AML Research: What’s New in Treatment?

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 AML expert, Dr. Jessica Altman, discusses the future of AML research, and new learnings that continue to improve current treatment approaches.

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University. More about Dr. Altman here.

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Transcript:

Patricia:            

Are there any new treatments on the horizon that you can talk about, Dr. Altman?

Dr. Altman: 

Absolutely. So, I love to talk about new therapies in AML. Until the last couple of years – it had been 40 years since we approved a sustained treatment in the marketplace in AML. We had been treating the disease the same. And over the last couple of years there have been a growth of therapies. We’re now trying to sort out exactly when we’re using one over another. We also have clinical trials where we’re combining novel therapies for adults with either newly diagnosed disease or relapsed and refractory disease. 

We are in an era of looking out at antibody therapy in AML – that’s one of the new waves of treatment. We are still exploring targeting therapies in the sense of inhibition of FLT3, IDH, and other mutations. So, it’s an era where there’s lots of excitement, and I’m hopeful for our patients.

Patricia:     

Yeah. Tell me what makes you most hopeful about the future of research in this area, and treatment?

Dr. Altman: 

So, I think that’s a great question. I think the fact that we now – the deeper the understanding we have of the biology of the AML, why AML happens, what mutations drive the disease, and then how to target those mutations with individual therapies is what excites me the most. So, our basic science research has exploded, and that occurs at a very quick pace, and that’s allowing us to develop therapies at a much faster rate than I would have anticipated before.

Patricia:

What a wonderful way to end our chat. Thank you so much, Dr. Altman, for taking the time to join us today.

Dr. Altman: 

It’s a pleasure to be here. Thank you so much.

Misconceptions in Clinical Trials: What’s Fact and What’s Fiction?

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AML expert, Dr. Jessica Altman, addresses common misconceptions patients have about clinical trials regarding treatments, regulations, and standards of care. Want to learn more? Download the Program Resource Guide here.

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University. More about Dr. Altman here.

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Transcript:

Patricia:            

What about clinical trials? What common misconceptions do patients have about enrolling in trials?

Dr. Altman: 

So, I think the misconceptions regarding clinical trials can be very masked. And I think it really depends on the intent of a clinical trial and the phase of the clinical trial. I think that a well-designed clinical trial is almost always the right choice for a patient with acute leukemia at any stage in their therapy. 

That is a bias as a clinical trialist. I think it’s the right bias, but it is still my bias. I think patients frequently worry that they’re being treated as a guinea pig, or they’re not getting an appropriate treatment. What I can tell you is the clinical trials that we and my colleagues across the country and across the world participate in are clinical trials where the patients are getting at least what we consider a standard of care for that phase of their disease, and they may be getting something in addition to that or something that is slightly different, but expected to have a similar response rate. 

We have this phrase in clinical trials, something called equipoise, that if there’s a randomization between options that we need to feel, as the practitioner and as the clinical trialist, that each option is at least as good as the other.  

Patricia:

That kind of goes back to the vetting of treatments before they go to a clinical trial. Tell me a little bit about history. How can we make patients feel more comfortable?

Dr. Altman: 

I want to make sure that I understand the question.

Patricia:

So, how thoroughly are treatments vetted before they go to a clinical trial?

Dr. Altman: 

Great. So, the way that agents get into early phase clinical trials and then later phase studies are these are compounds that have been studied in the laboratory, then studied in small animals, then larger animals. And then, frequently, a drug is started in a patient with relapsed and refractory Acute Myeloid Leukemia and found to be safe – that’s what we call a Phase I study. 

Once we know the right dose and the associated side effects from an early phase clinical trial, later phase studies – i.e. Phase II, where the goal is to determine the efficacy and response rate is conducted. And then, if that appears and looks like it’s promising, a larger, randomized, three-phase study is frequently conducted, where we compare a standard of care to the new approach. 

Patricia:

So, patients should be comfortable that the clinical trial that they’re going through has been thoroughly vetted, has gone through multiple stages before human trials occur?

Dr. Altman: 

That is accurate in terms of compounds get through animal studies, and then depending on the way that the trial is being connected, will then be studied in patients either with relapsed or refractory disease or very high-risk disease. But it’s also very important to mention that these pharmaceutical companies and physicians are not making these decisions alone. 

The clinical trials are all reviewed by scientific review committees through the cancer centers, which are other investigators making sure that everything appears appropriate. In addition, there are institutional review boards at every university whose goal it is to keep patients and research subjects in well-done clinical trials safe. That is their primary goal. And the IRBs – institutional review boards – are very involved with making sure that clinical trials are appropriate and that the conduct of clinical trials is appropriate.

