nice, healthy cells in your bone marrow that should be making your red blood cells, white blood cells and platelets.
And sometimes the analogy that’s given is you see these weeks on a lawn and once the weeds start growing they kind of take over all the healthy grass because they compete for nutrients and water, and then all you get is a lawn full of weeds.
Andrew Schorr:
Okay. So somebody comes to the emergency room, like Don looked sick. Is it fatigue? Is it bleeding? Is it just what‑‑how do people present, as you doctors say?
Dr. Borate:
So typically patients present with this feeling that they’re not‑‑they just don’t feel well. Typically it’s fatigue. Sometimes they’ll notice bruising or spots all over their bodies. They’ll notice that their gums are bleeding easily when they brush their teeth or they have nosebleeds when they’ve never had them before.
A fair number of patients actually present with an infection, so a sew throat that doesn’t seem to go away. They get swabbed for mono, and the practitioner sees these weird cells in their blood, and they think, well, maybe this is mono because they’ve had fatigue, sore throat and some lymph nodes, and so that’s the way people present.
Sometimes people present really sick, with a pneumonia or another infection, and then that’s when you go to the ER and you come into the hospital and it’s like, oh, wow something else is going on.
Andrew Schorr:
Okay. So they get to you, let’s say, at Oregon Health and Sciences in Portland, a major university center, and you run this genetic panel. Now, it’s seven days. So, first, what’s going to happen while you’re trying to figure out what version of AML they have and whether you have a therapy.
Dr. Borate:
Yes.
Andrew Schorr:
So let’s just talk about that. What happens first, and then based on the information what happens then?
Dr. Borate:
So it really depends on what you said before is how sick you are and what type of AML that you have. So I would say even now about 30 percent of our patients don’t come through the ER and are not that sick. They come through an outpatient clinic appointment where they’ve had low blood counts, they’ve been tested, there’s some testing that is done which indicates they may have leukemia, and they actually come to my clinic.
And if they come to my clinic and they’re relatively what we call stable we will perform all this workup, as we call it, as an outpatient. So we’ll to the bone marrow biopsy as an outpatient. We will let them be at home for those seven days so they can sort of start preparing for, hey, maybe there’s something going on and this will need some length of treatment in the hospital or multiple visits to the physician, in which case if you’re working you need to start thinking about time off and preparing, and if you have kids how to they get to their activities. So all the different things that people are struggling with when they get this diagnosis. So that is about 30 percent of patients.
And in those seven days while they’re waiting, sometimes it’s longer, we keep a really close eye on their blood work. So if they live close to a hospital or a clinic we make sure they go to the clinic at least two or three times a week to see what their white blood cells, platelets and red blood cells are doing, and then we get those results. So we monitor them before they come back to get the final diagnosis and what their position is.
If it’s somebody like Don who ended up in the hospital really sick, then they stay in the hospital while we’re doing this testing. Typically they will get blood. They will get platelets. They would get what we call a workup, meaning we will check their heart, their kidneys, their liver. We would put what we call a central line, meaning a line or an IV that can stay in their bodies for a longer length of time that can allow them to get treatment and allow them to get blood work and transfusions. So all this is happening in the background while we are figuring out the subtype of AML.
The other thing that we also do at that time is we collect what we call HLA typing, and this is to figure out what the tissue type of the patient is. So like Don, when he went on to the transplant it’s really important for us to know this beforehand. So while the patients are getting treatment in the hospital we can see if they have matches. So does your brother or sister, can they be a match for you to donate bone marrow, or does it have to be somebody through Be the Match, as Don said, would that‑‑would it be what we call an unrelated but matched donor that would then be an option for you in the future.
Andrew Schorr:
Okay. So now the testing comes back. This next generation sequencing, which is so cool now to see your cancer genes, what cancer genes are active, what could be driving your acute myeloid leukemia, and it says this gene. And I know they have a lot of different letters, IDH, FLT3. You could probably name a whole bunch others that different drugs have been developed for. So it comes back with this letters and you say, ah‑ha, if we have a drug that targets that we can tamp this down. Right?
Dr. Borate:
Yep.
Andrew Schorr:
Are these drugs infused? Are they pills? My understanding is now you have some pills that people can take as well.
Dr. Borate:
So I think that’s one of the really cool things moving forward is most of these new targeted agents are actually oral, so medications, so pills that patients can even take at home to treat their leukemia.
