Acute Myeloid Leukemia: Your AML, Your Treatment, Your Decision

Acute Myeloid Leukemia: Your AML, Your Treatment, Your Decision from Patient Empowerment Network on Vimeo.

AML experts Dr. Pinkal Desai, Assistant Professor of Medicine at Weill Cornell Medical College and Assistant Attending Physician at the New York-Presbyterian Hospital, and Dr. Tapan M. Kadia Associate Professor, Department of Leukemia at The University of Texas MD Anderson Cancer Center, share research-based insight on how AML is diagnosed, including the symptoms and recommended tests, and disease management strategies.
 
These experts give an overview of currently approved AML therapies and share clinical trial updates on treatments in development. The panel discusses AML management and how you can ask questions and talk to your doctor to feel confident with your care. Additionally, you will hear from an AML patient who shares their experience and advice for approaching the decision-making process.
 

What Are the Current Treatment Options for AML?

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

Downloadable Program Guide


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.

Coping With the Emotional Side Effects of AML

A dynamic panel discusses the important aspects of care for AML patients, with a focus on the emotional side effects, research on new treatments underway and finding suitable clinical trials.

Downloadable Program Guide


Transcript:

Beth Probert:

Hello, I’m Beth Probert, and I am a patient advocated and ambassador with Patient Power.

I am also an MPN patient. Thanks for joining us for this Patient Empowerment Program in partnership with The Leukemia and Lymphoma Society. Today, our program is: Coping with the Emotional Side-Effects of AML. And we are joining our AML community.

We’re gonna focus on where we’re headed with treatment of Acute Myeloid Leukemia; what patients can look forward to in the coming year.

We will also answer questions that you can submit to AML at patientpower.info. And please note that we cannot provide specific medical advice over the internet. And it wouldn’t be fair to you. We always recommend that you seek care from your own doctor or AML specialist, and that’s how you will get the best treatment for you.

I’d like to start now and introduce our panel. And we’ll start off with Dr. Thomas LeBlanc. A medical oncologist, palliative care physician, and patient experience researcher from Duke Cancer Institute. Welcome, Dr. LeBlanc.

 

Dr. LeBlanc:

Hi, thanks for having me.

 

Beth Probert:

And I would like to introduce Michelle Rajotte. She is the Associate Director of the Leukemia Lymphoma Society’s Information Resources Center. Michelle has been with LLS for 13 years. Hi, Michelle.

 

Michelle Rajotte:

Hi, good to be here.

 

Beth Probert:

Thank you. And last but not least, I’d like to introduce our patient advocate today, James Bond. And James has survived Multiple Myeloma for 27 years, and AML for the past 7 years. James and his wife, Kathleen, have shared their story in 29 states. And patients can contact him directly at his email, which is Jim.Bond48@gmail.com.

So, thank you for joining us today, Jim.

 

James Bond:

Happy to be here.

 

Beth Probert:

Great. Well, Dr. LeBlanc, I would like to start with you. Tell us a little bit about your background in AML and palliative care, please.

 

Dr. LeBlanc:

Sure. So, by training I’m an oncologist. But when I went through my cancer care training, I realized that oftentimes we fail to really attend to some of the issues that are most important to patients and families. And those might be things related to symptom burden, quality of life, emotional well-being, communication, understanding of prognosis.

And so, I ended up pursing additional training in palliative medicine where those types of issues really are the focus. And in doing so, got a sense that really adding specialist palliative care to cancer care and blood cancer care particularly, really could improve many aspects of the experience for patients and families.

And ultimately that is what my clinical practice and research have come to focus on. But in my clinical practice, I largely see patients with myeloid malignancies, including Acute Myeloid Leukemia and some related conditions.

 

Beth Probert:

That is so interesting and very unique because very often we see our doctors and we don’t get the whole palliative side of it. So, I can honestly and personally say that that is just a wonderful added bonus. Thank you.

And Michelle, can you tell us a little bit about your role at LLS, and really what the goal of the information resource center is?

 

Michelle Rajotte:

Sure. So, I’m part of the Information Resource Center at Leukemia Lymphoma Society. Which, The Leukemia and Lymphoma Society’s main goal is to help find a cure for Leukemia, Lymphoma, Hodgkin’s disease, Myeloma, and improve the quality of life of all patients and their families.

And IRC is apart of that. So, basically, what we do is – it’s staffed by information specialists who are master level, either social workers, nurses, or other healthcare professionals who’ve been trained in blood cancer. And we can do anything from answer questions, provide disease information, help with clinical trial searches, find different support resources, refer to other organizations if we need to for other resources. But really, it’s anything that someone needs in the moment.

So, we’ll talk with them over the phone, through email, or through chat online, and we figure out what it is that they need based on talking to them, or whatever they provide to us. And then, provide them with the resources and support they need.

 

Beth Probert:

Wow. And that is just invaluable. And we definitely need to bring focus to the cancer patients and what your department could ultimately provide to them. Thank you.

So, Jim. You are a long-time survivor. How has your cancer diagnosis impacted you emotionally?

 

James Bond:

Well, it’s been like riding a roller coaster. My care-giver wife, Kathleen, Kathleen is sorry she can’t be joining us today. But the lowest point, of course, was getting diagnosed with a deadly incurable blood cancer. My first one, Multiple Myeloma. And then the second one, AML, many years later.

And so, what we tried our best to do is, to try to even out that roller coaster ride emotionally. And, I’ll give you an example, after 10 years of dealing with Myeloma, I was told, Jim, there’s nothing left that can help you, you need to go to a hospice. And that was obviously crushing.

And what we tried to do is pull each other up and say, “Look, we’ve been through tough spots before.” And we figured out that just rely on the doctors, rely on our own research ability, and they’ll be something coming up. And we were able to figure out, hey, there’s a clinical trial that was mentioned to us, and within a month of being told to go to a hospice, we were out of town in a clinical trial, and within two weeks, I was told, “You’re in remission.”

So, that was a tremendous high. And again, what we try to do when we get really good news is pull each other down and try not to be so excited, but we try to even things out. And that’s very difficult to execute, but for 27 years now, we’ve had a good deal of experience. There are a few other tings we do emotionally, we say, “Look, let’s do all we can, and then let’s not look back and second guess ourselves.”

And even to make it more normal, we cut off all cancer discussions with ourselves, ideas, or with a family member at 8:00 p.m. our time. We say, “You know what? Let’s just do what we’re gonna do at night, and let’s defer that to the morning.” That tends to let our emotions calm down and let us live more normal lives. At least in our minds.

It has not been easy. It’s been very difficult and emotionally at times, we’ve actually played a role of trying to lift up the medical team who, the AML diagnosis in particular, they explained to me, “Jim, you’re 64 years old,” When I got AML, that was seven years ago. They said, “Your chances of survival are not good. The only way you can live is through a fourth bone marrow transplant. And this one has to be not from your matching sister, but from an unrelated donor, if we can find one.” 

So, they really encouraged me to consider just hanging up, but our approach, and this helps us emotionally is, no, we’re gonna treat this thing called cancer like a problem. We’re gonna put it in front of us, and we’re gonna deal with it as analytically, or unemotionally as we possibly can. And lo and behold, the doctors, as they’d come around in my, I don’t know, 10-week stay in the hospital, whatever it was, they would keep trying to say, “Jim, don’t get your hopes up. This might not work out.”

And it did work out, and we found ourselves much better off by, I do my favorite thing, and that is, I make myself exercise each and every day. And sometimes that exercise is not much, it’s walking with my IV pole around the floor section when doing a transplant.

Or it’s walking on my treadmill on snowy icy Ohio days like today. But that helps me emotionally because it gives me something that’s not cancer, it’s quiet time to think, and it really led to something that’s been just magical in terms of helping both of us emotionally.

When I had to leave town to do the clinical trial, my wife, Kathleen, got to thinking as a long-term volunteer of the American Cancer Society, she realized that there are not enough people in the country aware of these things that the ACS has called, “Hope Lodges.” So, she founded, launched, and leads, to this day – this was 13 years ago, she launched the first one. And I was not a cyclist, but I saw a link between the exercise that I think is so vital for me emotionally and physically, and this bike ride. So, I decided to buy a bike and trained. And I’ll be darned, I’ve ridden it every year four days, 328 miles from Cleveland to Cincinnati.

 

Beth Probert:

Wow, well that is really inspiring.

 

James Bond:

Thank you. And that helps me tremendously emotionally because that training and riding takes up a good three and a half, four months of my year, and I look forward to that, and the fundraising is tremendously exhilarating because I get to hear from people that I don’t hear from that often.

 

Beth Probert:

Yes.

 

James Bond:

So, the key there is emotionally, I think, is just having a long-term plan, and not letting –

 

Beth Probert:

And Jim, I’m just gonna jump in really quickly, this is amazing information. So, hold that thought, we are going to jump on some of the thoughts you said, and I do want to say real quickly, I love the, “We” in that. We.

 

James Bond:

Oh, absolutely.

 

Beth Probert:

And we’ll click back onto that. So, I’m gonna hop over now to Dr. LeBlanc. And could you go through, with your vast experience, what are the key emotional side effects that you see your ALM patients facing day to day?

 

Dr. LeBlanc:

Yeah, this is such an important question, and it’s one that we don’t ask often enough, and we don’t talk about these issues very often, unfortunately. So, I’m really excited that we’re having this webinar, first of all. And I’ll tell you, it’s important to recognize as well, every patient, every person is different. So, there is not one quintessential AML experience. That’s really important to recognize.

But at the same time, when we have studied this issue and interviewed patients, and care givers, and family members, there certainly have been some common themes that have come through about people’s experiences. And one of the one that is, I think, particularly important to recognize is the sense of shock at this diagnosis. Now, acute leukemias, we call them acute because they tend to come on very quickly and suddenly. 

And many of the patients we see will say things like, “I was fine three weeks ago. And now I can’t even walk up a flight of stairs.” And, “I’m so tired, I’m taking naps, this is not like me. I usually run marathons, and now I can only run a couple miles, something is going on.” And this really degenerates, for many, people over the course of day or a few weeks.

And sometimes it means they end up urgently in the hospital and are told, “You can’t leave. Something’s going on, we don’t really know what it is, but we’re concerned. You might have leukemia.” And if they’re not at a large medical center, they may get shipped off hours away from home to a place that’s not familiar where they don’t know anyone.

So, that shock and suddenness of the diagnosis makes everything else much more difficult, and it sometimes creates, even, social isolation related to where AML is treated. Where it tends to be treated more so at academic centers than it is in the community, although, certainly, some of these treatments are provided in the community.

