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Ask the AML Expert: What Do New Drugs Mean for My AML?

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Published on May 20, 2019

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During this “Ask the Expert” replay, noted myeloid specialist Dr. Daniel Pollyea, from the University of Colorado School of Medicine, responds to questions from patients and families living with acute myeloid leukemia (AML). How are treatment strategies changing with recent FDA approvals? Dr. Pollyea describes how doctors are individualizing AML treatment, ways to identify key disease features and important discussions to have with your healthcare team. Watch now to learn how advances in AML drug development are impacting patients.

Send in your questions to [email protected] to be answered on future AML programs.

This program is sponsored by AbbVie, Inc. It is produced by Patient Power in partnership with The Leukemia & Lymphoma Society (LLS) and NeedyMeds. These organizations have no editorial control, and Patient Power is solely responsible for program content. 



The Leukemia & Lymphoma Society (LLS)

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Transcript | Ask the AML Expert: What Do New Drugs Mean for My AML?

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

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

Andrew Schorr:

And greetings for this Patient Power program, Ask the Expert program for those of you dealing with acute myeloid leukemia. I'm Andrew Schorr, down near San Diego, and I want to welcome you, and thanks to AbbVie for supporting this educational program, although, they have no editorial control, it's all what we say, and what our leading expert says. Now, I'd like to introduce him. Joining us from Denver, Colorado is Dr. Dan Pollyea. Dr. Pollyea is the head of the research for the Department of Leukemia. Am I right about that? Of leukemia research? 

Dr. Pollyea:      

Clinical Director of Leukemia Services, so I'm more on the clinical side, but yes, yes. 

Andrew Schorr:           

Okay, I want to get the title right. Thank you so much, and of course, he is a myeloid specialist. That means people dealing with myeloid conditions, and of course, acute myeloid leukemia, he often sees them, and has been dealing with research, as well. Dr. Pollyea, welcome. Let's just start with a couple questions for me, and I want to tell our audience, if you have a question, send it in to AML at Many people have, and we'll be posing that to Dr. Pollyea. 

And one other note before we begin, and that is, Dr. Pollyea is going to give you an overview and answer some questions, but he can't tell you and your doctor what to do, nor would that be appropriate on the internet, right? So, from what you learn here, discuss it with a healthcare team that you trust, and see what's right for you. 

Okay. Dr. Pollyea, I understand it's a whole new ball game in AML in the last year or two after many years where you didn’t have, really, any new treatments, so am I right about that?

Dr. Pollyea:      

Yeah, you're absolutely right, Andrew. I mean, it's just an incredible time right now for AML. We've had years and years of research that we’ve been building upon to get to this point where we've been able to understand enough about the biology of this disease so that we can have new and exciting therapies that really make an impact. 

So, it's really this incredible time of a confluence between all the work that's been done on the research side for many years coming together with all the things we've been learning about and working with some of our pharmaceutical partners with new targeted therapies, and it's just everything's come together at the right time, it's very exciting. 

Andrew Schorr:           

Okay, but we're not done yet. And I know one of the questions we got in is, are there drugs in the pipeline where this evolution will continue?

Dr. Pollyea:      

Well, absolutely, Andrew. So, I mean, I think we've had an explosion of drugs approved in AML. In the last two years, I think something like eight new drugs approved, only yesterday a drug that was recently approved was approved by the FDA for a new indication in AML. So, the pipeline is happening right now. I mean, that's the first message, but yes. 

Even beyond the eight or so drugs that have been approved in the last two years, there are several that we think are on the launching pad, and then several more that are hopefully a little bit behind that. And then lots more that are several years out. So, it's really really an active pipeline between university academic leukemia doctors, community-based oncologists, and industry. So, a really nice partnership. 

Andrew Schorr:           

Okay, so for our viewers, family members, and patients, it sounds like if you're dealing with AML, it's crucial to have the right testing so that you and your doctor know of these different options, now, in ones that are investigational in clinical trials, what may line up with your personal situations, is that right?

Dr. Pollyea:      

That's absolutely right. So, this disease is very complicated. And so different from person to person. So, AML is one disease, but everyone's case of AML is unique to them. It really is. So, for a long time, for many years, that was a barrier because of those that heterogeneity; the differences in the disease. But over time, we've become able to really identify some key features of individual disease, and everyone has a unique disease, but we're using sort of the same parts. 

