AML Programs Archives
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Advice for Managing Emotions Around AML
How can AML patients and care partners cope with their emotions? Dr. Eric Winer advises taking it day by day and emphasizes the importance of open communication, involving care partners, and utilizing additional healthcare team members such as mental health professionals and social workers.
Dr. Eric S. Winer is Assistant Professor of Medicine at Harvard Medical School and Clinical Director of Adult Leukemia at Dana-Farber Cancer Institute. Learn more about Dr. Winer.
Related Resources:
Distinguishing AML Symptoms and Side Effects | Why Communication Is Essential |
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Expert Perspective | The Value of Empowering AML Patients and Care Partners |
Transcript:
Katherine Banwell:
How do you counsel patients on coping with the emotional side of AML? What advice and resources are available?
Dr. Eric Winer:
The advice I tell people is this is a journey. This is not a one-day process. My advice is we really need to take this on a day-by-day basis. Don’t think about what’s going to happen three months from now. Let’s work on going step by step, day by day. That is one of the easier things for me to say and a harder thing for a patient to do. But I think that giving that roadmap makes it easier and empowers them to understand what their next steps are going to be in this entire process.
Sometimes if we need to, we can have psychiatry involved. Sometimes we can have social work involved. Some patients even ask for medications in order to help with that, and we have those medications. That’s why it’s so important to have that communication so that we can help with things that are bothering the patient that may not be just related to the disease itself.
Katherine Banwell:
Why is the role of a care partner, a friend, or family member, so important? How can they help?
Dr. Eric Winer:
There is something to be said about having a familiar person go through a difficult process with a patient. I spend a tremendous amount of time with my patients, but I don’t have a history that dates back many years. Although I do end up having that at some point, in the beginning that’s not what we have.
It’s important, while I’m getting to know the patient, to have somebody who does know the patient, that knows the patient’s way of looking at things, their ins and outs of how they see the work, and how they’re going to react to different aspects of their care. Having somebody like that around is extraordinarily important from a patient comfort standpoint, but also from a practitioner standpoint. It’s good to have another person there, but the other reason I think that gets a little bit underrated is in those initial assessments, in those first days where we’re sitting down and talking to the patient, and saying, hey, here’s what’s happening. Here’s what’s going on. It’s really hard to sit down and listen and get a full understanding in one shot. We don’t try to do it in one shot.
We try to be available, but having a second or third or fourth set of ears is very important, because an hour later the patient may say wait a minute, what did that mean? Why didn’t we ask that? And having somebody else there at that moment to say, oh, we did ask that. Here’s what we talked about. This is what we said. Having that second set of ears in a time where it’s overwhelming is really very helpful to the patient.
Expert Perspective | The Value of Empowering AML Patients and Care Partners
How can patients with AML and their care partners feel empowered? Dr. Eric Winer, an AML expert, discusses the crucial role of the healthcare team and emphasizes the importance of open communication, asking questions, and understanding the care plan.
Dr. Eric S. Winer is Assistant Professor of Medicine at Harvard Medical School and Clinical Director of Adult Leukemia at Dana-Farber Cancer Institute. Learn more about Dr. Winer.
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Distinguishing AML Symptoms and Side Effects | Why Communication Is Essential |
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Transcript:
Katherine Banwell:
As a provider, Dr. Winer, how do you empower care partners and patients who have been diagnosed with AML?
Dr. Eric Winer:
From a provider standpoint, one of the most important things we need to do is listen and try to understand, first of all, what the patient is going through, and what the patient needs. Like I said, that’s where this is a team approach. It’s a team approach from not just a standpoint of patient and caregiver, patient and physician, patient and nurse practitioner or physician’s assistant, patient and social worker.
This is a traumatic experience, and there are things that we can try to do to make it less traumatic, but one of the best ways to make it less traumatic is making sure patients are informed, that patients understand plans, that patients understand what’s going to be happening. One of the biggest concerns with any of these diseases is the unknown. There is a certain amount of unknown that I can’t predict.
I can’t say to somebody you will or will not respond, but what I can say is, listen, here is what we’re going to be doing over these next few weeks, and here is our short-term plan, and here is our long-term plan, and making sure that we’re all on the same roadmap.
I think that’s really important in terms of empowering the patients. The other thing that is important is that the patients should feel comfortable asking questions, because we’re not expecting our patients to be experts in leukemia. We’re expecting our patients to be the patients. I think of us, in terms of the clinicians, as being sherpas. We’re guiding people through this process, but the reality is they’re doing all the work. We’re just the guides, and so it’s important as guides that we make sure all these questions are answered, all of the information is given to the patient, and that the patient asks for that information.
The other thing that is important, that is a little overlooked, is taking care of the caregivers as well. It’s very difficult to be a patient, but it’s also difficult to take care of a loved one or a friend during this time. So, it’s important that the caregivers have an adequate understanding as well about what’s going on, and what we expect to be coming forward in the next days and weeks so that they can plan for these processes as well.
AML Clinical Trials | When to Consider This Treatment Option
How do clinical trials fit into an AML treatment plan? Dr. Eric Winer highlights the importance of clinical trials to advancing AML therapies and encourages discussing your options and trial eligibility with your care team.
Dr. Eric S. Winer is Assistant Professor of Medicine at Harvard Medical School and Clinical Director of Adult Leukemia at Dana-Farber Cancer Institute. Learn more about Dr. Winer.
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Elevate | Expert Advice for Accessing Quality AML Care and Treatment |
Elevate | What You Should Know About Your Role in AML Treatment and Care Decisions |
Transcript:
Katherine Banwell:
When considering treatment options, where do clinical trials fit into the plan?
Dr. Eric Winer:
I think clinical trials are extraordinarily important. The way that I think many of us think about this is we want to continually do better, and have our patients continually have improvements. The only way we can do that is by bringing forth novel medications in order to gain that extra improvement. As mentioned, all of these small molecule inhibitors, every drug that we have out there, started off as clinical trials.
We were able to gain benefit, and patients are able to gain benefit by taking part in these clinical trials. Not all clinical trial is successful to be fair, and different types of clinical trials have different scientific knowledge beforehand. For example, Phase I clinical trials tend to be more experimental. We don’t know as much about the drugs.
Phase III experimental clinical trials are much more well-known. Then there are a bunch in between in terms of Phase I’s where we know the drug, but we’re studying more of a combination, but of these clinical trials, the purpose of this is to gain benefit.
If we didn’t have a drug that we believed was going to be helpful, we wouldn’t be doing that clinical trial. So, while some people may think of these things as experimental, I think of them as rationally evaluating a way to target particular forms of leukemia to gain better responses.
Katherine Banwell:
If a clinical trial isn’t offered, how can patients inquire about their potential options?
Dr. Eric Winer:
The first thing to do is speak to their physician. Many physicians, if they have clinical trials, they’ll know the eligibility. They’ll know who is and isn’t eligible, and why they’re not eligible. That’s something that can be easily explained to people. The second thing is if there aren’t clinical trials available at that institution, then it’s important for the patients to talk to their clinicians, and say, is there a clinical trial available someplace else that might be good for me.
Many of us field calls from other physicians, from other colleagues, who call us and say, “Hey, I have a patient with this particular disease. Do you have a clinical trial available?” We’re always willing to collaborate. The one nice thing about the leukemia field is it’s a relatively small field. We all know each other. We all realize that the purpose of this is to make patients better. And so, we all share information, and we all work together to try to get that accomplished.
Katherine Banwell:
There are a couple of really good websites available too, to find out about clinical trials, correct?