So just to back up a little bit, once we get back this genetic testing and we know their mutations and like you said IDH1, IDH2, FLT3, these are all mutations that can be targeted, we also determine a little bit‑‑and this can be somewhat arbitrary but is determined more by the patient, their age, their ability to, you know, how able are they to do their day‑to day activities? Are they somebody who really is not even able to go to the grocery store without being really tired? We call it, for lack of a better word, performance status. How do they do in their everyday life?
So we take all these factors to consider two broad categories: Is the patient what we call fit versus, and I know this is not the kindest word, we call it unfit. And I think those broad categories then lead us to what type of therapy should the patient get. Should they get what we call intensive induction, meaning we still give them very broad chemotherapy to kill all the leukemia, but now we’re adding targeted therapy to the chemotherapy so that you give this double‑whammy? You knock them with chemo, and you knock it also with the targeted therapy.
However, if you happen to be 85 and you’re a very functional 85, maybe, but you’re not somebody whose organs can tolerate this heavy intensive chemotherapy or a transplant in the future, then we go with what we call more therapy that’s what we call less intense even though it might be IV, but then we add these targeted agents which they can take at home as a pill and then they’re not in the hospital as much. They get this therapy as an outpatient while they’re getting treatment for their AML. So it’s very different based on our goals of care, the patient in front of us and what mutations they have.
Andrew Schorr:
Okay. So increasingly now you may‑‑maybe somebody would get some chemo, working on approaches where you don’t lose your hair.
Dr. Borate:
Yes.
Andrew Schorr:
You don’t develop mouth sores.
Dr. Borate:
Yes.
Andrew Schorr:
Or ad nauseam. You’re working a lot on lowering the toxicity.
Dr. Borate:
Correct.
Andrew Schorr:
And, Don, I know you went through that you’re being prepared for a transplant, heavy‑duty. But that’s been ameliorated to a greet degree for many people.
Dr. Borate:
Yes.
Andrew Schorr:
And then somebody may be on a pill. Now, do you have a growing range of treatments so if you try one and it doesn’t work or it doesn’t last you have something else? It’s kind of like an antibiotic. The doctor says we’re going to try this antibiotic. No. Your swollen glands and lymph nodes, it’s not going away. Let’s go to this one.
Dr. Borate:
We absolutely do. And I think that’s sort of the next frontier is what we call sequencing. How do you sequence treatments so that you can continue to get a good response even when the patient fails a treatment and the leukemia decides or figures out how to outsmart that treatment? And unfortunately that still happens even with targeted therapies that over time the leukemia figures out a way to survive despite a very targeted approach. And so how do you come back in with a different drug that can still work? And how do you sequence those drugs to give them maximum effect but the least toxicity as you said, to the patient? And those are sort of our next frontiers of clinical trials and therapy.
Andrew Schorr:
So at this interim stage, Dr. Borate, one key question: Don talked about the statistics, which were not good when he was diagnosed.
Dr. Borate:
Yeah.
Andrew Schorr:
Are you seeing a change in quality of life, and you believe in survival based on everything you’re talking about?
Dr. Borate:
Yep. I’ve seen a huge change in quality of life. And I will say interestingly in AML more than any other disease we have really pushed this aspect in our older patients. Because we have heard loud and clear from patients who are 70, 75, 80, that they want to live. They want to live as long as possible, but they also don’t want to spend all that time in a doctor’s waiting room in or the hospital. They are very, very determined to have a good quality of life and enjoy whatever it is that they want to do.
And I think we have really worked hard to deliver that with our targeted therapies, and I want to say the results are astounding. I have an 89‑year‑old right now who celebrated his birthday and has been outpatient for the last year since his diagnosis. An 85‑‑oh, an 86‑year‑old, and we celebrated her birthday on (?) Inaudible where she took a pill for 13 months and is in remission and is talking about taking a trip to Bolivia.
So to me these are huge success stories for my patients because I have a soft corner for my older patients. They have struggled, they have sort of supported their kids their whole life, and it’s their time now. And I think that’s so important to deliver that to them.
Andrew Schorr:
Well, Dr. Borate, first of all, thank you for your devotion to patients. We’re going to talk more about treatments. We’re going to be taking your questions from our audience along the way, aml@patientpower.info.
Don, you have been in Team in Training with (?) Inaudible, run I don’t know how many marathons, and it’s for research. This must make you feel good that research is paying off.
Don Armstrong:
On this side of the screen I am smiling so, so huge, and I’m actually very emotional about it. It’s great to hear what Dr. Borate is saying because it gives other people a lot of hope and encouragement. It’s just great to hear because it was not an experience that I would want anyone to go through, and so these are big moments. So thank you, Dr. Borate, for what you’re doing.