But patients getting high-dose therapies do tend to come to large research centers. So, we’ve certainly seen that issue impact many patient’s experiences. The other one that comes up quite often, that really compounds the decision making and the emotional difficulties, is the issue of uncertainty. So, unlike many cancers, we really don’t know what to expect when a person is diagnosed with AML. And everyone asks, “Well, what stage is this?” and we don’t really have stages for this disease.

We, certainly, have ways that we can try to get a sense for what we might expect for the patient who’s sitting before us. And we do all kinds of fancy testing, and we talk at length about those issues, but at the same time, we really can’t say what’s going to happen to you, my patient sitting across from me who I’m trying to help guide through the process.

And there are actually a lot more risks associated with Leukemia treatments as you heard Jim talk about. A stem-cell transplant is a difficult and risky process, and sometimes that’s part of curing AML, or hoping to cure AML. But even high-dose chemotherapies in the hospital, some people actually do have really difficult complications, and even can die from those treatments, and yet, those are the treatments that usually are required to cure a person.

So, we have to have these difficult decisions made sometimes under a lot of distress emotionally, and amid the suddenness of this diagnosis, where we say, this is probably the best treatment for you, and it gives you a chance at cure, but it’s not a guarantee. And some people end up not making it out of the hospital. And usually what happens is, that’s just such difficult information. Many folks shut down and they say, “I don’t know, what should I do. Tell me what to do.”

Or they’ll turn to a family member or a friend who might or might not be around and available during that difficult time, especially if they’re in another city away from home. So, these are some of the things we’ll commonly see when patients are newly diagnosed with AML.

 

Beth Probert:

Wow. That is very intense. And there’s obvious emotional connections. And sometimes we hear someone’s diagnosed, and we completely forget that emotional side. So, I wanted to ask you, as well, you’ve been involved with research into the relationship between the emotional stress in AML patients and the overall prognosis. Could you please explain how the study was conducted, and what were some of the prevailing results of this study?

 

Dr. LeBlanc:

Sure. So, we did a longitudinal study of patients with AML who were being seen and treated on our in-patient service. So, these were mostly patients getting high-dose chemotherapy who would be stuck in the hospital for a month or even a bit more.

Like Jim described, getting these really intensive treatment regiments. And this was a study basically aimed at helping us better understand what people go through when they live that. And certainly, I’ve seen that in caring for many patients with AML, as have our nurses, and other members of the cancer care team, but actually, there has been very little formal, objective study of the patient experience with AML and related blood cancers.

So, what we did is we actually surveyed patients using validated instruments, and we assessed their symptoms, their quality of life, their overall distress levels, and in addition, we assed their understanding of their illness. Their understanding of what we call, “prognosis,” The idea of what the likely outcome of the treatment or the disease is going to be. And we did all of those – that whole battery of assessments every week when they were in the hospital, and then when they were out of the hospital, we did that every month.

And we followed patients for six or even upwards of 12 months, and different things happened. Some people went into remission and were cured, some people had relapses, some people went into transplant, some people had transient remissions, or even multiple relapses, got additional treatment. And by following patients over time, we were able to develop a profile of the patient experience with AML and look at different versions of that. Including, what people understand about their illness, and how that relates to their overall emotional well-being.

 

Beth Probert:

That is amazing. And was there something that just jumped out real quickly as far as the largest response rate you saw when people were taking care of that emotional part?

 

Dr. LeBlanc:

Well, the concerning thing that we found, which unfortunately is an issue across all of cancer care is that many people who are diagnosed with AML, especially when newly diagnosed, really don’t have a good understanding of the likely outcomes.

And it’s certainly not for lack of talking with patients and families about these issues, but it probably is a manifestation of the fact that this usually happened suddenly, as I mentioned, and it’s so emotionally overwhelming and difficult that it’s actually really difficult to contextualize the information that’s provided. And we, I think, end up overwhelming a lot of patients and families with so much information, that sometimes there’s a bit of a forest and trees problem, where maybe the most important factors don’t always get explained clearly or don’t come through well.

And we don’t always go back and check in about whether we did a good job of explaining things, which is unfortunately a shortcoming that most of us struggle with in taking care of patients. Communication of complex information is very difficult. 

So, we found that many folks didn’t understand, for example, that the treatment they were receiving maybe wasn’t likely to yield a cure, which is true in some instances of AML. Or they might not have had a very good understand of the risks. So, one study, for example, suggests that AML patients may actually think the treatment is way more risky than it really is. And is that prompting some people to not receive intensive treatments that maybe could be the right choice for them and the most helpful for them. So, that was the one main interesting finding.

And then, related to that, unfortunately we also found that many patients who do come to more accurately understand the outcomes that are most likely with their particular situation, some of these are better than others, everyone’s different, the more accurately people understand their illness, there tends to me more emotional distress and sadness. Perhaps realizing that this is a very difficult disease to treat. And unfortunately, when we were doing this study, this was before we have eight new drugs approved in the last two years. 

So, hopefully some of this has changed. But that’s why we’re having this webinar, and why we need to talk more about these issues.

 

Beth Probert:

Absolutely. So, now, Michelle. I definitely see that your role at LLS plays a huge part in this. And in your experience, how do you deal with this? What resources seem to be the most effective that you can provide patients in coping and the emotional side of this cancer diagnosis, and also, taking parts of what Dr. LeBlanc just said, I would love to hear from you now and what your role is.

 

Michelle Rajotte:

Sure. I think it’s different for different people. We’ll talk to people who want to know everything, and we’ll talk to people who just say, “Just tell me the basics, I can’t get overwhelmed right now.” And it also depends on where they are in their cancer journey. Are they just diagnosed; are they relapsed? I think a big piece is being able to talk to other people who can relate to what they are going through.

So, other people who have been through this already and have gotten to the other side and feel like, okay, it can get better, there is hope. Here’s some things that might help. Because unless you’ve been through it yourself, I don’t think you can completely understand. You can empathize, you can be there for someone, but your friends and your family may not be able to give you that support that someone could that has been through it.

So, for example, at LLS we have the Patty Robinson First Connection Program, which can help over the phone to be able to talk with someone. And they may be across the country, but they may be very similar in background to you and be able to answer some questions that you have while you’re going through things.

There’s an LLS community where you can go on and talk with people. We have a lot of online support groups. There are online chats that are set up to be able to talk with, again, others, it could be across the country, they’re people you may have never had the opportunity to meet. Or if you’re not doing well, or you’re in the hospital, or your immune system’s compromised, you still can reach out and get that support.

And it’s not going to be something where you physically have to go somewhere, but there’s those options too. Or someone may not be ready to go sit in a group and talk about what they’re going through but can sit in front of a computer and just say, “All right, I really do need to talk to someone.”

Also getting professional support from a social worker, or a counsellor, or just anyone who can help you get through this because it is extremely stressful. And some people think, “Oh, I really don’t need that.” But it may be exactly what you need just to help you get through it.

Also, pulling in friends and family. And again, sometimes they may be more stressful because they don’t completely understand, and even though they’re trying to help sometimes it may have the opposite effect, but the intention is there, and it’s good to have them there, even if it’s just to drive you to a doctor’s appointment. And help you understand what the doctor’s talking about.

 

Beth Probert:

Wow, that is very impactful. It sounds like you really give the patients a complete tool kit as far as how to have these conversations and the unbelievable amount of resources that are available to them. So, that is invaluable. Thank you, Michelle. We’ll get back to you again.

And Jim, going back to you, did you reach out to your doctor in regards to this whole emotional turmoil, and you said earlier the, “Roller coaster.” Was that a talking point with your doctor, by the way, on how you’re feeling and how to cope? What direction did you take when you were first diagnosed, and was your doctor part of the conversation?

 

James Bond:

Well, we’ve been very blessed, very lucky. My first doctor who diagnosed me, he really helped me by answering this question that I asked.

[00:27:30]

And asking questions is a good way for me to relive stress and gain information, like the kind of information that Beth and Tom talked about.

When I was new to blood cancer, I said, “Doctor, now, if you were in my shoes, whom would you have treat your case?” And frankly he was shocked because he was at a leading cancer institute in my hometown here, in Cleveland Ohio, and he gave it real thought. And his compassionate answer blew me away. He said, “Jim, the professor who taught my blood cancer course at the medical school works in another hospital. Another leading cancer institute here in Cleveland. And if I were in your shoes, I would go to him.”

Now, what I did – and for 10 years, until he retired, that man helped us, my wife and I, emotionally and medically in more ways than I could ever describe. 

An 8:00 phone call one night, and which we had never gotten from him, his name was Bob and of course, it’s a doctor, it’s an oncologist, I’ve got a deadly cancer, he’s calling at night. I’m thinking, “Oh, my god. The world’s coming down.” He really relieved our stress, he said, “Jim, those shoes you had on at your last appointment, may I ask you where you got those?”

So, like you said, Tom, each case is unique, and in our case, stress has been relived in some very unusual ways. I got in a car accident after my first of four stem-cell transplants, and my wife was having real problems with stress because now I was in remission and seemingly home free until it came back five years later, but she was really stressed out until I had a car accident where, not my fault, but somebody t-boned me and it really was a tough accident.

I was okay, but the car was wrecked. But when I called her to tell her that, she flipped out. And all this pent-up emotional stress she was going through came out, and it manifested in her yelling at me, how could I possibly have an accident after all we’ve been through? And the thing is, she caught herself, she listened to herself, and she realized, oh my gosh, what’s the point in getting yourself all in a knot over your incurable deadly cancer? You can get taken out by a car accident as any time. Things like that.

 

Beth Probert:

Absolutely. So, she really put it in perspective for you, didn’t she?

 

James Bond:

It really did. It really did. It just happened, it was coincidental that it happened, but it did. And so, we’ve used that. 

Another thing that really helps us with stress is, and this is gonna blow some people away, but the longer we’ve survived with these two cancers, the more we’ve gotten asked to share our story around the country. And if fact, in two countries overseas.

And here’s the thing. We realized from the very first story telling we did in our home town, how much telling our story helped us emotionally. We looked at each other when the couple left our house and we realized, oh my gosh, just sharing our story and the roller coaster parts of it, not the technical parts, but just the emotional part, that really helped us. And so, we welcome other opportunities, and we encourage other survivors, whether it be short-term or long-term survivors, to consider the kind of things that the LLS has, and other organizations that get us out there, get people out there to share your story. It is very helpful for us. And that was a huge surprise to us.