Dr. Eric Winer:
There are, and I think that by contacting different institutions that can be helpful, such as the Leukemia & Lymphoma Society is a good one. There is a national clinical trial database called clinicaltrials.gov.
Those are all very important, but sometimes they can be a little difficult to navigate. And so, it’s always good to go back to your physician or your physician team and discuss these things to make sure that the clinical trial that someone is looking at is actually an applicable clinical trial for them.
Emerging AML Treatment Options | Inhibitor Therapies
What AML treatments in development are showing promise? Dr. Eric Winer, an AML expert and researcher, discusses emerging treatments and the importance of clinical trials, noting the shift toward more personalized, targeted therapies.
Dr. Eric S. Winer is Assistant Professor of Medicine at Harvard Medical School and Clinical Director of Adult Leukemia at Dana-Farber Cancer Institute. Learn more about Dr. Winer.
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Expert Overview | AML Treatment Options and Phases of Therapy |
AML Gene Mutations | Emerging Targeted Therapies in Development |
Transcript:
Katherine Banwell:
What can you tell the audience about menin inhibitors for AML?
Dr. Eric Winer:
Menin inhibitors are a very exciting, novel agent that’s come about in the next few years. The science of that actually stemmed from one of the laboratories at Dana-Farber, a physician named Scott Armstrong. Menin inhibitors are particular small molecule agents that are given orally that prevent the proliferation of leukemia cells.
And really, initially, as of a little while ago, we thought that it was two particular mutations, something called NPM1 and something called KMT2A.
But maybe we’re thinking that there may be other mutations that actually are also affected by these menin inhibitors. What’s important now is there are three different menin inhibitors in clinical trials in different stages that are very close to being approved, and so this is going to add just another agent in our armamentarium in order to treat AML and treat it specifically.
I think one of the important aspects of this is that these medications, although not approved right now, are really showing a lot of clinical benefit in clinical trials. And so, it really highlights the importance of thinking about clinical trials when being treated with these diseases because the patients that are on these trials right now, and that are doing well with these medications, if they’re not in a center that has a trial, or they choose not to go on the clinical trial, then they don’t have the opportunity to have these drugs, and they don’t have the opportunity to get that improvement.
Katherine Banwell:
Is there other research in AML that you’re excited about?
Dr. Eric Winer:
Yeah, there is a lot of different research going on in AML constantly. One of the important aspects of AML is that we’ve changed the way we think about the disease over the past, say, 10 years or so. Ten years ago, we really only focused on a couple of different genetic mutations and focused on chromosomal abnormalities.
What we’ve done now is we’ve been able to fine-tune things a little bit deeper, not just to look at chromosomes, but also to look at the genes that are involved. When we think 15 years ago, maybe we looked at two genes that were involved in these mutations and these mutational analyses. Now, we’re running panels that are between 50 and 90 gene mutations.
We can determine if there is a particularly actionable gene that we can go after to gain benefit gain better responses, and gain remissions. Over these past 10 years, we’ve had a number of different drugs approved that are what we call small molecule inhibitors. There are a number of different small molecule inhibitors targeting different mutations. For example, there is a particular mutation called FLT3. There have been three drugs that have been approved over the past eight years to target that particular mutation, midostaurin (Rydapt), gilteritinib (Xospata), and quizartinib (Vanflyta).
These actually are very specific to that mutation, and all three of them have shown significant improvement when patients are treated with those inhibitors. Similarly, there’s a mutation called IDH1 and IDH2. There have been small molecule inhibitors, ivosidenib (Tibsovo), enasidenib (Idhifa), and olutasidenib (Rezlidhia), which basically are targeting those particular mutations.
In targeting those mutations, you’re gaining improvement. You’re gaining a better response, but more so by targeting these mutations, you oftentimes are having less side effects than what you would see with what I call old-school, conventional chemotherapy.
The landscape of leukemia is dramatically changing right now, and it’s been dramatically under a transformation over the past, say, seven, eight years, where we went from looking at high doses of chemotherapy that was like a nuclear bomb exploding all over the marrow and wiping everything out, to really creating more of a targeted approach to how we treat patients. Almost thinking of it more as a personalized medicine in a way.
A lot of people come in thinking about chemotherapy as the chemotherapy that their grandmother went through, or that their friend went through in the 1970s or ‘80s. And it really is a very different world. So, I think with these progressions that we’ve made, and with these advances that we’ve made, leukemia, although it still is a very frightening prospect, it’s something that we’re really making large strides in improving, and that I expect to continue to improve over the years.
Distinguishing AML Symptoms and Side Effects | Why Communication Is Essential
How can patients determine if they are experiencing AML symptoms or side effects of treatment? AML expert Dr. Eric Winer underscores the importance of open communication with the healthcare team for timely, effective care, and better outcomes.
Dr. Eric S. Winer is Assistant Professor of Medicine at Harvard Medical School and Clinical Director of Adult Leukemia at Dana-Farber Cancer Institute. Learn more about Dr. Winer.
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Expert Perspective | The Value of Empowering AML Patients and Care Partners |
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Transcript:
Katherine Banwell:
It may be difficult to distinguish disease symptoms from treatment side effects. What advice do you have for patients who are experiencing any issues that stem from their AML? Why is communication important?
Dr. Eric Winer:
When I speak to my patients, I say to them, your job is to tell me everything that is going on. My and my team’s job is to figure out what’s important and what’s not because that’s what we’re trained to do. I think that brings up two aspects. One is the importance of openness and honesty, because we can’t treat a problem if we don’t know there is a problem. We have a plethora of drugs that we can use for different symptoms, but if we don’t know the symptoms, we can’t treat them. A perfect example is if somebody is nauseated, we can’t tell by looking at them if they’re nauseated.
We can tell when they’re vomiting and at that point, it’s too late, but if we know ahead of time they’re nauseated, then we can actually give a number of different treatments. What I say is it’s not just telling me, it’s telling people on my team as well, because it’s important to have an entire team involved in the care. So, for example, at Dana-Farber, we don’t just have a doctor-patient relationship. We have a doctor. We have a nurse practitioner. We have a physician’s assistant. We have nursing staff. We have social workers, care coordinators. It’s really a gigantic team effort, all working to try to make the best outcome and best situation for the patients.
Expert Advice | How to Elevate Your AML Care and Treatment
How can patients elevate their AML care and treatment? Dr. Eric Winer from Dana-Farber Cancer Institute emphasizes the importance of actively participating in the care journey, staying informed about treatment plans, and seeking a second opinion.
Dr. Eric S. Winer is Assistant Professor of Medicine at Harvard Medical School and Clinical Director of Adult Leukemia at Dana-Farber Cancer Institute. Learn more about Dr. Winer.
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Expert Perspective | The Value of Empowering AML Patients and Care Partners |
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AML Care Essentials | Health Literacy and Recommended Resources |
Transcript:
Katherine Banwell:
This interview is part of PEN’s Elevate series, which encourages patients to take an active role in their care to improve outcomes. What advice do you have for AML patients who are seeking to elevate their care?
Dr. Eric Winer:
I think it’s important for people to be an active participant in this care. While we recognize that not everyone has medical expertise when they are going through this, it’s important that they are informed that they would like to be informed. It’s important that they have a full understanding of what’s going on in terms of the treatment, the plan, the short-term plan, and the long-term plan.
So, there is a lot of information that needed to be digested. What’s important to note is that it doesn’t all have to be digested at that very moment at the time of diagnosis. These are processes. What I tell people is that this is not going to be your only opportunity to talk and ask questions. This is something that we’re going to be going through and doing this journey together. So, I think it’s important that they become an active participant in that journey, not just with themselves but also with whoever their caregiver is, and whoever is important to them that’s going to be going through this journey as well.