Andrew Schorr:
And if mom and dad want to go Bolivia or Thailand or Europe or wherever it is their time, as you say, Dr. Borate.
Dr. Borate:
It is their time.
Andrew Schorr:
So, Amanda, let me go to you. So the Leukemia & Lymphoma Society has lots of services. Some of it‑‑and your cat’s going to help us too, there‑‑is first of all getting information. Where can I or mom or dad or grandma or grandpa, Uncle Charlie get the right care, okay, state‑of‑the‑art care? But also then as we get to some of these treatments then they say, oh, my god. There’s all this medical care and medicines coming in, and there’s expense. So you all help people with that, too, right?
Amanda Fowler:
Yes, absolutely. We hear all the time how expensive treatment can be, and these new drugs are amazing but they do come at a cost. So we have various different options to help patients through a program like copayment assistance that’s available most of the time but is subject to availability. So we encourage people to call us. And if there’s something that we don’t have we will work with the patient to find other options.
It is a big burden to people, and it’s certainly one of the things that people worry about the most. And this does often come from the caregiver because, like we said, the patient may be feeling pretty ill or be in the hospital, so we just encourage you to call and see what’s available.
Andrew Schorr:
Okay. Yes, I know. I take an oral therapy for another leukemia, chronic leukemia, and I’m on Medicare, and I have Medicare Part D and I have a substantial co‑pay. Now there are foundations, the Leukemia Society, that depending upon your need can help. And if you’re on commercial insurance and you’re younger and not on Medicare there are other programs that come into play, right, Amanda?
Amanda Fowler:
Yeah. We will talk to patients, whether it’s through a nonprofit like us or through the pharmaceutical company directly. And I certainly encourage people when they’re making that transition to Medicare, which often they know about in advance, to call us and we can help talk to you about ways to make that transition a little bit easier because you do find that the cost out of pocket to the patient can sometimes go up when they make that transition.
Andrew Schorr:
Okay. Dr. Borate, I have questions for you. Maybe you is tilt your screen down a little. We’re just losing you. There we go. There, that’s good.
Dr. Borate, so you mentioned clinical trials. We talked about research. So you’re doing clinical trials, and many of your peers at other academic medical centers are doing clinical trials, and that’s what led to the approval of these drugs by the FDA based on data that you as researchers and drug companies and National Cancer Institute were able to present. So talk to us a little bit about what’s in the lab, if you will, that you may be offering patients in clinical trials. And then we’ll also understand, Amanda, when somebody is in a clinical trial what costs may be covered, too, okay?
So, Dr. Borate, first, what’s going on in research?
Dr. Borate:
So I think, as you said, clinical trials really pave the way to new therapies. And again I want to have a special acknowledgment and shout‑out to all the patients and their families who participate in these studies because it is a little bit of a leap of faith. You know, sometimes we’re not always sure these treatments will work and they have side effects, so for patients to put their trust in us and sign off for these studies is I think a big deal. So thank you.
When we start somebody on a clinical trial we always collect what we call a pretreatment sample. So we’ll get a sample of their disease before they’ve had any treatment, and then along the way as this treatment progresses we get multiple what we call post‑treatment treatment samples, one to look at the status of their disease, and secondly to send the sample then back to the lab to understand how these new treatments are affecting the disease. You know, what pathways in these leukemia cells are being inhibited so that the cells are dying? What pathways are deactivated, which also helps the cell to die?
And then thirdly, what pathways are being sort of turned on to help the cell resist these treatments. We call them mechanism of resistance and it’s similar to the antibiotic analogy you said where you take an antibiotic for a while and it seems to be working initially but then your body develops resistance to it and so the provider or the doctor has to change therapy because now this drug no longer works for your infection.
And so the same thing happens with leukemia or any cancer, and I think it’s very important for us to observe the samples as the patient progresses through therapy so we can figure out, first of all, why it worked, but also why did it stop working or why did it not work. And I think that’s where participation in clinical trials is so critical because without this valuable information we really can’t move the field forward.
Andrew Schorr:
Okay. Amanda, just about costs. So if somebody signs up for a clinical trial might some of the costs be covered?
Amanda Fowler:
Yeah. It’s actually a bit of a complicated question, but generally speaking the cost of the actual trial drug is covered through the trial itself. But it is important to talk to your insurance provider because hospital stays and some other supportive care may still go through your insurance.
Andrew Schorr:
Okay. So it’s complicated, but it’s something to discuss. Dr. Borate, one other thing about testing. So we mentioned somebody gets to the hospital, and ideally there’s this panel, this next‑generation sequencing done, fast track trying to get the results back to the specialist to decide do you have a targeted therapy or what’s going on, what’s your version of AML.