And so, we're able to identify commonalities between patient's diseases, and that has to be done at the time diagnosis. So, the only time—and here I'm really talking about gene mutation testing, almost every AML patient has at least one gene that is mutated that is responsible or contributing to their disease. And most patients have several. And we've developed mutation gene panels, you know, 50 different genes that we can test for, at the time diagnosis to determine what unique features a patient has.  

Because if a person has a combination of three of the 50 genes, that's likely to be unique from another patient who has another three genes, and the prognosis, and even the treatment plans are gonna differ based on what constellation of gene mutations you have. The only time to get that information, is at the time of diagnosis. We also repeat it if people relapse, but really, the time to really understand a person's disease is that very first bone marrow biopsy. That's an important message we’re trying to get out. 

Andrew Schorr:           

Okay, so some people may have gone to a community hospital because they were really sick and then suddenly, you're in a hospital, there may not be an AML program or a specialist there. So, let's acknowledge that people could be anywhere, not necessarily at the University of Colorado at your big medical center. So, what can patients and family members be asking so that this revolution in AML care can be brought to bear for them?

Dr. Pollyea:      

Yeah, so that is a great point. And resources are not the same all over the country. I think all of us have a preference for patients at the time of diagnosis to be treated at a large academic medical center. I think there is some data that outcomes are probably  better when that is a possibility. So, we often will take newly diagnosed patients as transfers to our center from all over the region. And sometimes that's appropriate. 

And so, that's a question that could be asked if you're at a smaller community center, whether or not it would be appropriate to be transferred over to a larger regional center, if you're already inpatient. If you're outpatient and things are going at a pace at which it's not so much of an emergency, then seeking a second opinion at a large center where a lot of AML experience is had would be important. 

Sometimes a person finds themselves, like you said, in a smaller regional hospital without much leukemia experience, and there really aren't options to go to a bigger place because there's limitations based on safety or maybe just insurance and things like that can be barriers. So, then you'd want to just nicely ask whether the treating physical, you know, if they don’t have a lot of experience with this disease, which is understandable, it's a rare disease, only 30,000 new cases in the United States each year, whether they have a colleague at another institution who they feel comfortable talking to and sharing the case with, and getting some advice, or having a discussion about. 

That's a very regular routine thing that most community-based oncologists are very amenable to. Most of them have that resource, that partner that they work with at a larger center. And so, just making sure that they're speaking to them in the early days of your diagnosis would be important.  

Andrew Schorr:           

Okay. Let's go on. So, first of all, if you have a question, folks, send it to AML at I have just a couple more because I've been around this for a long time and I'm a chronic leukemia patient myself. So, not everybody, but a lot of people who develop AML are older. And I had a cousin years ago, didn’t live long with what was available then. Are treatments changing for older people in what you’ve said, and they're sort of—I don’t want to say kinder, gentler, but maybe you're more frail when you're old, but even with this revolution can give older people, mom, dad, grandma, grandpa hope.

Dr. Pollyea:      

You know, older people is really ground zero for AML. So, this standard of care for many years has been a newly diagnosed patient would receive intensive induction chemotherapy. Sometimes we call it the seven plus three regimen. People might—that might sound familiar to them. Now, that is the standard of care, but because like, you said, the average patient with AML is older, the standard of care can be very difficult to apply to older patients because older patients have a very difficult time with intensive chemotherapy. 

So, for the last several years, decade at least, the population of AML that's really been the most intensely studies with respect to drug development and new clinical trials has been older patients, newly diagnosed, who can't tolerate intensive induction chemotherapy. And so, in fact, most of these advances that I've spoken about, the new drugs approved in the last two years, etcetera, most of them are specific to older newly diagnosed patients. 

And so, yes, I would say the bulk of advances in drug development in the recent past have been for older AML patients because that's the bulk of who AML is. Yes, like you said, AML can happen in younger patients, but that's really rare. That's uncommon. This is mostly a disease of older patients. 