Katherine Banwell:
I understand that some AML cases require treatment shortly after diagnosis. Is there room for a second opinion, and if so, what are the benefits?
Dr. Eric Winer:
So, there often is room for a second opinion. There are times, quite honestly, like you said, where patients have a really acute problem when they come in, along with the diagnosis, or that the diagnosis has gotten to a point where we need to initiate therapy as an inpatient, urgently. That is becoming less common. It used to be, when I started doing this, that if you had a diagnosis of AML you were admitted to the hospital, and you stayed there until you completed your first round of therapy. That is not the case now.
Generally, what we’ve learned is that there are studies that show you can actually delay therapy for a period of time in order to make sure that other things are established. Getting a second opinion is very important, particularly as a tertiary care center where physicians specialize in these types of diseases. And so, I think it’s very important to get expert opinions, not just in terms of how to treat the disease but also diagnostically, and to make sure the correct tests are run, the correct molecular studies are run, in order to figure out exactly what would be the best treatment for your individual version of AML.
AML Gene Mutations | Emerging Targeted Therapies in Development
What are emerging targeted therapies for AML? Dr. Daniel Pollyea discusses the current landscape of targeted treatments for AML gene mutations, while emphasizing ongoing research efforts surrounding less common mutations.
Dr. Daniel Pollyea is Clinical Director of Leukemia Services in the Division of Medical Oncology, Hematologic Malignancies and Blood and Marrow Transplant at University of Colorado Cancer Center. Learn more about Dr. Pollyea.
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Transcript:
Katherine Banwell:
Chris sent in this question: I would like to hear more about mutations found during molecular testing. Are there new AML drugs in trials for other less common mutations?
Dr. Daniel Pollyea:
Great question. So, at the moment, what we have clinically available are targeted therapies for patients with FLT3 mutations, IDH1, and IDH2 mutations.
And there are about 50 different genes that can be mutated in AML, and so that’s a small slice of the pie. Those are relatively common mutations, but still, small slice of the pie. A lot of the very uncommon or less common gene mutations we don’t have great paths to targeted therapies for them.
And is that just we never will? I don’t think necessarily, but I think those can be really challenging. Not every mutation is amenable to a targeted therapy, at least as far as we know now. The one that’s coming, that we’re hopeful about is NPM1, which may be able to be targeted with one of those menin inhibitors that we talked about. So, that’s the next big one up.
And that will probably constitute 40 percent of patients that have one of those mutations that I listed. But research is ongoing to kind of try and dig into this more. What I will say is that the AML research community is so fantastic that every lead is being pursued, and there is a lab somewhere in the world whose focus is on whatever small, even the most least common AML mutation; that’s somebody’s focus.
And so, if there were to be promising therapies developed for even rare mutations, I assure you, the field would take those forward and figure out a way to do those clinical trials and to get to approval if it’s appropriate. So, but I think that’s where the landscape is right now.
AML Treatment Planning | Key Questions to Ask Your Doctor
What key questions should patients ask about their AML treatment plan? Dr. Daniel Pollyea provides key advice and emphasizes the importance of sharing symptoms, while also highlighting the critical role of a care partner for support and advocacy in AML care.
Dr. Daniel Pollyea is Clinical Director of Leukemia Services in the Division of Medical Oncology, Hematologic Malignancies and Blood and Marrow Transplant at University of Colorado Cancer Center. Learn more about Dr. Pollyea.
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AML Treatment | Understanding Induction and Consolidation Therapy |
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Transcript:
Katherine Banwell:
Dr. Pollyea, when choosing a therapy what questions should patients be asking their healthcare team about a treatment plan?
Dr. Daniel Pollyea:
So, at the time of diagnosis I think it’s a reasonable question to say, “Is my disease amenable to a cure? Can I be potentially cured?” and “Is this treatment part of a plan for a cure?” If that is possible, then I would want to be walked through the steps that that’s going to executed. And if it’s not possible for me to be cured, then I would like to discuss what is the treatment plan that could potentially give me the longest duration of a remission and the best quality of life. And so, that’s the conversation that I think is important to have.
And then, everything that we discuss comes into play there; an individual’s sort of appropriateness for intensive chemotherapy versus less intensive regimens, and also the disease biology and what that maybe make them a candidate for.
Katherine Banwell:
Are there certain symptoms or side effects a patient should share with their care team?
Dr. Daniel Pollyea:
Yeah. So, we have a very, very sort liberal request that really anything, it should be shared. We have a 24/7 number to call with one of us on-call at all times. So, it’s very difficult for a patient to kind of be able to appreciate, when they’re going through such dramatic changes, “Hey, is this expected or not?” So, we really emphasize oversharing concerns about symptoms.
All these drugs have very different side effect profiles, and some of them are common and some of them are less common. The disease itself can cause symptoms and clinical issues. So, instead of really trying to educate yourself in an impossible way on what could be or is not related, it’s better just to ask.
Katherine Banwell:
What is the role of a care partner when someone is in active treatment?
Dr. Daniel Pollyea:
Having a care partner is crucial. This is physically and mentally extraordinarily stressful on the body and on the mind. Having that support person for those purposes is really important. Having that person be an advocate for a patient to ask those questions that may not be getting asked, to reframe questions to get the best answers is really, really important.
And then, there’s the more mundane things; just getting patients to their appointments and kind of keeping their morale up and those things. So, there’s data and research on this that patients with caregivers, they have better outcomes. When it comes to a transplant, a caregiver is not an option. You must have a caregiver. And the importance of that will be sort of relayed to you in the context of a discussion about a transplant. But a caregiver in the setting of a transplant is so important that it is a requirement to even be considered for that.
An Overview of Current AML Treatment Types
Dr. Daniel Pollyea provides an overview of AML treatment options, explaining the choices between intensive chemotherapy and targeted therapies, while also discussing when stem cell transplants are considered.
Dr. Daniel Pollyea is Clinical Director of Leukemia Services in the Division of Medical Oncology, Hematologic Malignancies and Blood and Marrow Transplant at University of Colorado Cancer Center. Learn more about Dr. Pollyea.
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AML Gene Mutations | Emerging Targeted Therapies in Development |
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Transcript:
Katherine Banwell:
Would you share an overview of the types of therapy for AML, and how do you decide which patient gets what?
Dr. Daniel Pollyea:
Yeah. Because things are very different at relapse too, but at diagnosis, the options still are intensive chemotherapy, which is a regimen that hasn’t changed much in several decades really, 50 years.
And then, there are other treatments. There’s a treatment called venetoclax (Venclexta) that we pair with a low-intensity chemotherapy treatment, either azacitidine (Vidaza), decitabine (Dacogen), or something called low-dose cytarabine (Cytosar U). Those are the three sort of partners for venetoclax.
And then, there’s a targeted therapy against leukemia cells that have an IDH1 mutation that’s called ivosidenib (Tibsovo) that we also give with low-dose chemotherapy. So, in most cases those are the sort of three general options. That last treatment that’s targeted against IDH1, we typically preserve that for older patients or those that really are not good candidates for intensive chemotherapy but who have that IDH1 mutation, which is only somewhere around 10 percent of AML patients.
And then, so then the main decision then is “Do we give intensive chemotherapy, or do we give the venetoclax regimen?” And our policy is sort of, if we think we can cure you within intensive chemotherapy, and there’s certain disease biology subtypes that can be cured potentially with intensive chemotherapy, then that would be our first choice for you.