Addressing Common Myths About AML Treatment

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AML expert, Dr. Jessica Altman, discusses common myths surrounding available AML treatment options, stem cell transplant and how leukemias are classified.

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University. More about Dr. Altman here.

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Transcript:

Patricia:            

Dr. Altman, let’s talk about some AML treatment myths floating around. I’ll throw some stuff out there, you let me know if you’ve heard this. “Leukemia is one disease.”

Dr. Altman: 

So, I have heard that. Leukemia is actually a number of different diseases, and it’s very heterogenous. There are acute and chronic leukemias. The acute versus chronic really depends on a couple of factors. The biologic factor is the presence or absence of 20% loss or more in the bone marrow, but that also coincides with how patients present clinically. Acute leukemias tend to present more acutely, more rapidly. And chronic leukemias tend to be a bit more indirect. And the treatments are very different for those entities. 

There are also myeloid or lymphoid leukemias, so there’s Chronic Myeloid Leukemia and Acute Myeloid Leukemia and Chronic Lymphocytic Leukemia and Acute Lymphoblastic Leukemia. So, those are the four major categories. We’re talking about Acute Myeloid Leukemia today. Within Acute Myeloid Leukemia, there are multiple different types of Acute Myeloid Leukemia that are really now best categorized by history – patient history – and the molecular and cytogenetic abnormalities of the disease. 

Patricia:

Now, we’ve already learned about a bunch of them. So, “There are limited treatment options” is definitely a myth. Correct, Dr. Altman?

Dr. Altman: 

So, we have had a major growth of the number of treatment options available for Acute Myeloid Leukemia really in the last couple of years. It’s been a very exciting time for practitioners and for our patients that we have now a number of new therapies. So, there is not just one treatment available. In fact, the conversation regarding treatment options becomes quite extensive with patients and their families, because there are choices. And that’s why consideration of goals in the intent of treatment becomes even more important. 

Patricia:

Here’s another one: “Stem cell transplant – the only chance for cure.”

  Stem Cell Transplant, also called a bone marrow transplant, is a procedure in which healthy blood stem cells are used to replace damaged or diseased bone marrow. This procedure can be used to treat certain types of blood cancers.

Dr. Altman: 

Okay. So, that is also a myth. There are certain types of Acute Myeloid Leukemia where stem cell transplant is the most appropriate treatment once the disease is in remission if the goal of the patient is of curative intent. Stem cell transplant is not appropriate for every individual, and for some types of Acute Myeloid Leukemia, stem cell transplant is not considered. 

Patricia:

What kinds of things do you think about when you’re considering a stem cell transplant with a patient? 

Dr. Altman: 

So, again, I go back to patient goals and understanding their goals of treatment. A stem cell transplant is among the most medically intensive procedures that we have. It is also not just a treatment that occurs over a short time. While the actual transplant is a relatively limited hospitalization and the administration and infusion of stem cells and preparative chemotherapy, it is something that can continue to have side effects and alterations in life quality that can persist for months to years afterwards. 

So, that’s one aspect of things that we talk about regarding stem cell transplant. And really understanding what the benefit of transplant is in terms of a survival advantage, versus what the risk and the cost in terms of toxicities are. And that’s the basis of a lot of the conversations we have.

Patricia:

Sure. Here’s one more: “AML patients require immediate treatment.”

Dr. Altman: 

Sometimes AML patients require immediate treatment, and sometimes they don’t. And that depends on the biology of the disease. How high is the white blood count when the patient comes in? What are the best of the blood counts? Is the patient having immediate life-threatening complications of their acute leukemia? 

And there’s some forms of acute leukemia that require immediate therapy to prevent complications, and there’s some forms of acute leukemia who present an extreme distress from their disease, but there are many patients who present with acute leukemia, and we have time to get all of the ancillary studies back – the studies of genetics and the molecular studies1 – to help further refine the conversation, and further design an appropriate treatment strategy. 

Patricia:

What else? What do you hear from your patients that you feel is maybe a misconception or something they’re not quite understanding about the AML?

Dr. Altman: 

So, I think one of the biggest things that I would like to mention is that response rate and cure are not the same. So, it is possible for one to be treated for Acute Myeloid Leukemia and the disease to enter remission, and yet still not be cured of their disease. 

Acute Myeloid Leukemia is a disease that frequently requires additional cycles of treatment or a stem cell transplant after the initial induction therapy to be able to have the best chance for a long-term cure. So, response and cure are not the same thing.