Andrew Schorr:           

Okay. One of the approaches has been, in blood cancers, generally, has been transplant, which is a big deal. Maybe not right for someone who's older. And so, now you have pills, as well. So, what kind of success are you seeing with these pills?

Dr. Pollyea:      

Yeah, so a lot of these pills are dramatic. I mean, we have venetoclax (Venclexta) is the main one that comes to mind in terms of a newly diagnosed older AML patient, and just this past November, the FDA approved the use of venetoclax with a low intensity chemotherapy backbone treatment for this population, a newly diagnosed, older AML patient. 

Prior to this, the standard of care had been using just low intensity chemotherapy treatments and the responses and the outcomes there have been quite disappointing. The response rates that we see with venetoclax, which is the pill that you mentioned in this setting are really really promising, unlike anything we've really seen. The duration of responses are very good. The patient's quality of life is quite good. 

So, that's just one example. There are several other pills that play that are relevant here. Some are for patients in the relapse setting when they have particular gene mutations. We talked before that almost every patient has at least one if not several gene mutations. We've been able to target several of those gene mutations with pill therapies, and most of those have been approved in the relapse setting. 

So, relapsed AML patients, usually older can now be candidates for these pill therapies that can work quite well and have a very tolerable side-effect profile for the most part if they carry a particular mutation; if their disease carries a particular mutation. So, that's a really promising new direction too. 

Andrew Schorr:           

We got a question, and people were asking, well, is there a venetoclax dose that is too low? So, how do you figure out the right dose so that it’s effective and side effects are non-significant?

Dr. Pollyea:      

Sure. Well, the FDA approval is for 400 milligrams of venetoclax. So, venetoclax comes in 100 milligram tablets, so four of those a day. There's scale up to get to that dose the first couple days of the first cycle we like to escalate up to 400 milligrams slowly for a variety of reasons. So, that is the standard accepted dose for venetoclax. There are some opportunities to reduce the dose of venetoclax depending on the situation. 

Sometimes, if a patient needs to be on a certain class of medications that can interact with the venetoclax, we have to reduce the dose. Sometimes, if a person's blood counts are low and the treating physical believes that they are low because of the venetoclax, it may be appropriate to reduce the dose. But the standard dose of venetoclax after that initial scale up is four pills or 400 milligrams a day.

Andrew Schorr:           

Now, we got a lot of questions, Dr. Pollyea about the effects of chemo in many people. We've talked about older people are often not strong enough to deal with it. So, are we moving away from chemo, or you mentioned even with venetoclax somebody might have a low dose of chemo, but people are concerned about the side effects, or whether they can withstand them?

Dr. Pollyea:      

That is our greatest concern, as well. I think intensive chemotherapy regimens for older patients, I shudder at the thought of that. Even the healthiest older patients, when you expose them to these intensive chemotherapy regimens can have very poor outcomes. And I often will tell patients that depending on the situation, a person has upwards of a 20 percent chance of death from the treatment, not from their disease. We really, at our institution, I know others have varying degrees of thoughts on this, but at least at our institution, we really do our best to avoid that experience, for almost all older patients, to expose them to that intensive chemotherapy. 

Now, in years past, there weren't many other viable options, and so you tolerated a great deal of risk because of the reality that there were few, if any, other treatment options. So, if you didn’t expose your patient to this horrible intensive chemotherapy regimen, then they would die of their disease. So, you made that calculation. The landscape is really different now.  

And so, we have two FDA approved therapies that are lower intensity for a newly diagnosed AML patient. One I already referred to is the venetoclax. A second is called glasdegib (Daurismo), that works a different way. And actually, as of yesterday, a third was—I alluded to this before, if you have an IDH1 mutation, and you're a newly diagnosed patient who's either older or can't tolerate intensive chemotherapy, then the FDA has said now that you can be prescribed an IDH5681 inhibitor, previously that drug has only be approved in the relapse setting, but now it's available in the untreated or newly diagnosed setting. 

So, in the end, that's a long answer to the question, but the shorter answer is, yes, we need to get away from intensive chemotherapy, particularly for older patients. I would like if I could look at a crystal ball, I would like to see in five, seven, 10 years that the amount of intensive chemotherapy we're giving to any patient, younger or older is very limited, low. I would hope that that's the direction the field is going to.