If we don’t think we can cure you with intensive chemotherapy, if you don’t have that disease biology or if you do but you’re just not a candidate for that type of an approach, that’s when we give the venetoclax regimen.
Katherine Banwell:
Are there other targeted therapies that you use?
Dr. Daniel Pollyea:
Yes. So, venetoclax is a targeted therapy against Bcl-2. Unlike some of these other gene mutations, you don’t have to have something; there’s no mutation in Bcl-2 that you need to be a candidate for venetoclax. We give venetoclax pretty much to any potential AML patients. Genomically-targeted therapies: you mentioned FLT3. Before I mentioned IDH1. There’s also one for IDH2. We hope there’s a couple more of these coming. Where these are approved, for the most part, at the moment, are in the relapse setting.
So, a patient who receives a treatment, and then either doesn’t respond or responds and then relapses, that’s typically where we bring in these genomically-targeted therapies. There’s an exception for IDH1 that, like I said, can be used now in the upfront treatment setting. But for the most part, these genomically-targeted therapies are relevant in relapse disease.
Katherine Banwell:
When would you use stem cell transplant?
Dr. Daniel Pollyea:
So, stem cell transplant for the majority of AML patients is still the only potential way to cure this disease. And so, a stem cell transplant is something that we give for that purpose. It’s something that we really reserve for people whose disease is in a remission. So, nobody comes in at diagnosis and goes right into a stem cell transplant; that wouldn’t work. So, you first have to achieve a remission with any number of one of the combinations of things that we’ve already discussed.
But once the patient is in a remission and doesn’t have a curative strategy with, like, intensive chemotherapy or some other approach and is a good candidate for a transplant, which is a whole other sort of set of circumstances that has to be considered, that’s patients who we offer a transplant for.
AML Therapy | Emerging Treatments and Clinical Trials
What new AML treatments are emerging? Dr. Daniel Pollyea discusses recent advances in AML therapy, including the new menin inhibitors in development, and which patients they may be right for. Dr. Pollyea also emphasizes the crucial role of clinical trials as a treatment option for patients.
Dr. Daniel Pollyea is Clinical Director of Leukemia Services in the Division of Medical Oncology, Hematologic Malignancies and Blood and Marrow Transplant at University of Colorado Cancer Center. Learn more about Dr. Pollyea.
Related Resources:
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AML Gene Mutations | Emerging Targeted Therapies in Development |
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Transcript:
Katherine Banwell:
What about new and emerging treatments?
Dr. Daniel Pollyea:
So much that’s really exciting here. So, we’ve had several new approvals. We have a new FLT3 inhibitor that we can use for newly diagnosed patients who have a FLT3 mutation and who are getting intensive chemotherapy.
We have, even now, a new therapy that’s given as a maintenance treatment. It’s called oral azacitidine or Onureg, which is really exciting as well.
But I think the next sort of big thing in the field is going to be a targeted therapy for another subset of patients who are defined by the presence of a gene mutation, NPM1, but also by a chromosomal abnormality, something we call KMT2A. But these patients have disease that’s potentially amenable to what we call a menin inhibitor.
And there are several companies with menin inhibitors. These therapies are getting pretty far along. We expect approval potentially soon for at least one of them. And then, I think these are going to have a big impact on the field for those patients who have that type of disease.
Katherine Banwell:
Oh, that’s exciting news. Where do clinical trials fit in?
Dr. Daniel Pollyea:
So, clinical trials are crucial for everything that we’re trying to do. We don’t make any progress without clinical trials. So, that’s the field as a whole. We don’t move forward. We don’t get any of these new treatments without clinical trials.
On an individual patient level, clinical trials are also really important because, for many patients we are still not doing as well as we want to be doing with this disease. We’ve made progress, but there’s still a lot of room for improvement. And so, for an individual patient, getting access to another therapy that, although we admit we don’t quite know yet whether it may be helpful but might be helpful, I think, is a really compelling situation to potentially consider participating because it is a guarantee you will help the field; and it’s a guarantee you will help every patient that comes after you through participation in clinical trial.
But all these clinical trials are also designed to help you; to help you in a situation where we as a field don’t feel like we’re doing well enough. So, clinical trials, totally crucial if we’re going to continue making progress.
And clinical trials are the reason why these last 10 years we have had such just dramatic improvement in availably of all these new therapies because literally thousands of patients have chosen to participate.
Katherine Banwell:
How can patients find clinical trials that might be right for them?
Dr. Daniel Pollyea:
So, back to The Leukemia & Lymphoma Society. They can be really helpful in guiding this. Asking your doctor, “Hey, are there any clinical trials her or at any other center that I should be considering?” And then, people who are interested in just going to the source. Every clinical trial that is available is registered at clinicaltrials.gov. And so, going to clinicaltrials.gov and then putting in some keywords like “acute myeloid leukemia,” you’ll see every clinical trial that’s available.
AML Treatment | Understanding Induction and Consolidation Therapy
How are AML induction and consolidation therapies defined? Dr. Daniel Pollyea explains these phases of therapy and how they are used in AML patient care.
Dr. Daniel Pollyea is Clinical Director of Leukemia Services in the Division of Medical Oncology, Hematologic Malignancies and Blood and Marrow Transplant at University of Colorado Cancer Center. Learn more about Dr. Pollyea.
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Transcript:
Katherine Banwell:
We’ve covered this in past programs, but I think it’s worth reiterating. Would you define induction and consolidation therapy for the audience?
Dr. Daniel Pollyea:
Yeah. So, traditionally when we only had intensive chemotherapy treatments, induction meant “Let’s get your disease under control.” That’s the first sort of line of treatment. “Let’s induce a remission.” That’s where that comes from.
And then, consolidation meant “Let’s do more stuff, more chemotherapy to consolidate that remission,” or you can think of it as maintain that remission, deepen that remission. All those are sort of the same adjectives there. So, induction was step one. Consolidation was step two. We’ve retained a lot of this language into a time when we don’t only have intensive chemotherapy.
So, we’ll still use the word induction sometimes to mean “Let’s get your disease under control, even if it’s not with intensive chemotherapy.” So, admittedly that can be very confusing, but if someone uses it in that manner, that what they’re talking about is “Let’s get your disease under control.”
And consolidation still meant “Let’s deepen your remission” or “Let’s prolong your remission.” So, those are the general terms. They’re very much linked to intensive chemotherapy, which we still use, but it’s not all we use anymore.
So, I think it has gotten confusing, and it’s perfectly reasonable to be confused about that terminology.
How Is an AML Treatment Plan Determined?
Dr. Daniel Pollyea explains the importance of collaborating with your healthcare team on your AML care decisions and discusses factors that guide an individualized AML treatment plan, such as age, overall health, and personal preference. Dr. Pollyea also addresses the role of common AML gene mutations when choosing therapy.
Dr. Daniel Pollyea is Clinical Director of Leukemia Services in the Division of Medical Oncology, Hematologic Malignancies and Blood and Marrow Transplant at University of Colorado Cancer Center. Learn more about Dr. Pollyea.
Related Resources:
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AML Treatment | Understanding Induction and Consolidation Therapy |
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Transcript:
Katherine Banwell:
When it comes to choosing AML therapy, it’s important to work with your healthcare team to identify what will be best for you. Would you walk us through the factors that are considered when choosing therapy for AML?
Dr. Daniel Pollyea:
Sure, yeah. So, we now have options in treatments for this disease and for decades, that wasn’t the case. This was a one-size-fits-all type of disease. And in the last eight years, that has completely changed.