But cancer is wily, and the cells can change, and the cancer gene that was driving your AML on day one could be different on day 50 or 100.
Dr. Borate:
Absolutely.
Andrew Schorr:
You would know the (?) numerical better than I. So is retesting sometimes needed if something changes?
Dr. Borate:
Yep. So I think this is a great question, Andrew, and I think it ties into what Amanda said about centers of excellence because this is something we routinely do in our AML patients every time. We sort of look back and see what their disease is doing, and we call it re-staging. And sometimes we see certain genetic changes or mutations come in even when the patient is in remission. When we can’t see the AML we can see some of the background cells acquire different mutations so we can keep a closer eye on the patient as they continue down the road. So I think retesting is one of those things that is not done enough if you don’t have experience with the disease.
Because you’re absolutely right. The disease is wily. It does change. The mutation that was driving your disease to begin with may not be the one driving it when it relapses, and I think that’s really where we need more information, and we need to have this testing done in order to treat the patient appropriately.
Andrew Schorr:
Okay. So let’s go back to what leads to AML for a minute. So Don, you worked on golf courses your whole life and eventually became the superintendent of one of the more famous PGA golf courses. Colonial, is that right?
Don Armstrong:
Correct, yes.
Andrew Schorr:
In Fort Worth. But over your years devoted to golf you sprayed a lot of pesticide, right?
Don Armstrong:
I did. I did. From the time I was 15 years old I get my first job on a golf course and the superintendent knew I wanted to be in the industry, and so he let me do a lot of things that probably somebody else wouldn’t. And one of them was spray the pesticides, pray the fungicides, herbicides. You name it, I got to spray everything. And back in that time frame the pesticides were a lot more potent. We had a lot of mercury‑based, lead‑based products in those time frames that the EPA had not stopped in terms of use.
And so I think back about whether that may have had an impact on where I ended up with the leukemia. It’s hard to say. I’m sure Dr. Borate would probably agree with that, but it seems to me there could be some correlation, yes.
Andrew Schorr:
Probably so. So environmental factors. Dr. Borate mentioned hereditary to some degree. And I want to ask about another thing. I am not just living with chronic lymphocytic leukemia but I have another blood condition called myelofibrosis. And some people with myelofibrosis progress to AML. My understanding was this secondary version of AML was often harder to treat.
Dr. Borate:
Yes.
Andrew Schorr:
And I also understand, Dr. Borate, you’re researching it.
Dr. Borate:
Yes.
Andrew Schorr:
So are the options, is the chance for hope for people who developed this secondary AML?
Dr. Borate:
So thank you for bringing that up. I think secondary AML has always been this sort of thing that people don’t want to touch and especially when you’re looking at clinical trials investigating your new agent because we knew that, hey, if you were treating patients with secondary AML with a newer therapy maybe the results wouldn’t be as good and then your clinical trial results overall don’t look at good.
And to me that is a huge disservice to our AML patients because, as you describe, either because you had a prior cancer, let’s say you had breast cancer and you had chemotherapy for that breast cancer or lung cancer and you survived it, and so you’re a survivor. And 10 years later you develop AML as a result of exposure to prior chemotherapy, which in many ways does things to your stem cells like a pesticide that Don might have been exposed to. (?) Sorry, I apologize.
And so I think the thing that secondary AML has taught us is when a patient develops secondary AML there is a large number of genetic mutations already existing in the patient from their prior chemo, from their prior cancer, from their prior exposures. And so because the treatment is so hard it’s something that we’re looking about very carefully in the lab to understand it much better.
And especially about secondary AML from myelofibrosis or these diseases we call MPNs, meaning myeloproliferative neoplasms maybe essential thrombocytosis, polycythemia vera, all these big names. We have a study here at Oregon Health and Sciences University which combines our targeted therapy. You might have heard about it. It’s called Jakafi is the commercial name or ruxolitinib is the pharmaceutical name, and it targets a mutation called JAK or JAK2. And then we combine it with a chemotherapy we call, just called Vyxeos, which has also been recently approved specifically for secondary AML.
And so we’re taking that approach I describe where we’re combining the therapy that’s already on the market for secondary AML and has shown benefit, but then we’re adding this targeted agent which is also on the market for myelofibrosis but they haven’t ever been combined together. But we’re doing it in a way that’s slow and careful and cautious because, you know, we have to talk about safety, and we want our patients to get the benefit but not the toxicity. And we want to see what this does. The response rates for secondary AML from myelofibrosis are anywhere from zero to 15 percent, which is terrible. And so we really want to improve on that.