 

Beth Probert:

Well, that is wonderful. And Michelle, I’d like to come back to you for a moment. Do you have resources that you can provide to caregivers and patients with AML, that if they do want to share their story, and is that part of what you do, as well?

 

Michelle Rajotte:

Yes. That is part of the Patty Robinson First Connection program. What it is, it’s trained patient volunteers and family members who’ve been through this, who then want to be able to reach back out and help others who are going through it now. So, that’s one of the things they can do. They can volunteer at their chapters, and there’s always a way to get involved that way. There’s things on our website where people can share their story. There’s lots of different things. On the LLS community, there’s a way for them to be able to post what they’ve been through. There’s blogs that we do. There’s tons of different things. And as far as the care giver, Jim, you bring up a good point, they are going through a lot of stress, as well. And they need just as much support.

And we do have a lot of good caregiver resources now. We have a caregiver workbook that we can send out that has everything you could possibly need as a caregiver to know. And it’s divided into sections, so it’s not overwhelming, but it’s a way to have a roadmap to try to figure out, okay, what do I do? Because just like the person who’s been diagnosed, the care giver gets thrown into this and doesn’t know, what do we do; where do we go; what questions do we ask? I don’t even know where to start.

And a lot of times that’s the question we get at the IRC is, “I’m calling you, but I don’t even know why I’m calling you because I don’t even know what I’m supposed to know.” So, it’s really helping people try to figure out, what is that next step? And that’s really all you have to focus on. If you try to look at the big picture, it can be really overwhelming. But if you can get to the next step, that’s something that’s doable.

 

Beth Probert:

Wow, that’s wonderful to know you folks do provide those resources. Thank you.

So, Dr. LeBlanc, I’d like to shift over to managing fear, anxiety, depression. So, you mentioned a few times that being diagnosed can be so overwhelming, and we can’t ignore that this could lead to anxiety and depression. What sort of things do you recommend to your patients to allay these fears, and to put into place in their life in dealing with this? It’s obvious that for most people it is going to lead to the anxiety and depression.

 

Dr. LeBlanc:

Yeah, this is such an important question, and it’s a really difficult one to address. Most of the time, I do recommend that people talk about it. And sometimes that’s the most difficult thing to do even though it sounds obvious, but it’s often the elephant in the room.

So, many times, the doctors and other clinicians seeing patients with AML and other cancers are just so incredibly busy and also fixated on all of the medical details, and the labs, and the scans, and other treatments, complications, doses of chemo, all of these things that we need to be focused on, of course, that we forget about the person, and the way that they’re struggling with these issues.

And it’s not that we don’t care or that we’re bad people or anything, it’s just that’s never the number one priority when you have to get all of the details straight to make sure the person gets the right treatment. I try to ask, but sometimes we don’t do this. So, for example, if there are other clinicians listening and wondering how to do this, one thing that I do is to just say, “This is really difficult to go through. How are you holding up? What do you look to for strength?”

And I will ask the person there with them. Usually patients aren’t there alone, and typically the person who’s with them is the person who’s really helping them keep it all together. Whether it be a spouse, a family member, a caregiver, a friend.

And I usually turn to them and also ask them, “How are you holding up? What are you seeing? What is the patient not telling me?” You know? What are they going through that they’re – sometimes people will put on a brave face for the doctor and they won’t tell me how much they’re suffering at home, and I really need to know so that I can help.

So, really the best recommendation is open and honest communication. And the other great one is to seek out resources like the ones we just heard about. I sometimes will encourage patients to seek out a family therapist, a psychologist, somebody who they could see in the cancer center, a social worker, someone who they can sit down with for an even longer period of time and just talk about how difficult this experience is. And just talking about it sometimes is really some of the best medicine, honestly.

 

Beth Probert:

Wow. Yeah, I love the tie in to with making sure that the caregiver is doing okay, as well.

 

Dr. LeBlanc:

Yes.

 

Beth Probert:

We often look at them as a pillar of strength and forget that they need those resources.

And one of the things that I personally feel is really really helpful, and I’ve heard from the AML community as well, is the mind, body, soul; exercising, meditation. And Michelle, I wanted to ask you, is that something that, when you talk about resources, that your department provides? Do you find that that’s a very often asked question, and/or it’s a topic that you like to recommend to patients?

 

Michelle Rajotte:

We get a lot of questions about, what can I do? How can I help myself get healthier, or stay well? Or how can I help myself get stronger, or what can I do? And I think a lot of it is, you feel very powerless when you’re diagnosed, and you really don’t have any control. So, if there’s something you can do to feel like you’re taking part in your care and making decisions about some things you want to do, that’s great. We always say, “Check with your doctor to see what’s okay; what’s safe for you to do right now depending on how you’re doing.”

But we have a lot of resources on nutrition, we have a nutritionist that can do a consult, either over the phone or online through email. We have a lot of different resources, and webcasts, and podcasts, and videos, and we have a ton of resources.

It can be a little overwhelming just to go on to the website and try to figure out, okay, what do I look at? Where do I go? So, I would encourage them to call the IRC. We can walk you through, depending on what it is that you’re looking for in the moment, where to find it. How to bookmark it, so you can find it later.

But again, I think it’s really important to discus it with your doctor to see – obviously if you wanna go for a walk, and you’re okay to do it, that’s great. But if there’s a chance that your platelets are very low, and if something can happen, then you gotta check with them about that too. So, we’ll get an idea of what they can do, but we always send them back to the doctor, make sure whatever they decide to do, whether it’s supplements, again, check with your doctor because they can interact.

But anything they want to do to help themselves get stronger or take care of themselves is always a positive.

 

Beth Probert:

Wonderful. And, Jim, I’d like to ask you to stay on this topic for a minute. Could you give any advice about support groups?

 

James Bond:

Yes.

 

Beth Probert:

Is that something that you found to be a great resource in dealing with this kind of anxiety and depression?

 

James Bond:

Yes, I think support groups are for people who want to go to a support group. Put yourself back 27 years ago when we first we introduced to blood cancer. There were not a lot of support groups available. And we started out with keeping it more to ourselves and our family, and then as we grew comfortable with living an managing the fear, the risk, the anxiety, our circle spread out. But it really was not until we got invited to share our story that our eyes were opened of the power of support groups. And we could see it happening.

One other thing that, Tom, I’d like to mention to you, one of the most effective way to manage fear for my wife and I was late one Sunday night lying in week number six, or something, in the last transplant for AML, and I’m on the ropes, I’m in tough shape. And the phone rings, and it was my myeloma doctor from Boston where we go twice a year, his name’s Paul Richardson, he’s an outstanding, compassionate doctor.

It was Paul, and Paul said, “Jim, I’ve just talked to your wife Kathleen at her home,” she had just left for the night. And he said, he said to her what he then said to me. He said, “Jim, I know you’re in a tough spot, but I want you to know, that we’ve got other patients here at Dana Farber, who have been through exactly what you’re going through, myeloma followed by AML, bad, bad prognosis.”

And he assured me that I could do it. And, Tom, what that doctor’s phone call meant to my wife and I could have been the difference between getting through this thing, and not getting through it. Giving up, and not giving up.

And we really believe it’s because our doctors have taken the time to help us build a relationship with them. Knowing how busy they are, and how many patients they have, we found the world of oncologists and the nurses and the others, very compassionate people. And it’s worth that time to build that relationship whether it’s your ongoing doctor, or one that you go out of town for a second opinion with, those relationships mean everything. And the doctors who are willing to take their time, when it’s not really on the clock and help their patients, they are doing tons and tons of good for the world that we live in.

So, we’ve got some other techniques, but those are the things that really stand out to me in terms of managing in this area.

 

Beth Probert:

That’s wonderful, Jim, and it circles right back to you, Dr. LeBlanc, when you introduced yourself and you told us that there is just more than coming to the doctor, and reading the chart, and giving the blood results. It’s definitely very impactful. And what you spoke about earlier about how you bring in the palliative care and the emotional care. And on that note, I know this is a little cross-over, but can certainly add to anxiety and depression on everything that we’ve talked about, Dr. LeBlanc, but do you encounter, through your care and conversations with clients, their anxiety over the financial part of care? Is that something that you hear often?

 

Dr. LeBlanc:

Absolutely. The idea of financial toxicity, sort of like other kinds of systemic toxicities you would have from chemotherapy, it’s just as real as a patient who gets neuropathy from their chemo. And in some cases, may be more crippling.

One of my colleagues here at Duke is a leading researcher in this area, and he’s taught me a lot about this, and unfortunately, I’ve seen it a lot in my clinic. And as we are fortunate to have a number of new therapies available for AML and other diseases like multiple myeloma, the unfortunate aspect of this issue is that many of them are pills, and may states do not have parity laws in place that require insurers to treat pills the same way as they do chemo therapy that you would get in an infusion suite.

North Carolina is unfortunately one of those, where I practice, where we still don’t have a law. 

And that’s sometimes means I’m talking to a patient about an exciting new therapy, and then I find out that their monthly copay is going to be $3,000.00, and who can afford that? That’s just the copay amount for the patient just for one month of medication. This is, unfortunately, happening a lot. And thankfully, there are many resources that we can engage to help patients with these issues, but it is an increasing problem as medications are more sophisticated, they also have gotten much more expensive.

 

Beth Probert:

Yeah, and we hear this so often. And, of course, Michelle, I’m sure you’re hearing this, as well. And your department can direct people to the appropriate resources?

 

Michelle Rajotte:

Yes. It’s something we hear every day, unfortunately. Like Dr. LeBlanc was saying, we’ve got all these great new treatments now, but so many of them are oral, and a lot of patients, if they’re on Medicaid/Medicare especially, their copay is extremely high.

We do have copay assistance through LLS, we will also refer people onto other organizations that have assistance if we know of them. So, anywhere we can get people to get the help. We also do a lot of advocacy on that end, and we’re in Washington a lot and we’re sharing a lot of patient stories, and we’re trying to get the word out there that we shouldn’t have these barricades to treatment. We do all this research, we find all these wonderful treatments, and then people can’t have access to them. And that shouldn’t be.

So, that’s one of the things, along with the research and the patient assistance we have, we also focus on the advocacy part, and making sure that the oral parity bills are passed in hopefully every state. And that things are little bit more on an even plane, so people can use these wonderful treatments that are coming out.

 

Beth Probert:

Wonderful. And Michelle just hit on treatments, so Dr. LeBlanc; I would like to now go back to you. 