So, there are approaches and diagnosis that vary between very intensive chemotherapy and less intensive treatments. What we call “targeted therapies” in some cases can be considered or be appropriate.
And so, having a sense, after learning a little bit about this, of how much would you be willing to tolerate an intensive chemotherapy regimen and all the risks inherent in that, if that’s even being presented as an option, and if so, what does that look like? And if not, hey, what are the other options if that sort of doesn’t sound like something that you would be willing to accept? So, I think those kind of probing questions.
First, asking yourself and then sort of translating that into your treatment team, into “Hey, this is sort of how I define quality of life.
And these are some red lines that I wouldn’t cross,” that can really help the healthcare team because, again, this is not one-size-fits-all anymore. We do have several options to consider at the time of diagnosis.
Katherine Banwell:
What other factors would you take into consideration? Do you look at age and overall health and fitness, test results?
Dr. Daniel Pollyea:
Absolutely. So, the relevant factors at the time of diagnosis would be, as you described, age, to some extent. And there’s no magic cutoff. “When a person is a certain age, this is no longer a treatment.” But age just gives us guidelines. Other comorbidities, other disease that you may be dealing with, things in your past, organ dysfunction; all those things are really, highly considered.
And also, sort of your own attitude toward “Hey, would I be okay with a month-long stay in the hospital or is that something that there’s no sort of outcome that that would be okay for me to withstand?” But then, the other huge part of this are things that are sort of, at diagnosis, unknown to you and unknown to your doctor for a little bit. And those are disease factors. So, what are the mutations that make up your disease? What’s making your disease tick? And now, just with normal clinical care, we have unbelievable access to this information. We can essentially learn within a week or two every relevant mutation that’s contributing to your disease.
And that helps us tremendously with respect to prognostication, sure, but also treatment selection because there are some treatments that will work, we think, better with certain disease biology, and other treatments that will work less well.
And we even have targeted therapies; so, based on particular mutations or other abnormalities, sort of a rationally designed therapy for exactly that disease biology. So, that is also a huge part of treatment selection, and we call those disease factors.
Katherine Banwell:
Why is molecular testing important following an AML diagnosis?
Dr. Daniel Pollyea:
Right. So, this basically just gets into what we were just discussing. So, that molecular testing is the testing that will tell us all the mutations that make up your disease biology. And so, that is crucial for prognostication, but also treatment selection.
And frankly, also when thinking about how to potentially cure your disease, those will be factors taken into account to make decisions that are pretty significant, such as should you receive a bone marrow transplant at some point in the future or not. And the reason it’s so crucial to get this done at diagnosis is, after diagnosis, we start a treatment, and hopefully we put your disease into a remission. And at that point, we no longer have access to your disease cells.
They’re gone, or they’re too low to even measure. And so, we need to get this information at diagnosis so that we can have it later on so that we can really understand your disease and make the best treatment plan for you.
AML Care Essentials | Health Literacy and Recommended Resources
How can patients learn about acute myeloid leukemia (AML)? Dr. Daniel Pollyea defines AML, how the condition develops, and highlights the importance of health literacy. Dr. Pollyea also recommends resources like the Leukemia & Lymphoma Society for learning about AML and shares key questions to ask your doctor.
Dr. Daniel Pollyea is Clinical Director of Leukemia Services in the Division of Medical Oncology, Hematologic Malignancies and Blood and Marrow Transplant at University of Colorado Cancer Center. Learn more about Dr. Pollyea.
Related Resources:
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Transcript:
Dr. Daniel Pollyea:
AML, acute myeloid leukemia, it’s a type of a cancer. You can think of it as a cancer of the bone marrow, and it’s the likely result of several abnormalities, or sometimes I call them mistakes that can occur in stem cells or a stem cell in the bone marrow. And those mistakes that occur, most times, we don’t understand why they happen.
In most cases, they’re completely out of a person’s control. This isn’t something that comes on because it runs in a family in most cases, or because of something somebody did or didn’t do. These appear to be pretty random events that occur. But these mutations that occur in these sort of stem cells in the bone marrow cause a cell to become a cancer cell.
And over a course of a variable amount of time, these can evolve and develop into this condition, AML.
Katherine Banwell:
Okay, thank you for that. Health literacy, which is defined by the ability to find, understand, and use information for health-related decisions, is essential. Would you expand on the term “health literacy” and why it’s important to accessing quality AML care?
Dr. Daniel Pollyea:
Yeah. So, I think health literacy in our field is a challenge, because these are acute conditions that come on oftentimes very quickly. And these are not diseases that are top of mind. Most people don’t know somebody who’s had this. They’re not common; only about 30,000 people every year in the United States will have AML. So, it’s very hard to have any sort of background in this.
And for most patients because of the pace at which this disease occurs, it can be very difficult to sort of read up on it before meeting with a provider or an expert or a specialist. So, there’s a lot of challenges or barriers to health literacy. But like anything, the more a person knows, the more sort of empowered they can be, the more ability they have to ask questions and seek care at sort of the optimal place.
What I find often is that health literacy is best harnessed by a patient’s team; so, in other words, their support system, their family and friends. Because it’s so much to deal with in such rapid succession, to get this diagnosis and to usually be feeling very poorly. To also be expected to sort of have read the most relevant literature and come armed with that information is often too much at the beginning.
So, in the beginning, I think it’s best to leave that to your support system, and then as time goes on and as you start treatment, get comfortable, health literacy in our field, it becomes a more prevalent issue. And I think that when patients learn the most about how the field has evolved and where we are, the better that they can potentially do.
Katherine Banwell:
Well, that leads us perfectly into my next question. What resources do you suggest for boosting knowledge about AML?
Dr. Daniel Pollyea:
AML is like so many fields in medicine, but probably more so, moving so quickly that sort of the usual Google search is not going to, in most cases, bring up the most important, the most relevant information.
So, I think that there are some organizations out there that do a really good job of educating patients. The Leukemia & Lymphoma Society is one. They have a good website.
They have people you can contact, and they have really good information that’s available to patients and their families. That’s where I typically recommend people start. And then from there, based on our interest in education level and things like that, there can be other resources. But I think The Leukemia & Lymphoma Society’s a great place to start.
Katherine Banwell:
Okay. Newly diagnosed patients and their care partners are often overwhelmed, as you mentioned earlier. What advice do you give them at their first appointment?
Dr. Daniel Pollyea:
Right. So, this is a huge challenge. Anybody in the situation would be feeling like this. So, first of all, it’s sort of like, it’s okay to feel like this. It’s normal.
It would be unnatural to not be overwhelmed with what you’re going through; that’s an important message. And then, I think there’s this period of time between diagnosis and a plan that is particularly anxiety-provoking. And so, as your doctor and their team sort of sorts through the necessary information to get a plan together, just know that that this a very anxiety-provoking time when you’re being told that you have a really significant and serious disease, and we don’t have a plan yet.
So, making sure that you sort of comfort yourself during that period, knowing that that his temporary and that is potentially the worst anxiety you will feel, I think, can be helpful. And then, from there once the plan is sort of in place and enacting it, it really is just focusing on short-term goals.
So, instead of thinking three steps ahead and how’s the transplant going to work, in the early days, focusing on “Okay, how am I going to get into a remission?” and “How am I going to feel day-to-day? How can I feel as best I can day-to-day? What’s the best path to a remission?” And then, once you sort of meet the goal of remission, “Okay, what’s next? How are we going to cure this?” So, thinking through sort of in short bites, I think, is best.
Katherine Banwell:
Are there other key questions that they should be asking their doctor or their healthcare team?