Valerie share’s her story for AML Awareness Month

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This video was originally published by CancerCare on June 17, 2016, here.

 

AML Genetic Testing: Could It Lead to a Targeted Treatment for You?

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AML Genetic Testing: Could It Lead to a Targeted Treatment for You? from Patient Empowerment Network on Vimeo. 

AML expert, Dr. Pinkal Desai, outlines the reasoning behind the necessity of cytogenetics and molecular testing when managing an AML diagnosis. Want to Learn More? Download Your AML Navigator Resource Guide, here.

Dr. Pinkal Desai is an Assistant Professor of Medicine at Weill Cornell Medical College and Assistant Attending Physician at the New York-Presbyterian Hospital. More about this expert here.

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Transcript:

Dr. Pinkal Desai:         

So for patients who are undergoing molecular testing or any diagnosis of AML, both cytogenetics and molecular profiling are important, so they do not supersede each other. This is the conglomerate information that we need from the diagnosis to make important medical decisions. Usually the diagnosis would include: looking at the cells under the microscope by the pathologist; flow cytometry, which is a way to identify the subtype of leukemia; chromosomes or karyotypic analysis, which is to look at the individual chromosomes and whether they are abnormal in these leukemia cells; and the last one would be the molecular mutations, which would be single-gene profiling of the leukemia cells.

All of these are important, and it’s not that one can be omitted. They’re all part and parcel of the diagnosis of AML, and all of them should be done.  

So my advice to patients whenever this topic comes up of molecular mutations is always an unequivocal – there should be no question that this should not be done. The advice is plain and simple. This has to be done at diagnosis and, in certain cases, at relapse as well in order to figure out the best treatment possible. If they’re at a site or a clinic where this molecular testing is not available, then they should seek a second opinion to a site that would do this testing because in this day and age of leukemia, there is no treatment and diagnosis that can be done without all of these components in place.

In the old days, we didn’t have a lot of treatment in AML. It was either chemotherapy or hypomethylating agents, and that’s it. But now we have several drugs, five or six of them, that were just approved in the past two years specifically for leukemia and targeting some of these mutations. We have Midostaurin, Gilteritinib, Ivosidenib, Enasidenib, and I don’t want to go on and on about these drugs, but the most important thing is that in this day and age where you have so many drugs, how to incorporate these drugs into the management for patients, both upfront and in the relapse setting, it’s extremely relevant to do this testing, and this is highly encouraged and should be done as part of the diagnosis and treatment.

What’s Next in AML Treatment and Research?

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What’s Next in AML Treatment and Research? from Patient Empowerment Network on Vimeo.

Dr. Pinkal Desai, an AML specialist, discusses research in-progress on MRD testing and pre-disease mutations in leukemia.

Dr. Pinkal Desai is an Assistant Professor of Medicine at Weill Cornell Medical College and Assistant Attending Physician at the New York-Presbyterian Hospital. More about this expert here.

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Transcript:

Dr. Pinkal Desai:         

So we at Weill Cornell are a big leukemia center, and we are leading a lot of the clinical trials in AML, both in the upfront and the relapse setting. There are several research initiatives that we are highly interested in. One of them is how to incorporate some of these targeted treatments, both in the upfront and in the relapse setting.

The most important one that we’re actively working on is to monitor these patients, so MRD testing, or minimal residual testing, is extremely relevant in order to figure out whether the treatments are working in the right fashion, and would you change treatment or would it impact the patient’s overall survival if some of these mutations persist or not.

And we are really interested in monitoring these patients and these mutations to figure out a plan which is targeted not only for the mutation but also for the specific patient, and that is one of the things that we are very interested in and doing at Cornell.

We’re also looking at pre-disease mutations. There are several mutations – this is personally my research interest as well – there are certain people who are at risk of developing leukemia; for example, people who are undergoing chemotherapy for other cancers, and the presence of some of these mutations before the diagnosis of leukemia would highly be relevant because if we’re monitoring some of these people and figuring out who can develop this leukemia and can you do something about it, so this is sort of more on the prevention aspect of leukemia or secondary leukemia, which is also something we are interested in at Cornell and ongoing research is for us.

But the most important things is obviously for patients who actually have the diagnosis of AML, the best available agents as part of clinical trials, the best way to monitor them and design treatments so that we can achieve the best possible results for the patient is what we are striving for at Cornell, and it would be extremely helpful for patients to enroll into these trials and contribute both to their own treatment outcomes and also to the AML community at large.