And could you tell us, in regard to treatments, advances in clinical trails for AML, what’s happening in research and should patients be hopeful?

 

Dr. LeBlanc:

Yeah, it’s really a very tremendous time in cancer care and in biomedicine in general. As I mentioned earlier, we had, if I remember correctly, eight new drugs approved for AML in the last two years. And we had been mostly using the same treatment for patients for the prior 40 years. The seven plus three induction regiment was developed in the ’60s or early ’70s, and mostly that’s the same regiment or related ones to it that we’ve been giving to people when we give them high-dose therapies for this disease.

Other things have improved during that time, as well, that are really improving outcomes, so we have much better supportive care medicines. We have growth factor injections that work better. We have better antibiotics. We have anti-fungal medicines that work a lot better.

So, when you add those developments, even to the old chemotherapy, that had improved outcomes prior to this spirt of approvals in the last two years, but now, especially, we really are in a new ear of how we treat AML. And now, we need to actually molecularly and genetically profile each individual patient’s leukemia, so that we can best know how to treat their disease because at this point, we have several testable targets that we might then prescribe a medication to address in an individual person’s case of AML. So, it’s getting more complicated, at yet at the same time, there are many more options, and it really is a time to be very hopeful about how things are going.

 

Beth Probert:

That sounds so encouraging. And Michelle, going back to you. How can you lead clients and their care givers to these clinical trials that are on the horizon? 

And can you talk a little bit about what that process looks like?

 

Michelle Rajotte:

Sure. If someone reaches out the IRC, we do have a group of nurses who do clinical trial searches specifically for blood cancers. And it’s not just, we’re gonna hand you a list as say, “Here, go talk to your doctor.” They will help through the process. So, they’ll really in-depth dig, and try to find trials that might be an option. Have you go back to your doctor, but then walk through it with you to help you get into that trial.

Because there’s so much research now, it’s wonderful, but it’s also really overwhelming if you try to do it by yourself. And a lot of them are more focused trials now, so you have to know what kind of mutations you have and that kind of thing. So, it’s a partnership where there’s a form that you would need to fill out for us to have that information, and then we help you walk through that process of, is there a trial that’s out there for you; is it something that’s appropriate for you, along with your doctor. And then, how do we help you make sure that you can get through the whole process.

 

Beth Probert:

Wonderful. What a fantastic resource. Thank you.

So, I would like to take a few questions that we’ve received, and, Jim, I’d like to hear your feedback on this one. And the viewer asked, “People keep asking me how I’m doing, and it just makes me worry more.” Do you have any advice, Jim, for people to tell those that love them and just want to help them that all these questions are causing anxiety, what would you suggest, Jim?

 

James Bond:

Yeah, a couple things that I’ve found useful. I explain to them I just got done with playing nine holes of golf, or I just got done exercising, and I’m quick turn it back and say, “How are you doing?” and try to get as much out of the other person, so that they understand that I’m comfortable in my skin, and I’m not stressing out or how things are going.

But it’s easy for people to understand that, hey, this guy’s got an incurable deadly blood cancer, or two, and we worry about him. So, I try to just loosen up, and turn it back on them, and hopefully they get more reassurance that, hey, the guy’s not stressing out, he’s okay. But you know, once you do all you can do, the rest of it is just fate, luck, whatever. So, that’s what we try to do.

 

Beth Probert:

I love that response. And people mean well, but putting the focus back on them is just fabulous. That’s really great, thank you, Jim.

 

James Bond:

Oh, you’re welcome.

 

Beth Probert:

And Dr. LeBlanc, we have a question from Shannon from Boston, and her questions is, “How can I manage the daily stress of like with AML? Are there proven strategies to cope with the stress?” So, we did talk about a few things earlier, but what advice would you give Shannon?

 

Dr. LeBlanc:

Yeah, I’m not aware of proven strategies specifically for AML, which is part of where we all struggle. Not knowing what to do and how to best support individual patients. And as I mentioned earlier, every individual is quite different. but I usually recommend meeting with a professional to talk about it. And some people are opposed to that and they don’t want to do that, but more people are at least open to the idea. And so, Shannon, if you’re somebody who’s open to that idea, I would actually encourage you to seek out a specialist in palliative medicine.

And many people misunderstand what that means. So, I want to just take a moment to explain why I would think that’d be helpful and what the evidence shows. So, clinicians who are trained in palliative medicine are basically experts on well-being. 

They know how to address symptoms, they know how to help with quality of life maintenance, and they know how to help people cope with difficult diagnoses and serious illnesses like cancers. Regardless of the expected outcome. So, they can be helpful if we’re aiming at cure and we think there’s a really good chance of that, and they can be helpful in cases where we know that’s not gonna happen, and anywhere in between.

So, one of the misconceptions is that they can only be helpful when people are dying, but actually, what we found in a lot of research is that when you add a palliative care specialist to the cancer care team, even from the point of diagnosis, that patients feel better, and they do better, and even live longer. Several studies, now, have shown that in a recent medi-analysis that we publish, for example.

So, part of the mechanism by which palliative care specialists help patients feel better and live better is not only by addressing physical symptoms, but also at addressing these difficult emotional and existential kinds of issues.

Helping with coping. How do I get through the day; how do I live with the fear that this diagnosis instills in me; how do I enjoy life? Those kinds of questions are very common. And palliative care specialists are often very equipped at helping. Or even psychologists would be another great resource, where this is a person you’re going to see where the entire focus of the visit on those issues, so that they definitely don’t get pushed to the last 30 seconds of the visit when the doctor has their hand on the door knob and they’re trying to get out to the next patient.

 

Beth Probert:

Wow, and I love what you said that your study shows that people who do seek out the palliative care will live longer. And seeing a counsellor or psychologist too, both of those are just amazing suggestions. Thank you.

We have one last question from Doug from Boise, and, Michelle, I’m going to direct this question to you.

Doug says, he doesn’t know how to find a support group. So, where does he start? Could you give us some feedback?

 

Michelle Rajotte:

Sure. He can start by calling us. We can try to find out if there’s one locally for him. There’s also access to the online chat, which meets in the evening and he can talk with people that way. There’s a lot of different options. So, there’s the traditional support groups that you go to, but there’s other ways of getting support, as well. So, that’s a good way to start. It can be very overwhelming to try to find one. The other thing you can do is if you’re being treated at a hospital, talk with the hospital social worker because they’re usually pretty knowledgeable about what supports are in the area. But I would say those would be the two good places to start.

 

Beth Probert:

That’s wonderful. And Michelle, can you give us your specific phone number and email where people can reach you and your department?

 

Michelle Rajotte:

Sure. So, the number to the Information Resource Center, we’re available Monday through Friday, 9:00 a.m. to 9:00 p.m. is 1-800-955-4572. The other way to access us is through the website, which is LLS, short for Leukemia Lymphoma Society, much easier to type, .org. When you get on there, there’s a way to reach the information resource center either by email, by chat, or the phone number will be there, as well, if you need it. But that’s really the best way to reach us.

 

Beth Probert:

That’s wonderful. And we just got one last question, and we have enough time for it. And Jim, I’m wondering if you have some advice about this question. And it is, “My partner’s often struggling deeply with the diagnosis. I don’t know the right words to say to help him feel better.” Could you give some advice to this topic, Jim?

 

James Bond:

Yes. The weekend I was diagnosed, the very qualified oncologist rightly said in response to my question, “How long do you think I’ll live?” He said, “At most you’ll live three years.” And so, I struggled, my wife struggled, I was in my early 40’s, that weekend was hell. And here’s what got me out of my funk and got us back to problem solving and putting this thing on our agenda to do all we can. I looked back at my own life, here I am in my 40’s, two boys, I’ve been healthy most all my life. And I thought of, there was a real setback, medically I had, a bad injury playing sports when I was in high school, and to me as a high school kid, that was the end of my life. Sport was gone; a lot of recuperation.

 And as I looked back on that with this cancer diagnosis, I said, “You know what, as tough as that was at the time, as devastating as that was, a lot of good things happened because of that setback.” Real things. Like it got me studying a lot more in college, I got a nice job as a result of it. Lots of good things happened. It caused me to overcome things, and I said to myself and my wife, “Hey, we’re gonna make this deadly cancer diagnosis the same thing.” And like all of us I think have been saying: every case is unique.

So, I don’t get bummed out when people give me their prognosis or whatever, or I read something that’s not good. My case is different than everybody else, and we can look at it that way. And in the end, this can happen to any of us. So, it got me off my back, it got us in there fighting, and that’s the way I look at it.

 

 

Beth Probert:

That is wonderful advice, and what I hear you saying is that, really, with your care partner, and your family, and I’ve heard this from Dr. LeBlanc and Michelle, as well, and, of course, Jim, that you’re a team. And finding that way to survive this as a team, so that’s great advice, Jim. Thank you.

 

James Bond:

You’re welcome.

 

Beth Probert:

So, I do want to thank the Patient Empowerment Network for this really impactful program today. I’d also like to thank The Leukemia and Lymphoma Society for partnering with us on this webinar. And I would like to thank our guests as we come to a close to this program. So, Dr. Thomas LeBlanc from the Duke caner institute, thank you so much for taking the time today and sharing the real benefits of the focus on the palliative care.

And Michelle Rajotte, from The Leukemia and Lymphoma Society. Your contribution has been wonderful, the resources that your department does provide. And Jim Bond, it’s just been so great hearing from you and your very long journey with AML, and what you’re dealing with, and how you have made the best life possible, and all of your dedication to advocacy. So, thank you so much for joining us today.

 

James Bond:

You’re Welcome.

 

Beth Probert:

And if our viewers have missed anything or just want to re-watch the webinar, a replay will be available in the coming weeks. Thank you for joining us, I’m Beth Probert, and I look forward with meeting with the AML community again. Thank you.


We thank Celgene Corporation, Daiichi Sankyo, Genentech, Helsinn, and Novartis for their support.

Emerging Research and Promising AML Treatment Approaches

Downloadable Program Guide

Leading experts shared recent breakthroughs in AML treatment and research announced at the 2018 American Society of Hematology (ASH) annual meeting. The panel discusses new drug approvals, emerging clinical trial data, innovative, individualized approaches to treat distinct AML subtypes, and how these advances translate to the real world and impact AML patients.


Transcript:

Beth:

Hello, and welcome to today’s webinar. I’m Beth Probert. I am an MPN patient, was diagnosed a few years ago with polycythemia vera. Today’s webinar is where are we headed with the treatment of acute myeloid leukemia. What can patients look forward to for the coming year?