Dr. Daniel Pollyea:
Yeah. Depending on the situation, this is a disease that can be cured; and so, from the first day, asking “Is that a possibility for me? Is there a curative plan for me, and what might that look like?” I think is an important question to ask from the beginning.
Making sure you communicate your goals and your wishes, how you define quality of life, what that means to you. And in that way, that can really help inform your doctor and their team to put together a plan that sort of is most customized to you.
Elevate | Expert Advice for Accessing Quality AML Care and Treatment
How can you access the best care and treatment for YOUR AML? Dr. Daniel Pollyea, an AML expert, discusses the importance of patient education, including understanding the available treatment options for AML, how test results may impact care, and he shares advice for advocating for yourself.
Dr. Daniel Pollyea is Clinical Director of Leukemia Services in the Division of Medical Oncology, Hematologic Malignancies and Blood and Marrow Transplant at University of Colorado Cancer Center. Learn more about Dr. Pollyea.
Related Resources:
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Expert Overview | AML Treatment Options and Phases of Therapy |
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Transcript:
Katherine Banwell:
Hello and welcome, I’m your host Katherine Banwell. Thanks for joining us for another webinar in the Patient Empowerment Network’s Elevate Series. The goal of these programs is to help AML patients and care partners feel educated and informed when making decisions with their healthcare team.
Before we get into the discussion, please remember that program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you. Well, let’s meet our guest today. Joining us is Dr. Daniel Pollyea. Welcome. Thank you so much for being with us. Would you introduce yourself?
Dr. Daniel Pollyea:
Yes, thanks so much for having me. I’m Dan Pollyea and I work at the University of Colorado where I lead the leukemia team.
Katherine Banwell:
Thank you so much for joining us today. As part of this new series we’re learning more about researchers like you. You’re on the frontlines of advancing AML care. What led you here and why is it important to you?
Dr. Daniel Pollyea:
I think my path is everyone’s, is distinct and a bit different.
In short, I think working in AML is one of the most exciting areas in medicine that a person can be in right now. It’s this incredible intersection between delivering potentially curative treatments to patients and sort of harnessing the most unbelievable research-driven sort of drug development, new therapies to patients. So, it’s just a really, really exciting time for all of us who work in the AML field because of all that those opportunities bring to bear.
Katherine Banwell:
Let’s start by having you define AML for the audience.
Dr. Daniel Pollyea:
AML, acute myeloid leukemia, it’s a type of a cancer. You can think of it as a cancer of the bone marrow, and it’s the likely result of several abnormalities, or sometimes I call them mistakes that can occur in stem cells or a stem cell in the bone marrow. And those mistakes that occur, most times, we don’t understand why they happen.
In most cases, they’re completely out of a person’s control. This isn’t something that comes on because it runs in a family in most cases, or because of something somebody did or didn’t do. These appear to be pretty random events that occur. But these mutations that occur in these sort of stem cells in the bone marrow cause a cell to become a cancer cell.
And over a course of a variable amount of time, these can evolve and develop into this condition, AML.
Katherine Banwell:
Okay, thank you for that. Health literacy, which is defined by the ability to find, understand, and use information for health-related decisions, is essential. Would you expand on the term “health literacy” and why it’s important to accessing quality AML care?
Dr. Daniel Pollyea:
Yeah. So, I think health literacy in our field is a challenge, because these are acute conditions that come on oftentimes very quickly. And these are not diseases that are top of mind. Most people don’t know somebody who’s had this. They’re not common; only about 30,000 people every year in the United States will have AML. So, it’s very hard to have any sort of background in this.
And for most patients because of the pace at which this disease occurs, it can be very difficult to sort of read up on it before meeting with a provider or an expert or a specialist. So, there’s a lot of challenges or barriers to health literacy. But like anything, the more a person knows, the more sort of empowered they can be, the more ability they have to ask questions and seek care at sort of the optimal place.
What I find often is that health literacy is best harnessed by a patient’s team; so, in other words, their support system, their family and friends. Because it’s so much to deal with in such rapid succession, to get this diagnosis and to usually be feeling very poorly. To also be expected to sort of have read the most relevant literature and come armed with that information is often too much at the beginning.
So, in the beginning, I think it’s best to leave that to your support system, and then as time goes on and as you start treatment, get comfortable, health literacy in our field, it becomes a more prevalent issue. And I think that when patients learn the most about how the field has evolved and where we are, the better that they can potentially do.
Katherine Banwell:
Well, that leads us perfectly into my next question. What resources do you suggest for boosting knowledge about AML?
Dr. Daniel Pollyea:
AML is like so many fields in medicine, but probably more so, moving so quickly that sort of the usual Google search is not going to, in most cases, bring up the most important, the most relevant information.
So, I think that there are some organizations out there that do a really good job of educating patients. The Leukemia & Lymphoma Society is one. They have a good website.
They have people you can contact, and they have really good information that’s available to patients and their families. That’s where I typically recommend people start. And then from there, based on our interest in education level and things like that, there can be other resources. But I think The Leukemia & Lymphoma Society’s a great place to start.
Katherine Banwell:
Okay. Newly diagnosed patients and their care partners are often overwhelmed, as you mentioned earlier. What advice do you give them at their first appointment?
Dr. Daniel Pollyea:
Right. So, this is a huge challenge. Anybody in the situation would be feeling like this. So, first of all, it’s sort of like, it’s okay to feel like this. It’s normal.
It would be unnatural to not be overwhelmed with what you’re going through; that’s an important message. And then, I think there’s this period of time between diagnosis and a plan that is particularly anxiety-provoking. And so, as your doctor and their team sort of sorts through the necessary information to get a plan together, just know that that this a very anxiety-provoking time when you’re being told that you have a really significant and serious disease, and we don’t have a plan yet.
So, making sure that you sort of comfort yourself during that period, knowing that that his temporary and that is potentially the worst anxiety you will feel, I think, can be helpful. And then, from there once the plan is sort of in place and enacting it, it really is just focusing on short-term goals.
So, instead of thinking three steps ahead and how’s the transplant going to work, in the early days, focusing on “Okay, how am I going to get into a remission?” and “How am I going to feel day-to-day? How can I feel as best I can day-to-day? What’s the best path to a remission?” And then, once you sort of meet the goal of remission, “Okay, what’s next? How are we going to cure this?” So, thinking through sort of in short bites, I think, is best.
Katherine Banwell:
Are there other key questions that they should be asking their doctor or their healthcare team?
Dr. Daniel Pollyea:
Yeah. Depending on the situation, this is a disease that can be cured; and so, from the first day, asking “Is that a possibility for me? Is there a curative plan for me, and what might that look like?” I think is an important question to ask from the beginning.
Making sure you communicate your goals and your wishes, how you define quality of life, what that means to you. And in that way, that can really help inform your doctor and their team to put together a plan that sort of is most customized to you.
Katherine Banwell:
That makes sense. Excuse me. When it comes to choosing AML therapy, it’s important to work with your healthcare team to identify what will be best for you. Would you walk us through the factors that are considered when choosing therapy for AML?
Dr. Daniel Pollyea:
Sure, yeah. So, we now have options in treatments for this disease and for decades, that wasn’t the case. This was a one-size-fits-all type of disease. And in the last eight years, that has completely changed.
So, there are approaches and diagnosis that vary between very intensive chemotherapy and less intensive treatments. What we call “targeted therapies” in some cases can be considered or be appropriate.