Andrew Schorr:
Okay. Let’s talk about transplant again. So, Don, you went through a transplant, and I interviewed a transplant survivor years ago who did well afterwards, but he said, Andrew, it is not a walk in the park. Now I know there have been a lot of refinements and you had it a number of years ago now, but what was the transplant experience like?
Don Armstrong:
It wasn’t a walk in the park. As a patient I think we hear what we want to hear, and I didn’t really hear about a lot of the side effects that might occur from having a transplant because I was so focused on just surviving. I thought I had actually skated through the GvHD, the host‑versus‑graft disease resistance, until probably day 30 or so, and I started having presentation on my skin, and I started having issues with my throat, my eyes.
So I had to go through all those additional struggles on top of fighting the initial treatment from the leukemia. So it was tough, and it was something that, it was‑‑it’s difficult to be prepared for that because you’ve already been through all the chemo for the leukemia. I was grateful that the stem cells did what they were supposed to do, which was graft in my body, but the side effects were difficult. They made it quite a challenge.
Andrew Schorr:
And you continue to take some medicine related to anti‑rejection?
Don Armstrong:
No, sir. I haven’t taken anything now probably for, for probably six or seven years.
Andrew Schorr:
Good. Good. Dr. Borate, just so we understand with a transplant. So you’re getting somebody else’s cells to‑‑you get the chemo to knock back the disease in your body, and then you’re having somebody’s healthy cells be infused to try to take offer the immune system. My understanding is even your blood type can change. I’m B‑positive. I get somebody who is O or something that becomes my blood type, right?
Dr. Borate:
Yes.
Andrew Schorr:
So it’s a whole rebooting and a changeover, right?
Dr. Borate:
Right. I think the one thing I do want to clarify is you mentioned you get a lot of chemo to take care of the disease in your body, but most successful transplants are actually done when you’re in remission, so when the patient is in remission. Because the chemo that’s given is really given to destroy your own existing bone marrow and immune system and to make a home and to make place for these new donor cells that will then come in and survive. If you did not do that then your immune system and your bone marrow would immediately reject any of those stem cells that were being given to the patient.
If you still have disease in your body and a lot of disease, and then you get all this chemo to prepare you for a transplant, the risk of rejection or the disease coming back is actually quite high because when you‑‑when you destroy your own bone marrow including bone marrow that has disease in it and then you infuse somebody else’s stem cells, you have a period of about two to three weeks when you have no immunity. And the stem cells are trying to grow, but if the patient still has leukemia in their body or had disease when you started this process, those disease cells just grow out of control, and they kill all the donor stem cells, and then it’s just not a good outcome.
So we really want the patient to have as less disease, preferably no disease in their body when they get a stem cell transplant. And this is not something that is intuitive to a lot of patients because it’s seen as a treatment for AML, which it is, but you really want to give it when the disease is really under really good control.
Andrew Schorr:
Amanda, could you talk a little bit about the support programs that the LLS, the Leukemia & Lymphoma Society, has, and Don I’ll have talk about it as well, so that somebody going through this, first of all they and their family have never heard of this before. They don’t know anybody with it. The treatments can be significant. Hospitalization could be long or not. How do you know you’re not alone? Not just calling you on the phone but there are other resources you have too.
Amanda Fowler:
Sure, absolutely. I think, like you said, just knowing you’re not alone it’s really important for people both caregivers and patients to connect with other people who have been through this. It’s very likely until diagnosis a person may have never heard of AML. They just have a vague idea of what leukemia is. So we have ways to connect patients and caregivers with other people.
We have a wonderful program called the Patti Robinson Kaufmann First Connection, and that is a telephone peer‑to‑peer connection. So we will match you with someone of a similar age and gender who has the same diagnosis and has already been through treatment. And they are trained volunteers. We also have in‑person family support groups. We have online support groups, including ones specifically for leukemias, one specifically for caregivers as well as young adults.
We also have an online community, so if you’re not available for the live chats you can post there and connect with people all over the world who have been through a similar experience. While every patient’s journey is really unique there’s going to be some similarities where you can know that you’re not alone. And I think all of these technologies are great because you can even connect while you’re still in the hospital.
Andrew Schorr:
And, Don, you are a First Connection volunteer. I’ve done it too, with people with my leukemia. So you talk to people one‑on‑one.
Don Armstrong:
That’s correct. By the way, I do want to add that my blood type did change because of my stem cell transplant.
Dr. Borate:
Of course.
Don Armstrong:
You’re absolutely right, yeah.