This is a Patient Empowerment Network program. And I’d like to thank our sponsors. As always, our sponsors have no editorial control over the content. Today, we’re going to talk about topics like recent breakthroughs in AML treatment and research announced at the 2018 American Society of Hematology ASH Annual Meeting. We’ll look at emerging clinical trials and how to access them, individualized approaches to treat distinct AML subtypes, and how will these advances translate for patients. You’ll also hear from AML patient Steve, as he shares his first-hand experience facing AML and how he’s doing now.

We will also answer viewer questions. And if you have a question, please keep in mind, we can’t get real specific with these questions, so try to keep them general, really geared more towards information and questions.

And we’d like you to send your questions throughout the program. We will try to answer all questions that come through. And if we can’t get to all of them, we will certainly address them through future webinars. Now, I’d love to introduce you to today’s guest. Our first guest is Dr. Naval Daver, associate professor, Department of Leukemia at the University of Texas MD Anderson Cancer Center. Welcome, Dr. Daver. I’m so glad you could join us today.

Naval Daver:

Hello. Thank you for having me. Glad to join.

Beth:

And our next guess is Leah Szumita. And Leah provides clinical trial support at the Leukemia and Lymphoma Society. So, Leah, thank you. I’m glad you can be here today.

Leah:

Thank you. I’m so happy to be here.

Beth:

And our next guess is our patient panelist, Steve Buechler, and he is an AML patient who has had a remarkable journey. Steve, welcome from Minnesota.

Steve:

Thank you. I’m happy to be here.

Beth:

Great. Well, Steve, we’d like to get started with you. I’d like for you to tell our viewers a little bit about your life with AML. And if you can include how did you get diagnosed, what was that like getting diagnosed, and how did you react, who is your support team, and just what you’ve been though. So, I’ll turn it over to you now.

Steve:

Well, at age 64, I was living what I thought was a normal, healthy life. I had no symptoms. My primary care physician had been monitoring my white blood cell count for a couple of years because it was borderline low but not too alarming. And then, in the spring of 2016, it began to drop more precipitously.

So, he recommended I see a hematologist, and I went to do that. And the hematologist said I should probably have a bone marrow biopsy. And so, I agreed to do that sort of to humor them because I didn’t feel sick. I didn’t have any symptoms. I didn’t have any idea anything was wrong. It was a memorable week. The biopsy was on a Monday. On Tuesday, I swam my normal 50 laps. I did some shopping. I ate dinner out. Wednesday morning, I played in a weekly poker game with some retired guys. So, life was normal, until that phone call that came Wednesday afternoon informing me I had acute myeloid leukemia, and I had to get to a hospital right away.

So, the next day, I checked into a hospital. The day after that, Friday, I started chemotherapy. So, in 48 hours, I went from feeling perfectly healthy to 24/7 chemotherapy drip. And they started me on this standard treatment that’s been use, I think, for a very long time called 7 + 3 Cytarabine and Daunorubicin to try to get the cancer into remission. And so, I spent a week on that medication.

And then, I waited for the inevitable drop in my white blood cell count and my immune system. I was going to be very vulnerable to various kinds of infections. And as predicted, I came down with colitis and an E coli infection, body rash, and a bunch of other stuff that they couldn’t even identify. But the infectious disease doctors stepped in and dealt with those issues one at a time. So, I ended up spending 5.5 weeks in the hospital for my counts to recover. But the good news was, one month after starting chemo, they did a bone marrow biopsy that found there was no residual leukemia. So, the first goal had been reached, at that point. I was in remission.

Adding to the story, of course, the first night I spent in the hospital, my wife was with me and left late in the evening to go home. And as she arrived home, she had a stabbing pain in her right leg. The next morning, she got up and could hardly get out of bed, called 9-1-1. They brought her to my hospital in an ambulance through the ER.

And it turned out she had a fractured femur. So, I was on one floor of the hospital in the chemo ward, and she was on another floor of the hospital awaiting subsequent surgery to repair her leg. And then, she went off to a transitional care unit for rehab. So, when I realized our house was going to be unoccupied for about a month, I started to write to our neighbors on email. And I found it was a really useful way to communicate. So, I ended up, over the many months that followed, adding maybe 60 people to that email list and sending over 60 emails out, over the course of a year and a half to keep people informed of what was going on.

I, subsequently realized, as I was writing for other people that I was really using that writing to make sense of my own experience. I struggled to figure out what was going on and how I could capture it and how I could explain to people. And it was useful to get their responses back, but it was useful for me. It was very therapeutic for me just to have that writing experience to make sense out of what was going on.

After 5.5 weeks, I got permission to leave. I went home for a while. But I was awaiting the genetic testing of my cancer to figure out what the next round of treatment would be. Because I think people know, with AML, there needs to be a second arm of the treatment. It can come back very fast and very ferociously. I was told that the genetic testing of my cancer would put me in either a low risk or a high-risk category for recurrence. And that would point towards either chemotherapy, if it was low risk, and stem cell transplant, if it was high risk.

When the results finally came in, they said, “Well, you’re kind of in an intermediate category.” So, the way forward was not as clear as I thought it might be. So, I talked to my initial oncologist. I did my own research. I, subsequently, went and talked to a transplant oncologist at the University of Minnesota Medical Center who sort of nudged me towards the transplant option. I went to the Mayo Clinic and got a second opinion. And all of the indications really were that I would be a good candidate for transplant. I had no comorbidity. I had no other health problems.

And everybody thought I should probably be able to withstand the conditioning fairly well. So, eventually, I came around to that decision to have a stem cell transplant. I had a brother who was a half match donor. But the folks at the BMT unit said we also have some good umbilical cord blood matches for you. And so, I was, again, faced with the decision about which way to go. But it turned out they had a study. Don’t they always have a study? I was randomly assigned to the cord blood donor option. So, my brother was off the hook.

And I ended up having a double cord blood stem cell transplant in October of 2016, about four or five months after I was initially diagnosed. That procedure went very smoothly. And within three weeks, a biopsy revealed that one of my cord donors was 99% engrafted, which is pretty early for a cord blood procedure. So, that was good news. I was able to go home, at that point, and begin a pretty long, extensive, and sometimes arduous process of recovery.

The first 100 days, they offered me to come back to clinic daily for the first month or so to get blood tests, to get platelets, to get red blood cell transfusions, whatever it is that you need to keep you healthy. It’s a pretty vulnerable time. One of the oncologists, at the transplant unit, described this whole procedure as, first, we bring you to the brink of death by killing off diseased immune system. And then, we try to bring you back again. Well, it worked, in my case, I’m happy to report. So, by early 2017, I was beginning to taper off my anti-rejection medication. That ended in April of that year.

And then, it was just a process of gradually getting more strength, getting better. And, in my case, very fortunately, I avoided any trace of graft versus host disease. So, that allowed me to have a pretty healthy recovery. One year after my transplant, of course, I had to go in and get my baby shots, my vaccinations and needles because my previous immune system had been obliterated. And they only gave me the dead vaccines, at that point, because they reasoned I couldn’t handle the live ones.

So, that happened at Year 2. And that was recently completed about two months ago. I got the rest of my vaccines. The other good part of the story is, although there was a 60 to 70% chance of graft versus host disease, I never had any trace of that. I’ve since become very active in talking with other patients as a volunteer, doing some writing, becoming involved in the cancer community. And I’ve come to appreciate really how fortunate my story was. I think the three big indicators were I got into remission on the first try. I’ve talked to a lot of patients who haven’t been able to do that.

My transplant engrafted within three weeks, which was a very solid, early result. And a lot of patients don’t have that kind of success. And I had no graft versus host disease. So, that’s about as good of a story as you can have with AML, as I understand it. So, obviously, I’m very grateful to have done that. And something like that gives me a lot of motivation to try and give something back. So, I’ve been participating in various ways in the cancer community.

Believe it or not, that’s the short version.

Beth:

Steve, you have a remarkable story. I just heard – we talked earlier, and just to keep hearing your story again is really just so noteworthy. And the three points you made, just having the early remission the first time with chemo is amazing. And early engraftment just within three weeks and no graft versus host disease. And your enthusiasm and wanting to give back and just with your writing. And we’ll talk a little bit later. I know that you have a book that’s coming out. So, your story is, for someone like me, amazing. But Dr. Daver, I’d like to turn to you, for a few moments, and tell me, is Steve’s story typical?

And what kind of feedback do you have on his journey?

Naval Daver:

So, Steve’s story is a very good outcome story. It’s not necessarily typical, as Steve mentioned. About 70 to 80% of our patients will go into remission with the first induction. So, it’s a high number, but it’s not 100%. And if you don’t go into remission with the first induction that is actually one of the very high risk or adverse features. It’s called primary refractory AML. And those patients usually do have a much harder time. The second thing is about 60% of patients will fall in what we call intermediate groups. So, we do do molecular and cytogenetics. And if we find that we have favorable molecular cytogenetic changes, then, those are considered to be good.

And we may not do transplant. On the other hand, if you have unfavorable cytogenetic molecular, then, it’s very clear a transplant probably is the only hope for long term survival. But, unfortunately, a lot of patients fall into intermediate group.

Now, that intermediate group is becoming smaller and smaller because we are understanding more and more about the molecular machinery, the cytogenetics, and the prognostic impact of new molecular mutations. So, we are able to triage patients better into high risk or low risk, which helps us make the transplant decision. But I think the most fortunate thing, in Steve’s case, was the lack of GVHD. And that actually is very uncommon. Most of the patients we see will have some degree of GVHD. It may be acute. It maybe chronic. In most cases, I will say that it is manageable. We rarely see very severe ICU requiring GVHD or fatalities from GVHD.

But about 60 to 70% will have some degree of GVHD, will require some treatment for it with steroids or additional immunosuppression. And in some cases, it can take many months and can be a major discomfort and affect quality of life. So, I think that was fantastic that he did not have the GVHD. And I think all of those features, although are seen in a traditional AML story, I think Steve was fortunate, and the outcome was very favorable so far.

Beth:

Great. I really like that feedback. And what I wanted to ask you, as well, in regard to the no graft versus host disease, you said about 60 to 70% will actually encounter that. So, am I correct in assuming then that, when you do a transplant with someone, you account that that’s probably going to happen, the graft versus host disease and you have treatments and things lined up in anticipation of that happening?