And so, having a sense, after learning a little bit about this, of how much would you be willing to tolerate an intensive chemotherapy regimen and all the risks inherent in that, if that’s even being presented as an option, and if so, what does that look like? And if not, hey, what are the other options if that sort of doesn’t sound like something that you would be willing to accept? So, I think those kind of probing questions.
First, asking yourself and then sort of translating that into your treatment team, into “Hey, this is sort of how I define quality of life.
And these are some red lines that I wouldn’t cross,” that can really help the healthcare team because, again, this is not one-size-fits-all anymore. We do have several options to consider at the time of diagnosis.
Katherine Banwell:
What other factors would you take into consideration? Do you look at age and overall health and fitness, test results?
Dr. Daniel Pollyea:
Absolutely. So, the relevant factors at the time of diagnosis would be, as you described, age, to some extent. And there’s no magic cutoff. “When a person is a certain age, this is no longer a treatment.” But age just gives us guidelines. Other comorbidities, other disease that you may be dealing with, things in your past, organ dysfunction; all those things are really, highly considered.
And also, sort of your own attitude toward “Hey, would I be okay with a month-long stay in the hospital or is that something that there’s no sort of outcome that that would be okay for me to withstand?” But then, the other huge part of this are things that are sort of, at diagnosis, unknown to you and unknown to your doctor for a little bit. And those are disease factors. So, what are the mutations that make up your disease? What’s making your disease tick? And now, just with normal clinical care, we have unbelievable access to this information. We can essentially learn within a week or two every relevant mutation that’s contributing to your disease.
And that helps us tremendously with respect to prognostication, sure, but also treatment selection because there are some treatments that will work, we think, better with certain disease biology, and other treatments that will work less well.
And we even have targeted therapies; so, based on particular mutations or other abnormalities, sort of a rationally designed therapy for exactly that disease biology. So, that is also a huge part of treatment selection, and we call those disease factors.
Katherine Banwell:
Why is molecular testing important following an AML diagnosis?
Dr. Daniel Pollyea:
Right. So, this basically just gets into what we were just discussing. So, that molecular testing is the testing that will tell us all the mutations that make up your disease biology. And so, that is crucial for prognostication, but also treatment selection.
And frankly, also when thinking about how to potentially cure your disease, those will be factors taken into account to make decisions that are pretty significant, such as should you receive a bone marrow transplant at some point in the future or not. And the reason it’s so crucial to get this done at diagnosis is, after diagnosis, we start a treatment, and hopefully we put your disease into a remission.
And at that point, we no longer have access to your disease cells. They’re gone, or they’re too low to even measure. And so, we need to get this information at diagnosis so that we can have it later on so that we can really understand your disease and make the best treatment plan for you.
Katherine Banwell:
Right. We’ve covered this in past programs, but I think it’s worth reiterating. Would you define induction and consolidation therapy for the audience?
Dr. Daniel Pollyea:
Yeah. So, traditionally when we only had intensive chemotherapy treatments, induction meant “Let’s get your disease under control.” That’s the first sort of line of treatment. “Let’s induce a remission.” That’s where that comes from.
And then, consolidation meant “Let’s do more stuff, more chemotherapy to consolidate that remission,” or you can think of it as maintain that remission, deepen that remission. All those are sort of the same adjectives there. So, induction was step one. Consolidation was step two. We’ve retained a lot of this language into a time when we don’t only have intensive chemotherapy. So, we’ll still use the word induction sometimes to mean “Let’s get your disease under control, even if it’s not with intensive chemotherapy.” So, admittedly that can be very confusing, but if someone uses it in that manner, that what they’re talking about is “Let’s get your disease under control.”
And consolidation still meant “Let’s deepen your remission” or “Let’s prolong your remission.” So, those are the general terms. They’re very much linked to intensive chemotherapy, which we still use, but it’s not all we use anymore.
So, I think it has gotten confusing, and it’s perfectly reasonable to be confused about that terminology.
Katherine Banwell:
Would you share an overview of the types of therapy for AML, and how do you decide which patient gets what?
Dr. Daniel Pollyea:
Yeah. Because things are very different at relapse too, but at diagnosis, the options still are intensive chemotherapy, which is a regimen that hasn’t changed much in several decades really, 50 years.
And then, there are other treatments. There’s a treatment called venetoclax (Venclexta) that we pair with a low-intensity chemotherapy treatment, either azacitidine (Vidaza), decitabine (Dacogen), or something called low-dose cytarabine (Cytosar U). Those are the three sort of partners for venetoclax.
And then, there’s a targeted therapy against leukemia cells that have an IDH1 mutation that’s called ivosidenib (Tibsovo) that we also give with low-dose chemotherapy. So, in most cases those are the sort of three general options. That last treatment that’s targeted against IDH1, we typically preserve that for older patients or those that really are not good candidates for intensive chemotherapy but who have that IDH1 mutation, which is only somewhere around 10 percent of AML patients.
And then, so then the main decision then is “Do we give intensive chemotherapy, or do we give the venetoclax regimen?” And our policy is sort of, if we think we can cure you within intensive chemotherapy, and there’s certain disease biology subtypes that can be cured potentially with intensive chemotherapy, then that would be our first choice for you.
If we don’t think we can cure you with intensive chemotherapy, if you don’t have that disease biology or if you do but you’re just not a candidate for that type of an approach, that’s when we give the venetoclax regimen.
Katherine Banwell:
Are there other targeted therapies that you use?
Dr. Daniel Pollyea:
Yes. So, venetoclax is a targeted therapy against Bcl-2. Unlike some of these other gene mutations, you don’t have to have something; there’s no mutation in Bcl-2 that you need to be a candidate for venetoclax. We give venetoclax pretty much to any potential AML patients. Genomically-targeted therapies: you mentioned FLT3. Before I mentioned IDH1. There’s also one for IDH2. We hope there’s a couple more of these coming. Where these are approved, for the most part, at the moment, are in the relapse setting.
So, a patient who receives a treatment, and then either doesn’t respond or responds and then relapses, that’s typically where we bring in these genomically-targeted therapies. There’s an exception for IDH1 that, like I said, can be used now in the upfront treatment setting. But for the most part, these genomically-targeted therapies are relevant in relapse disease.
Katherine Banwell:
When would you use stem cell transplant?
Dr. Daniel Pollyea:
So, stem cell transplant for the majority of AML patients is still the only potential way to cure this disease. And so, a stem cell transplant is something that we give for that purpose. It’s something that we really reserve for people whose disease is in a remission. So, nobody comes in at diagnosis and goes right into a stem cell transplant; that wouldn’t work. So, you first have to achieve a remission with any number of one of the combinations of things that we’ve already discussed.
But once the patient is in a remission and doesn’t have a curative strategy with, like, intensive chemotherapy or some other approach and is a good candidate for a transplant, which is a whole other sort of set of circumstances that has to be considered, that’s patients who we offer a transplant for.
Katherine Banwell:
Okay. What about new and emerging treatments?
Dr. Daniel Pollyea:
So much that’s really exciting here. So, we’ve had several new approvals. We have a new FLT3 inhibitor that we can use for newly diagnosed patients who have a FLT3 mutation and who are getting intensive chemotherapy. We have, even now, a new therapy that’s given as a maintenance treatment. It’s called oral azacitidine or Onureg, which is really exciting as well.
But I think the next sort of big thing in the field is going to be a targeted therapy for another subset of patients who are defined by the presence of a gene mutation, NPM1, but also by a chromosomal abnormality, something we call KMT2A. But these patients have disease that’s potentially amenable to what we call a menin inhibitor. And there are several companies with menin inhibitors. These therapies are getting pretty far along. We expect approval potentially soon for at least one of them. And then, I think these are going to have a big impact on the field for those patients who have that type of disease.