The First Connection program is probably one of the most rewarding things that I’ve done since my treatment and making it through that. To be able to talk to another person that’s going through something that you went through and really just answer any questions they may have is really very rewarding, and I know it’s great for the patient as well.
When I was going through treatment it was wonderful to hear the doctor say that you’re doing well, it was wonderful to have the support from the nurses, but I really wanted to talk to somebody that had gone through is so I could say, okay, tell me about it. What was it like? Did you have this problem? Did you have that problem? So it’s just a great one‑on‑one, as Amanda said, opportunity to help answer questions and hopefully give some additional encouragement to patients.
Andrew Schorr:
I want to add some additional resources. So, first of all, the originator of this program is a group called the Patient Empowerment Network. Their website is powerfulpatients.org in partnership with the Leukemia & Lymphoma Society. And we’ve worked with them many times at Patient Power.
Most recently, we did also a program at the big American Society of Hematology meeting, and that’s where all the hematologists, like Dr. Borate, from around the world come and where research is presented and they talk about it. This year there was a lot of AML, and so we sat down and had a discussion with peers of Dr. Borate’s, and that is Dr. Kadia from MD Anderson and Drs. Lee and Dr. Ritchie from Weill Cornell in New York, other NCI cancer centers like where Dr. Borate is.
And so I urge you to take a look at some of these programs. If you go to patientpower.info and just go to the leukemia and then the AML area you can see the replays of these. So that’s another resource for you.
And then I will tell you that some people are connecting on a platform like Facebook, and there are some AML groups there. A couple of caveats we’ll give you about the internet. I’m sure Dr. Borate warns people about Dr. Google. First of all, some of the information sometimes when you just search is not current. And you’ve heard for the first part of this program things are changing, right? And it’s very nuanced. And Dr. Borate may have a meeting with her colleagues tomorrow, and they’ll say, oh, well, now we know this. So there’s no way some of these services can keep up, particularly just general web searches. So be careful about that. That’s why you need to call Amanda because she’s staying on top of that.
The other thing I’d say is in Facebook you may go to an AML group, which is wonderful, people talking to one another, but we’ve talked about all these different situations in AML so one person’s situation may be different from another. So there may be general support, but remember, we talked about it at the very beginning of this program, you need to talk to your healthcare team to get a clear picture of your or your loved one’s situation and have a plan that’s, in this age of personalized medicine, is right for you.
And I’m sure, Dr. Borate, you have people come in sometimes with no information, but often you have people who come in who somehow have wrong information, and maybe you could talk about that, about how people‑‑how can they ask the right questions to get to the right answers.
Dr. Borate:
I think you’ve touched on such an important point because, as you said, patients are scared, their caregivers are scared. They’re looking for information. There’s information that they getting from the providers or the physicians, but obviously there is this need to help their loved one and so there is a lot of Google searching, joining groups.
Some of the big things that we see that are difficult is there’s a lot of information about supplements and alternative therapies, which while I think absolutely can help with many, many things including fatigue, nausea, feeling of well‑being, I think you have to be careful about what that resource is and what studies have been done on it. So I think those are questions that we get a lot and we try our best to have evidence‑based data on these different‑‑they’re like medications because they are something you’re putting into your body and while some are more natural than others, for example, turmeric is one that is used extensively by people. They do interact with some of the other treatments you might be getting for your leukemia.
So just understanding how your supplements are interacting with the treatments that you’re being prescribed I think is important, and there’s some resources that can actually do this in a very evidence‑based manner.
I think going to societies like the Leukemia & Lymphoma Society, we have the ASH, as you said, American Society of Hematology, ASCO, which is the American Society for cancer, these websites‑‑I think the NCI website, the national center for, you know, all cancers, they’re just really great resources that tell you what you’re up against in a very sort of patient‑friendly way that explains the treatment.
And I think something that you guys discussed before, the First Connection resource, I think that is something that‑‑I mean, it’s a resource that is so underutilized because even as a provider who treats AML for many years I don’t have the experience that Don had. I didn’t go through a transplant. I didn’t go through GvHD. So while I can discuss side effects I can’t really present a patient’s perspective, and that’s what they are looking for a lot of times. Just looking at a role model or reassurance that this is what a patient or somebody who looks like me went through and came out on the other side. And, yes, it wasn’t a walk in the park, and, yes, it was awful at times, but he came out on the other side and this was what he needed to do that. I think that is so valuable.
Andrew Schorr:
Right. Just one aside. GvHD. You hear all these acronyms. Graft‑verse‑host disease, where those new cells from somebody else are fighting with your immune system. They’re going to win because they did for Don, but it’s a fight, and you have side effects with it that could continue for a long time.