Dr. Daver:

Yeah, absolutely. When we do the stem cell transplant itself, we actually do prophylaxis for graft versus host disease. Almost all patients will be on steroids, some form of immune prophylaxis. It may be tacrolimus. It may be sirolimus. There are some newer drugs. And in spite of that, if we see graft versus host disease, we have some very good medications.

In fact, some recent drugs approved such as Ruxolitinib, Ibrutinib, etc., which can work. But in spite of all of that, I would say a majority of patients do face a struggle with graft versus host disease. And they do have some degree. Now, again, it may not be severe. It may be in the form of graft versus host disease of the mouth, which causes your ability to eat to be decreased, or it may be the skin, which may be itchy or uncomfortable, or it could be ocular, which causes eye irritation and burning and requires eye drops. So, they may not be severe, but they hugely do cause discomfort of that quality of life.

But yes, we do try our best to avoid it. And in some patients, we are able to get away with none. And in some patients, they will have mild to moderate, which has to be treated. Luckily, with the newer generation of immune prophylaxis monitoring treatment, we have very few severe graft versus host disease, which is a good thing.

Beth:

Great. I’m so glad you touched on that. So, I wanted to shift gears a little bit, Dr. Daver, and find out from you what are some of the key takeaways for AML patients and care partners from ASH.

And I also wanted to say what I’ve heard a lot, in regard to AML is that, for almost 40 years, there was just a standard way of treating. And all of a sudden, in the very recent years or maybe year, I’m hearing that there’s so much now, new drugs and things happening. So, would you mind touching upon some of those key takeaways?

Dr. Daver:

Absolutely. I think, this year 2018 was clearly the year of AML. There’s just, compared to all of the other malignancies, in the last two years, there’s just been a huge amount of progress in the way of approvals. Now, what I do have to say is, although we are seeing the fruits of a lot of efforts, actually, the research in AML has been very intensive for the last 15 to 20 years. And what we’re now seeing is really the combination of a lot of those efforts. Molecular, immune analysis, which have led to these drug approvals.

But today, really, I think, compared to even three years ago, when we did not have a number of these drugs, the whole outlook for treatment of AML has changed dramatically. So, we’ve had eight new drugs approved in a few years. And, to put it in perspective, for the 40 years before that, we actually really had almost no drug approved. There was one drug, Gemtuzumab, approved, but it was actually withdrawn from the market. So, when they say when it rains, it pours, that kind of really did happen, in the case of acute myeloid leukemia. But what’s really important, I think, I that there are now a number of targeted therapies towards particular mutations.

And some of these have actually been approved, in the frontline setting. So, now, it has become very important that we don’t just treat all AMLs as one disease. In fact, that’s something we knew for about 20 years that AML is one of the most heterogenous of all malignancies. Lung cancer and AML, these are probably the two most heterogenous cancers where it’s not really this is AML, it’s different types of AML, which can have prognosis of 95% cure rate all the way down to 10 to 15%.

So, identifying these groups was very important for prognosis. And that’s something we have been doing but more important for treatment. So, for example, a mutation that is called an FLT3 mutation is very, very important because, on its own, it is associated with an adverse prognosis. These patients had high white counts, proliferative disease, and their three year or five-year survival was usually 20 to 25%, when we first identified this mutation in 2001. Now, there are new drugs called FLT3 inhibitors that specifically inhibit the FLT3 mutation pathway.

And with the addition of FLT3 inhibitors, specifically a drug called Midostaurin that was FDA approved 1.5 years ago, plus stem cell transplant, and even more so, at the recent ASH 2018 meeting doing post stem cell transplant, FLT3 inhibitor, when we do all of these three interventions, we’re now getting up to five year plus survival rates of 75%. So, this is amazing.

The patient who was 25% 12 years or 13 years ago, when we first identified this mutation, could today, if appropriately treated with FLT3 inhibitor transplant and FLT3 inhibitor maintenance, could be in a 75% long term survivor rate. So, tripling those outcomes. And similar things are being seen for other groups. For example, APL, acute promyelocytic leukemia, is one disease where we actually are able to treat these patients without chemotherapy. So, you can give a combination of ATRA arsenic, which gives you 95% cure rates.

So, the key now, and what I tell a lot of our community doctors, our fellows, other academicians is it’s not about just rushing in treatment, which has been the paradigm for 30 or 40 years, but more important, it identifies specific molecular mutations or cytogenetic changes and choose the best treatment because the impact of choosing the appropriate molecular or non-chemotherapy or antibody based treatment is, actually, much more than quick therapy. And I think that message now is going out.

And things are improving overall.

Beth:

Wow. And what I’m hearing are two things. Eight new drugs, however, those eight drugs are specifically going to be used, in regards to different mutations. And so, my question to you is it’s very obvious that genetic testing, for these mutations, is a huge puzzle piece to this. And could you talk a little bit about that. At what point can a patient get this genetic testing from the mutations. And if you could just speak to that because it just sounds that is essential?

Dr. Daver:

Yeah, absolutely. I think that is probably the No. 1 takeaway for both patients, caregivers, and physicians. So, the genetic testing should be done for all new AMLs at the time of diagnosis. And there are a number of different labs across the country, commercial labs, that are able to do this new genomics, foundations, hematologic, all of these are not insurance approved and covered.

Some of the larger academic centers have their own molecular testing analysis. The most important thing is that we should usually wait for these results before rushing into therapy. And just to give an example, when we see a new AML at MD Anderson, we will rush their cytogenetics and molecular testing. We’re looking for cytogenetics to rule in or rule out APL, acute promyelocytic leukemia because this can be treated without chemotherapy with 95% cure rates. The other big group we’re looking at is what we call core binding factor leukemia. These are a group of specific chromosomes associated leukemias.

And if you find those, then, that is the group or the addition of the antibody treatment called Gemtuzumab Ozogamicin or Mylotarg, which is FDA approved, can improve the survival rates by almost 20%, which is a huge amount on top of chemo. So, you don’t want to miss identifying this core binding factor of chromosomes. Then, if we don’t find one of these two, then, we rush our molecular panel.

We are fortunate. We get the molecular results in 48 hours. That’s one of the places in the country. There are a few other groups that are in the same range. But even in the commercial setting, I know for a fact that they’re able to get these results in six to seven days. So, I think it is actually possible and feasible. And even on some of the large trials we’ve done across 100 plus centers, we were able to safely wait for those results. Two molecular results were most important looking for our FLT3, if you find that mutation. We want to add the FLT3 inhibitor up front, and then, IDH1, IDH2 mutation.

And if you find those, we may consider, on a trial basis, adding IDH1, IDH2 mutations. And then, if none of those mutations or chromosome groups are identified, then, we will consider standard treatment. But even there, we have trials where we’re adding new drugs, which have shown very high activity like Venetoclax or Nivolumab or immune therapies to standard chemo. So, really, this is now personalized therapy. There are five clear subsets of AML that will have different treatment approaches.

And addition of the appropriate agent could improve your survival and cure rates from anywhere from 10 to 30 or 40%. So, I think this is quite important.

Beth:

It’s just amazing. And what I’m also picking up on, and what I’ve been told about AML, is that you need to move quick. This is, once diagnosed, time is of the essence, and especially with the different subtypes. So, we’re talking about genetic testing. And I really, really was very interested in hearing how it works and how quick it could be turned around. But what would you say – we very often hear, like in Steve’s case, it was his doctor who referred him to a local hematologist and then, eventually, to a specialist? Sometimes, we hear people being rushed to the hospital or going to their local doctor. But time is of the essence, in getting this genetic testing.

What advice do you give patients who, typically, might go to a local doctor, how to move along in this process and how to advocate for that genetic testing? Do you have any feedback on that?

Dr. Daver:

Yeah. I think there’s a fine balance. And that’s where it’s hard to make a generalized recommendation across the board because there are some AML patients who come to us who have a very high white count, more than 100,000, for example. They may have evidence of leukemia already infiltrating their liver or kidney, with organ abnormalities and lab changes. And in those patients, we may have to start treatment very early. But those are the minority. We’ve published, as other groups have looked at this, those make up about 5 to 10%. So, in the majority, it is, actually, a mindset change.

And this is something we’re doing a lot of education on, as well, is that that mindset of the sun should never set on AML. We have to treat right away, actually, was true, when you didn’t have other effective therapies that could be added that could change your outcome from 25% to 75%.

But today, in fact, I think it’s much more important to select the appropriate treatment or the addition of the appropriate molecular immune therapy than rushing into treatment. In fact, our group, as well as a number of other groups in the country, have published it. So, what we recommend, in general, is we get a new AML. We would admit those patients. I still think this is an inpatient disease. We would monitor them closely. We send, on the same day that we see them, a molecular chromosome panel. We ask it to be rushed. And then, usually, we can get these results in three to five days.

And I would wait to get those results because, based on those results, we may choose a FLT3 inhibitor. We may choose the antibody Gemtuzumab. We may choose IDH therapy. We may choose ATRA arsenic. So, I think, for most patients, what you could do, of course, you have to be careful when you’re discussing it with a physician, you don’t want to push on them too much. But I think it’s important to ask about molecular therapies, molecular trials, whether we could get the molecular information early, and how we could incorporate that.

I think, the good thing is we’re seeing, across the country, most of the physicians are taking this approach. And there is very intense education. But I still think it doesn’t hurt to ask about it and make sure that that testing is being done because I think it could make a huge difference in your outcome.

Beth:

Great. Wonderful feedback. Now, Leah Szumita, I’d like to bring you in on this conversation because we heard eight new medicines right now. That’s huge. And as Dr. Daver said, those are the results of clinical trials. And, recently, I heard that only about five to eight percent of adult cancer patients are participating nationwide, in the United States, in clinical trials. That seems like such a small number. And we depend on these patients to participate in these clinical trials to come out with these eight new meds.

There’s definitely a gap. And I’d like to hear your feedback about just that. And then, if you can go into – I’m going to ask you a few more questions about how people get involved in clinical trials. So, take us through that.

Leah:

Great, I will. So, I have to echo Dr. Daver’s sentiments about the importance of the genomic testing as well. And really, the new breakthrough in AML therapy is just a testament to the ongoing research. As he said, the research has been happening for 15 or 20 years. And we’re finally seeing the fruits of the labor. So, it’s encouraging. And that five to eight percent is low, but there’s room for improvement. And I think many different organizations have identified barriers to why these enrollment rates are so low. I will say that, of all of the clinical trials, somewhere between two and ten percent of clinical trials have to close because of low accrual rate.