Katherine Banwell:
Oh, that’s exciting news. Where do clinical trials fit in?
Dr. Daniel Pollyea:
So, clinical trials are crucial for everything that we’re trying to do. We don’t make any progress without clinical trials. So, that’s the field as a whole. We don’t move forward. We don’t get any of these new treatments without clinical trials.
On an individual patient level, clinical trials are also really important because, for many patients we are still not doing as well as we want to be doing with this disease. We’ve made progress, but there’s still a lot of room for improvement. And so, for an individual patient, getting access to another therapy that, although we admit we don’t quite know yet whether it may be helpful but might be helpful, I think, is a really compelling situation to potentially consider participating because it is a guarantee you will help the field; and it’s a guarantee you will help every patient that comes after you through participation in clinical trial.
But all these clinical trials are also designed to help you; to help you in a situation where we as a field don’t feel like we’re doing well enough. So, clinical trials, totally crucial if we’re going to continue making progress.
And clinical trials are the reason why these last 10 years we have had such just dramatic improvement in availably of all these new therapies because literally thousands of patients have chosen to participate.
Katherine Banwell:
How can patients find clinical trials that might be right for them?
Dr. Daniel Pollyea:
So, back to The Leukemia & Lymphoma Society. They can be really helpful in guiding this. Asking your doctor, “Hey, are there any clinical trials her or at any other center that I should be considering?” And then, people who are interested in just going to the source. Every clinical trial that is available is registered at clinicaltrials.gov. And so, going to clinicaltrials.gov and then putting in some keywords like “acute myeloid leukemia,” you’ll see every clinical trial that’s available.
Katherine Banwell:
Oh, that’s excellent. I’d also like to add for our viewers that if you’re interested in learning more about AML care and treatment, PEN has a number of resources available to you.
You can find these at powerfulpatients.org/AML or by scanning the QR code on your screen.
So, Dr. Pollyea, when choosing a therapy what questions should patients be asking their healthcare team about a treatment plan?
Dr. Daniel Pollyea:
So, at the time of diagnosis I think it’s a reasonable question to say, “Is my disease amenable to a cure? Can I be potentially cured?” and “Is this treatment part of a plan for a cure?” If that is possible, then I would want to be walked through the steps that that’s going to executed. And if it’s not possible for me to be cured, then I would like to discuss what is the treatment plan that could potentially give me the longest duration of a remission and the best quality of life. And so, that’s the conversation that I think is important to have.
And then, everything that we discuss comes into play there; an individual’s sort of appropriateness for intensive chemotherapy versus less intensive regimens, and also the disease biology and what that maybe make them a candidate for.
Katherine Banwell:
Are there certain symptoms or side effects a patient should share with their care team?
Dr. Daniel Pollyea:
Yeah. So, we have a very, very sort liberal request that really anything, it should be shared. We have a 24/7 number to call with one of us on-call at all times. So, it’s very difficult for a patient to kind of be able to appreciate, when they’re going through such dramatic changes, “Hey, is this expected or not?” So, we really emphasize oversharing concerns about symptoms.
All these drugs have very different side effect profiles, and some of them are common and some of them are less common. The disease itself can cause symptoms and clinical issues. So, instead of really trying to educate yourself in an impossible way on what could be or is not related, it’s better just to ask.
Katherine Banwell:
What is the role of a care partner when someone is in active treatment?
Dr. Daniel Pollyea:
Having a care partner is crucial. This is physically and mentally extraordinarily stressful on the body and on the mind. Having that support person for those purposes is really important. Having that person be an advocate for a patient to ask those questions that may not be getting asked, to reframe questions to get the best answers is really, really important.
And then, there’s the more mundane things; just getting patients to their appointments and kind of keeping their morale up and those things. So, there’s data and research on this that patients with caregivers, they have better outcomes. When it comes to a transplant, a caregiver is not an option. You must have a caregiver. And the importance of that will be sort of relayed to you in the context of a discussion about a transplant. But a caregiver in the setting of a transplant is so important that it is a requirement to even be considered for that.
Katherine Banwell:
Sounds like that’s vital. I’d like to get to a few audience questions that we received before the program. Chris sent in this question: I would like to hear more about mutations found during molecular testing. Are there new AML drugs in trials for other less common mutations?
Dr. Daniel Pollyea:
Great question. So, at the moment, what we have clinically available are targeted therapies for patients with FLT3 mutations, IDH1, and IDH2 mutations.
And there are about 50 different genes that can be mutated in AML, and so that’s a small slice of the pie. Those are relatively common mutations, but still, small slice of the pie. A lot of the very uncommon or less common gene mutations we don’t have great paths to targeted therapies for them. And is that just we never will? I don’t think necessarily, but I think those can be really challenging. Not every mutation is amenable to a targeted therapy, at least as far as we know now. The one that’s coming, that we’re hopeful about is NPM1, which may be able to be targeted with one of those menin inhibitors that we talked about. So, that’s the next big one up.
And that will probably constitute 40 percent of patients that have one of those mutations that I listed. But research is ongoing to kind of try and dig into this more. What I will say is that the AML research community is so fantastic that every lead is being pursued, and there is a lab somewhere in the world whose focus is on whatever small, even the most least common AML mutation; that’s somebody’s focus.
And so, if there were to be promising therapies developed for even rare mutations, I assure you, the field would take those forward and figure out a way to do those clinical trials and to get to approval if it’s appropriate. So, but I think that’s where the landscape is right now.
Katherine Banwell:
This question comes from Rita: Outside of changes in bloodwork, what are signs that AML is returning?
Dr. Daniel Pollyea:
Great question.
So, this can be a really tough one, and bloodwork is what we sorta hang our hat on. There are some times that patients sort of have clinical symptoms that proceed changes in bloodwork. I will say, I find that to be pretty uncommon. But some of the things that are pretty rare but might happen, would be leukemic involvement of the skin; so, it would appear as a rash. Some people might have some fatigue that comes on before the blood counts really change. That’s also pretty rare.
And then, if this disease were to work its way into any other organ or tissue in the body, and that’s rare, it’s possible that that could present with clinical signs and symptoms before a blood count change. But for the most part, the blood counts are really early sign that something is changing, and typically we’ll see that before any clinical signs.
Katherine Banwell:
Thank you for that, Dr. Pollyea, and those were great questions. Please continue to send them to question@powerfulpatients.org, and we’ll work to get them answered on future programs. So, as we close out the program, Dr. Pollyea, what would you like to leave the audience with? Why are you hopeful that about the future of AML care and treatment?
Dr. Daniel Pollyea:
Well, we’ve made unbelievable progress in just the last 10 years. And so, just looking into the future, I see nothing stopping that progress. So, it’s really exciting to think about where we’ll be two, five, 10 years from now. We never could have envisioned 10 years ago where we are now in terms of the therapies we have, how active and effective they are, and the impact that it’s had on patients.
Again, just so proud to be part of this community, both on the patient care side and on the research side. It’s such a committed group of people, working around the clock on this disease to figure it out and to make some improvements. For all those reasons, I’m just super hopeful that we’ll just keep making progress, and I see no signs of anything slowing down.
Katherine Banwell:
That’s a promising outlook to leave our audience with. Dr. Pollyea, thank you so much for joining us today.
Dr. Daniel Pollyea:
Thanks so much for having me.
Katherine Banwell:
And thank you to all of our collaborators. To learn more about AML and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell. Thanks for being with us today.