Amanda, what questions‑‑I know it varies by where somebody is in their journey with AML, but what questions do you suggest to people that they ask their doctor or a new center that they go to so that they or mom or dad get the right treatment?
Amanda Fowler:
Sure. I think that’s actually a big part of what we do in the Information Resource Center is talk to patients about what to ask the doctor. We can’t always answer their questions, particularly specific medical ones, but we help them get (?) Inaudible to rate that list out to what’s most important to them.
It really does depend on the (?) fees of where they are on their treatment, but an important question, something that Dr. Borate alluded to earlier, is what the goal of treatment is. Is my goal curative, or is my goal to extend my life for as long as I might be able to? Because people are on that borderline sometimes of fit and unfit, and they need to understand which type of drugs the doctors are prescribing for them and why.
And then of course the question of am I a transplant candidate, why is why not? I encourage people to ask why because they may think I’m a great candidate and the doctor says well, actually you know, your cardiac function is very poor and you won’t be able to survive a transplant. I think it’s good for peace of mind to understand the reasons doctors are making these decisions for them.
Andrew Schorr:
Amanda, what about second opinions? So even if you’re in a major city there could be one big hospital over here and one‑‑I think like New York. There are more than one NCI cancer center. So what about that? What do you tell people about that?
Amanda Fowler:
Absolutely. So AML can be unique in that sometimes there isn’t a chance for a second opinion upon diagnosis, right? Sometimes they go in and they need to start chemo right away. But generally there’s next phase. There’s a maintenance phase or there’s a transplant phase. Great time for a second opinion. I’m a huge believer in second opinions.
With the way treatment is evolving there’s options now. Really there used to be, like we said, for 40 years there was one option. Now there’s many, and so you want to be sure that you’re comfortable with your choice, comfortable with your physician because this is going to be a long journey. No matter whether you have transplant or not this is something‑‑it’s a long relationship, and so we encourage you to call us and we can help guide you to those centers of excellence. Even if you’re at one you may want to talk to a second one as well.
Andrew Schorr:
Okay. Dr. Borate, you are a specialist so you’re at a center of excellence. But how do you feel if somebody or a parent or family member says, you know, we’re going to go over here and see what they say. Are you okay with that?
Dr. Borate:
So I think any physician or provider who takes care of patients should really be okay with that because we’re all in this for the same reason and that’s to make our patients better and hopefully cure them. And I think the way we get there shouldn’t be something that you worry about what one person says versus the other. So absolutely.
I do agree with Amanda that sometimes for AML, and fact a majority of times for AML when the diagnosis is made time is of the essence, so unfortunately sometimes our patients don’t have the luxury of being able to go for a second opinion or get a second opinion simply because they’re so sick. They’re in the hospital. Their disease needs to be treated right away.
But I completely agree, once that first step is done I think taking a pause, talking about different options with your current physician and then saying, hey, do you mind if I go to the next center? So for example we’re close to Seattle or California. Just taking a flight and having a conversation and usually the second opinions really reassure the patients and the caregivers that they’re on the right track, and so they can come back to their original center and continue their treatment.
The one caveat I have to a second opinion before starting therapy‑‑or after starting therapy, rather, is once you’ve already received a therapy for AML you may not be eligible for a clinical trial for newly diagnosed AML patients. So we do have patients that come to us after having several cycles of therapy and then want to participate, and unfortunately the way trials are designed you want to get all the information right from the beginning, and so that’s when you enroll on a study when you’re starting your treatment.
With that said, I mean, it is what it is sometimes just because of the rapidity of the disease and the symptoms, but that is if you’re considering a clinical trial very strongly you may want to go to a center of excellence sooner rather than later.
Andrew Schorr:
One other point I want to make about clinical trials, because I’ve been in two, and about second opinions actually, is don’t drive yourself crazy. Dr. Borate, and maybe you go to another center, you go to Seattle up the road or California down the road. And then you say oh, no, now I’m going to the Mayo Clinic and then I’m going to Northwestern and then I’m going to New York, you will go crazy, and you will probably start hearing the same thing, right? As well as you went through a lot. So pick a team.
Amanda, any comment you want to make about that? Because I’m sure you have people, they’re very stressed out.
Amanda Fowler:
I agree. There is a number of second opinions that’s too many. But I also wanted to say on the clinical trial piece we at LLS have a wonderful service called the Clinical Trial Support Center that can help you find those trials. It’s a team of nurses who do individualized trial searches for people.