So, there is just serious work to be done. I think, you can look at barriers in two different ways. There are patient barriers. There’s just a lack of awareness that clinical trials exist for all stages of diseases. So, many people believe that a clinical trial is only for those who have exhausted all other treatment options. And so, that’s actually not true. There are trials for every stage of disease. Previously untreated, newly diagnosed, relapse refractory, maintenance and remission. There are other barriers that people are afraid to be a guinea pig.

And so, I think, as healthcare providers, that’s our job to really educate that clinical trials are very controlled, closely monitored situations, provide education on the different phases and what those mean. There are very complex and stringent inclusion/exclusion criteria to clinical trials, which, in one way, can make it very difficult to understand, if you’re even eligible for a trial.

And so, that’s why clinical trial nurse navigators, such as myself, can really help patients and caregivers sort through that information. And then, sometimes, physicians aren’t aware of all of the trials that are out there either. And that is not to slight practitioners, but, again, it’s just an overwhelming amount of information. It takes time to stay on top of all of this research. It takes time to go through all of this research and all of the different protocols.

And so, it’s really important for patients and caregivers to have an advocate to try to identify what clinical trial is right for them.

Beth:

And so, through the Leukemia and Lymphoma Society, you offer this service, if I understand you correctly. So, patients and their caregivers can reach out to your department and find out what is there for me. What comes to mind, also, I hear quite often, and we’ll get Dr. Daver’s opinion on this as well, in just a moment, but there seems to be roadblocks to people, not only I don’t want to be a guinea pig and understanding that piece of it, but also are there some financial hurdles, geographic hurdles?

I hear from patients, quite often, that I live so remotely. I’m in a rural area. How would I manage this? So, could you give a little feedback about that?

Leah:

Sure. First, with regards to the financial barriers, another common myth is that a clinical trial is free. And, unfortunately, it’s not. I would say that, often times, whatever is being studied, either a new drug or a combination of drugs that usually is covered by the sponsor of the trial. But the rest of the care needs to be billed to insurance. And then, there’s this third bucket of cost, which is the money it takes to get someone and their family members to and from all of these appointments, prolonged hospital stays away from home. So, those are significant financial barriers to participate, in a clinical trial.

There are resources out there to help navigate through some of these obstacles. And, again, I would encourage people to contact Leukemia and Lymphoma Society. We can help steer you to those resources. With regards to the geographic barrier, it’s correct. A lot of these large, academic medical centers are not in proximity to people in rural areas. And that is one key point of clinical trials that needs to be improved upon. And I think a great goal would be to get some of these later stage, later phase trials out into the community setting where they may not require quite as intensive monitoring.

But it can also be available to more patients and really diversify the patient populations.

Beth:

Great. Really great feedback. And then, Dr. Daver, I know that your center is very proactive with communicating clinical trials to patients. And could you just speak about that a little bit?

I know it must be overwhelming. You’re doing your research. You’re a clinician working with your patients and to keep on top of every clinical trial. But, again, I know that that’s something you’re very, very on top of. But could you give a little feedback about how you approach that?

Dr. Daver:

Yeah. As an AML expert, I would still say I’m not really aware of every AML trial, in the country. It’s not possible. There’s 200 or 300. And they keep changing every week. So, nobody really, at a clinician level, is going to be completely aware. Now, what we do know is the comorbid areas, the targeted groups, the particular mutational groups of trial, the new trials, and, of course, what’s looking more exciting, whether it’s in Phase 1, Phase 2, or Phase 3 development. I completely echo the sentiments. I think 100% of our efforts should be to get patients on trial. And, at MD Anderson, we have 180 trials in leukemia alone of which about 70 or 80 are in AML.

And, of course, this is on the higher end of the spectrum. But the focus is really to enroll people on trial. And, I think, what patients often, and I hear this almost every day in clinic, is that they’re concerned because, when you say a trial, they are thinking experimentation. I think there’s a big difference in experimentation and clinical investigation. So, our effort is always to offer trials that give you standard of therapy plus something. And, in fact, whenever we’re treating a frontline patient, no leukemia expert, least of all, in a very large academic center, is going to randomize the patient to something other than standard of care.

But what we do want to see is can we improve the standard of care. And that’s how all of these new drugs go approved. So, we were doing these trials with FLT3 inhibitors added to chemotherapy for almost 10 or 11 years at some of the large centers in the country. Similarly, with IDH inhibitors or Gemtuzumab. And I have many patients who, seven, eight, nine years ago, were able to go on these trials, many, many years before the FLT3 inhibitors approved and get those benefits.

So, the way we like to put it is to try to get you tomorrow’s therapy today. So, you’re going to get access, approximately, four to five years before a drug is approved. And almost always, you will get the standard treatment plus something. So, you’re not going to get less. You’re going to get more. Now, of course, all of the additions may not work. But the chance is that at least you’ll get the benefit of standard agent plus something. And a lot of times, when we explain that, then, patients, of course, say I would like the trial rather than just standard of care.

The other thing is, with the cost, although it’s true that the drugs may not all be free, at least you may get some or part of, in some cases, all of the drugs free. So, at least there is some incentive there because, a lot of times, people say the insurance covers it. But the cost of a lot of drugs is astronomical. And even if you’re paying just 5% for an average AML drug targeted therapy, which is somewhere between $15,000.00 to $20,000.00, that 5% can be $1,000.00 to $1,500.00 a month.

So, a lot of times, what I see from my patients is, when they go on our trial for FLT3 inhibitors and IDH inhibitors, and even the fact that they’re not paying their co-pay, often offsets their cost of coming to MD Anderson or coming to Dana Farber or Sloan Kettering or whatever it may be. So, I really think that one should definitely talk to the Leukemia and Lymphoma Society, other major organizations, so that they can find out what trials are there. And many times, patients say, well, don’t think there’s a trial for me, or their local physician may not be aware.

And I can guarantee you, almost 99 to 100% of the time, there will be not just one but many, many trials that are available to you. So, I think that little bit of effort, emails, phone calls can go a long way.

Beth:

Great feedback. And Leah, going back to you, excuse me – I’m sorry. I just need to stand up a moment. I’m in a room that decided the lights would go off. But you can all hear me. Speaking to you, and I’m getting towards our lights, can you talk about what questions someone can ask their doctor, in regard to clinical trials?

What are those important questions?

Leah:

Absolutely. So, there are so many of them. And one of the things that my group of nurses and myself do is really provide people with education about the basics of clinical trials and then, the language and the questions they can use, when they go back to their provider. And then, also, when they go to make that connection with the clinical trial group. So, the list is long. I would say first and foremost, asking what the risks and benefits are. Many times, in a clinical trial, there are different requirements about how often someone might come to and from the site, what the finances might be related to that.

Also, a lot of studies or drugs used in studies have been used in other studies. So, asking if there are any early results or any results from prior studies using those medications is important.

And asking about how this may affect quality of life, all of those different kinds of questions. There’s a very long list. We do have a fabulous clinical trials booklet that patients and caregivers can obtain that have lists of questions. And we always encourage people to read through that material as well. But knowledge is power. So, the more knowledge and research someone does, and bringing someone with them to these appointments to really take notes because it can be so difficult to absorb all of this information, would be some of my recommendations.

Beth:

Wonderful. Great feedback. So, Steve, I’d like to circle back to you now. You have this overwhelming, very intense journey. Where did you get information about AML? Where did you get support? We hear that so often, when someone is diagnosed, and they have to handle and make decisions fast, what kind of resources did you utilize. And tell our viewers out there, so the can understand what to do and how to do it.

Steve:

Well, one thing I did not do is go on the internet and scare myself half to death. I trusted my doctors. It did happen so quickly that I was in treatment before I even understood the nature of my disease. So, for better or worse, I was getting on that train and going wherever it was going to take me. But I had a great team of social workers. I had great nurses. My oncologist was excellent in spending as much time with me as I wanted. And so, it was a gradual kind of learning curve for me. And the fact that the early treatment went pretty well, obviously, helped give me confidence.

And the same thing, when I went down to the University of Minnesota Medical Center. They gave me a very thorough explanation of what was going on, recommended the stem cell transplant. I had a colleague whose father actually worked in this area decades ago.

And I talked with him. He stressed the importance of getting a second opinion. So, I was able to go to the Mayo Clinic, which is about an hour and a half drive from where I live. And I talked, first, to a hematologist who said I can tell you some things, but you should come back and talk to the transplant experts here. So, I did that as well. So, between my initial oncologist, my transplant oncologist, my second opinions at the Mayo Clinic, I was pretty confident that not that it would all work out, but this was the best path to follow. And as I followed that path, I did get invited to a clinical trial.

Just from a patient’s perspective, some years ago, I was the caregiver for my mother, as she was struggling and eventually dying of breast cancer. And her oncologist wanted to put her in a clinical trial. And I was very suspicious, and wondering is she not going to get the kind of care that she needs because you want to use her as a subject in a study. And I declined that study. And some years later, I find myself being invited to join a study. And I asked a lot of questions, especially when I saw that 22-page consent form.

That’s pretty daunting. There’s a lot there, and there’s a lot to ask about, and I did. And people patiently answered my questions. And I just came to realize, essentially, in my case, the trial wasn’t even close to experimental. What they were saying is this is how we’re going to treat you regardless. But if you’re willing to do the study, we’re going to track the results. And that can help people down the line. So, at that point, it seemed almost like a no brainer. And I could have chosen my brother as a donor or a stem cell as a donor. Instead, I went into a study that randomized me. And I went into the stem cell, and it turned out just fine.

But they said the five-year survival rates for either path are about the same, so that’s why we’re doing the study to try to figure out what the different pathways are to that outcome and when something will benefit patients in the future. So, at that point, it just seemed like a reasonable thing to do. Helping people understand that you’re going to get the best treatment they can give you regardless, even though you’re in the study. I think that’s, for many patients, the key point. And it sounds like Leah and her folks are working on that angle.

That’s really important for patients.

Beth:

Wow, that is fabulous feedback. And if you could say – what I’m hearing you say is that you got a lot of support from, it sounds like, the hospital where you received your care. That there was you mentioned social worker, and they sounded like they were really there to give you support. Would you agree that everyone really worked together to help you through this journey?

Steve:

They did, both the professionals and circle of friends and colleagues. Of course, those email correspondences, as I said, I was getting multiple responses to every email that I sent out, from various people. Sometimes funny, sometimes dark humor, which I especially appreciate. Thank you, Dave, from Milwaukee. So, a variety of things that came in, people prayed for me. I’m not especially religious, but whatever they wanted to do was fine with me. So, the writing, again, was therapeutic.

I practiced a lot of mindfulness and meditation and yoga. I was a very active patient. I walked the halls five miles a day. When I couldn’t leave my room, I was on a treadmill. I just needed to do things that sort of kept my body up and moving. And I think that really helped my recovery. I had nurses tell me, at one point, I was doing better than any other patient, at that stage in treatment. I’m not bragging about it, but I think, again, initial good reactions made it easy to get in this upward spiral. I exercised, I ate as well as I could. And I’ve seen patients have a bad time. And they’re kind of in a downward spiral.

And it’s really hard to reverse that. If you don’t feel good enough to teat, if you don’t feel good enough to exercise, it’s really hard to get out of that box. And so, anything you can do or anything nurses or social workers can do to help patients be proactive, be as active as possible, ask lots of questions, in whatever fashion suits their needs. Try and tell your story, whether it’s Caring Bridge, or emails, or verbal recording of what’s going on, I think there’s a great therapy to just trying to put together, from a patient’s perspective, what the hell is going on here and what’s happening to me and how might it turn out.

And those are some of the things that helped me get through.

Beth:

That is just great feedback. And Dr. Daver, I’m picking up that Steve has just an amazing attitude. And what kind of feedback do you give about that? These patients, these wonderful people, their lives have been turned upside down. As you tell us, it’s just very quickly, they’re living one life and now another. How much do you see, listening to Steve’s attitude and trying to be proactive and advocate for himself, do you feel that’s an impact on overall success in treatment and moving forward?

Dr. Daver:

Yes, absolutely. I think that the attitude plays a major role. But I think a few things that Steve said are very important.

One is that he did seek out second opinions. He did go to Mayo Clinic, a very large academic center. He got additional input. He learned about clinical trials and outcomes. And a lot of times, we have patients who may contact us or physicians from outside who contact us or come to us. And sometimes, we may not have something different to offer. There may be a standard treatment. A lot of times, the peace of mind of knowing that you have consulted with a large academic center, one of the top centers, whether it’s Mayo or MD Anderson or Sloan Kettering, whichever it may be, often helps a lot.

And then, there may be other times when we actually do say, and this happens quite frequently, that, actually, we have a trial that I think will be a better FLT3 inhibitor or better IDH inhibitor or a better antibody. And this is what I would do, if I was in your place, or if I had a relative in your place. So, I think that helps your peace of mind and your mental framework. And the second thing is – and that’s not something we can control is how you do to the initial treatment.

If you have good responses, if you tolerate it well, then, of course, we do see that those patients are always more optimistic, have a better mental framework, it helps. But I also see that there are some patients who come in, with a very negative framework. And that’s where I think learning that there is so much new progress, that there are so many options, not only in the frontline setting, in the relapse setting, in the maintenance setting, even after post-transplant relapse. We have things that, potentially, could cure patients, which we didn’t have even five years ago.

So, I think knowing that there’s a huge amount of progress, that the cure rates have doubled, tripled, in some cases, in elderly AML and FLT3 AML. And no longer having AML is the end of the world. In fact, in our most recent data update that we are going to publish soon, we see that, in the young patient, 65 and below, the overall survival, if you gave all patients who visited MD Anderson is about 66%. So, 23 patients actually had a long-term cure.

And people are shocked, even physicians I know of in the ICU and ER settings, don’t realize this fact. In elderly AML, it’s tougher, but we are going from 10% to almost 45 or 50% cure rates in patients 65 plus. So, I think, once people hear these numbers, they completely change their mind and are much more optimistic. But getting that information across to patients, to caregivers, to make them do the referral or make them consider treatment, I think, is the first big hurdle that we have to kind of overcome.

Beth:

Wow. And that is just very right on target. So, I’d like to shift gears a little bit. We do have a few questions we have time for. And Dr. Daver, the first question I’d like to get your feedback on, and forgive me with the pronunciation of the actual medication, I’ll try my best. So, this question comes in, what is the role of Venetoclax, if any, in treating AML. And when might that be FDA approved, from what you might know about this?

Dr. Daver:

So, the Venetoclax is probably one of the most exciting drugs in AML, especially elderly AML. In elderly AML, it is the most exciting drug that we have had probably forever. So, we used to treat elderly AML, meaning above 65 years of age. And these are hugely people not just by age, but also based on the physician’s review who are considered not fit for intensive chemo. They may have kidney problems, liver disease, poor performance status, immobility. And so, we cannot give the high chemo, the 3 + 7 that Steve got. And we have to use lower intensity therapy.

And we used to use Azacytidine alone, with the response rate of about 20 to 25%- and 3-year survival of about 15 to 20%. And now, we’ve done a study using Azacytidine in combination with Venetoclax where the response rates were 73%. So, going from 25% to 73% not doubling or really tripling, and that the survival is now 46 or 48% going from 15 to 18%.

So, that’s a huge, dramatic shift, three times response rate, three times of the potential cure rates. So, I think, right now, we believe that Azacytidine and Venetoclax really should be the standard of care for elderly AML, if they’re not going to get induction chemo. And, in fact, it was FDA approved very recently. So, three weeks ago, in fact, right before the ASH meeting, end of November, Azacytidine in combination with Venetoclax, as well as low dose Cytarabine and combination with Venetoclax were FDA approved.

And I think, now, with the approval, although we were doing this even before the approval, no elderly AML patient should get Azacytidine or low dose Cytarabine alone. I really think addition of Venetoclax now is the standard of care, triple response rate, triple survival. There’s no reason not to do that.

Beth:

Wow. That is an amazing shift and such good news for our elderly patients. That is great. I do have another question. And I believe it’s targeted for you as well, Dr. Daver.

For those young folks, under 35, who relapse quickly, within about 100 days after MUD allo transplant for AML, M5, no mutation target, what will be a sustainable way to buy time and bridge for that next transplant. Could you talk a little bit about that?

Dr. Daver:

So, that’s a very tough scenario. Relapsing post-transplant itself is a very high-risk feature. It, basically, indicates that disease is aggressive and may not respond to further chemotherapy or transplant. But relapsing early post-transplant, which we usually consider within 100 or 120 days is actually quite an adverse feature. So, for those patients, I think the best chance is if we can find a targetable mutation. So, we will be looking for FLT3 or IDH1, IDH2 mutations. If we find those, then, I think we do have some chance with either a FLT3 inhibitor alone or, more likely, in a FLT3 inhibitor, in combination with low intensity therapy.

And there are a number of these agents either approved, but I would actually go for a trial where we’re combining either FLT3 inhibitors or IDH1, IDH2 inhibitors with other exciting agents like Azacytidine and Venetoclax. I think that will be the best shot of getting a long-term remission, potentially, a second transplant. Of course, there are a lot of caveats and variables. And you have to look at the individual patient to make that determination. The other group of therapies that you could use, if we don’t find the FLT3 or IDH because only about 30 to 40% of patients will have one of these three mutations, is immunotherapies.

And these can work really well, especially in the post-transplant relapse setting. And we have drugs such as antibody drug conjugates. These are antibodies that carry a toxin and can attack the leukemia cells. Or what we call immune check point antibodies. They’re also agents that activate your own immune system post-transplant to fight against tumor. And with these, we have seen some very exciting activity, specifically, in the post-transplant relapse.

And a lot of these are all under clinical trial setting because the antibodies and the immune checkpoints are not yet approved. They may be in the next couple of years. So, I think this would be an ideal scenario to find the academic center close to you and try to consider getting into one of the trials, either targeted therapy or immune therapy.

Beth:

And another question would be do you see post-transplant relapse more in specific mutations? Are those with specific subtypes of AML?

Dr. Daver:

Yes, we do. So, we see the post-transplant relapse most common in what we consider the adverse risk AML. So, the adverse risk AML are the patients we definitely take to transplant. But, unfortunately, even after transplant, they remain the group that have a high risk of relapse. So, these are patients who have what we call TP53, one of the worse mutations. They will often have a high risk of relapse post-transplant or chromosome changes like deletion 7, deletion 5, deletion 17, also another high-risk group.

And the third group is what we call secondary AML. So, there are two ways you could get AML. You could have spontaneous AML, most common. We have a patient, no prior history of chemo radiation, other cancers, who comes in with acute diagnosis of AML. But then, there’s another group making about 20 to 30% called secondary AML. So, these are people who have prior breast cancer, colon cancer, bladder cancer, and got either chemotherapy or radiation for that. Or people who had prior MDS, which is an AML precursor and then, developed AML.

And these people who have secondary AML are much more risky and also more prone to relapse post-transplant. There are a few new drugs like Vyxeos that can work well, in this situation. But, in general, these are probably the high risk molecular or morphological groups that could relapse post-transplant.

Beth:

Very interesting. Well, I so appreciate all of the wonderful information and feedback that our guests have provided today.

And the timing is great. Just coming off of ASH has been extremely encouraging, Dr. Daver, with you sharing all of these wonderful new eight new drugs and insight that’s going on. And, Leah, your feedback has just been phenomenal. And really, I believe it’s going to ease people’s concerns and fears about clinical trials, and between you and Dr. Daver speaking about the clinical trials, why they’re so essential, and they’re doable. And, Steve, your feedback, not only about clinical trials, but your journey is phenomenal. And I hope our viewers look forward to seeing information.

We may not have mentioned this. Steve has written a book soon to be published about his journey. He has some very interesting feedback that we just didn’t have enough time to share on today’s webinar.

So, thank you, again, to our guests and our sponsors. And a replay will be completed soon. And you’ll receive it via your email. So, our audience, please look forward to that. And remember, be your own advocate. Thank you.


We thank Celgene Corporation, Daiichi Sankyo, Genentech, Helsinn, and Novartis for their support.

Finding the Right AML Treatment for Me

Finding the Right AML Treatment for Me from Patient Empowerment Network on Vimeo.

What is personalized medicine, and how can it help AML (acute myeloid leukemia) patients? Broadcasted live from the 2017 ASH (American Society of Hematology) meeting, Patient Power’s Andrew Schorr was joined by AML experts Dr. Gwen Nichols, Executive Vice President and Chief Medical Officer at The Leukemia & Lymphoma Society (LLS), Dr. Ross Levine, the Laurence Joseph Dineen Chair in Leukemia Research at Memorial Sloan Kettering Cancer Center, and Rick Ross, an AML patient advocate. The expert panel shared an overview of the AML news coming out of ASH, an update on personalized medicine options currently available and highlighted helpful resources for patients and their loved ones.

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