Sometimes that can help inform where you go. If you’re debating between California and Seattle and our nurses narrow it down and say, you know, there’s a trial in Seattle you could be interested in, it might help inform where you actually go for your second opinion. And you would reach them the same way you would reach me, through the Information Resource Center.
Andrew Schorr:
I want to recap just a few key points for our audience, and then get some closing comments from everyone. So, first of all, remember what you heard at the beginning. You want to, with the help of the Leukemia & Lymphoma Society, you want to get to where they’re really knowledgeable in this changing landscape of AML. You want to be tested or your loved one so that you know what version of AML you have. And if things are changing now that treatment has gone on for a while, retesting to say what’s going on now, and consider all your options.
And Amanda said it just a few minutes ago, so important, discuss your goals for treatment. What kind of life do you want to have? Do you want to just knock it back, take pills at home? Do you want to go to a transplant if you’re qualified for that? You know, these are all the things to discuss with a knowledgeable team.
And consider a clinical trial. And connect with Don or his peers around the country as First Connection people. So just a few key points. So first of all, Amanda, what do you want to leave people with? Probably call the LLS, right? Call.
Amanda Fowler:
Absolutely. You know, if you have any questions give us a call. We’re happy to help. We’re there to talk to you and help find those important resources that you need. And also just what a hopeful time it is for AML. When I started at LLS there had been nothing, and now, as Dr. Borate has said, there’s nine new drugs and (?) evolving quicker than we can almost keep track. So it’s a really hopeful time right now.
Andrew Schorr:
Okay. Dr. Borate, first of all, we all want to thank you to you and your peers around the world who are doing research, but a final comment you want to say to this audience and family members and patients who are so worried about this diagnosis.
Dr. Borate:
I do want to echo what other folks have said. This is a specialized disease, so reaching out to a center that has experience is I think critical. Even though we might not be able to physically travel at least getting advice from that center, whether it be through your physician or in‑person.
Secondly, I do want to say participating in clinical trials is a wonderful way not just to get treatment but also to get access to new drugs that could benefit you. And the other part of a clinical trial that’s never discussed is just by being in a clinical trial you are monitored way more closely and observed way more closely than if you were not on one just because that’s what a clinical trial mandates us to do. And I think the experience of our patients who are on it is always satisfying, whether the trial is helpful or not. Just the close connection and the follow‑up that they get is just‑‑it’s a great way to be supported through your therapy.
And the last point I want to make is something that Don said. You need a support system. You need your family members. Don’t be shy about calling your sister in Florida or your father or mother that live half‑way across the country from you. This is what family is for. Surround yourself with your family and friends. You need it. You don’t want to do this alone.
Andrew Schorr:
Right. And also remember for you as a family member, there is support for you too. Leukemia & Lymphoma Society can help. There are other groups. The cancer support community on Patient Power. There’s a care partner section and even with First Connection volunteers there may be some who can help the family as well.
Amanda, thank you so much for the work you do. Don, final comment from you because here you were treated in 2005 and then took medicines for a while, and you get to speak, but you learned lot today. Hearing all this what do you want to say to our audience?
Don Armstrong:
I just want to say just be encouraged by all the great new technology that’s come down in the last several years. Be encouraged by that. I also want to add to ask a lot of questions. Make sure that the time you spend with your doctor is quality time. Don’t let the doctor leave without a question being answered that’s on your mind. And third, use the resources that are out there that can really benefit you like the Leukemia Lymphoma Society IRC.
Andrew Schorr:
All great advice. I want to thank everybody for sticking with us. Remember, there will be a replay of this program. There will be all sorts of video clips. There will be a transcript. All that coming your way. So look for that.
Also look for these earlier programs that we’ve produced with support from the Patient Empowerment Network and with the Leukemia Society that really will even broaden your knowledge.
But we had some great information today. I want to thank our guests. I want to thank you for being with us. I want to thank the companies that have been devoted to research with the physicians like Dr. Borate who have been supporters of this program, Celgene, Daiichi Sankyo, Jazz Pharmaceuticals and Novartis for their support and devotion to the leukemia community.
And remember, please consider clinical trials in this fast‑changing field as they’re learning to combine medicines. Does that help you or mom or dad, grandma or grandpa, so they can live a longer, better life? In Los Angeles with our friends who have been in Colorado and in Texas and in Portland, Oregon, and wherever you are, I’m Andrew Schorr.
Remember, knowledge can be the best medicine of all.
Please remember the opinions expressed on Patient Empowerment Network (PEN) are not necessarily the views of our sponsors, contributors, partners or PEN. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.
