Where Is AML Research Headed Next?

Published on

Topics include: Treatments

Researchers have made great strides in understanding the genetic components of AML and the key mutations to watch for. What is the focus for the future? How will these breakthroughs impact patients? Tune in to find out from leading experts, Dr. Lee Greenberger, from The Leukemia & Lymphoma Society (LLS), and Dr. Amit Verma, from the Montefiore Medical Center.

This is a Patient Empowerment Network program produced by Patient Power, in partnership with The Leukemia & Lymphoma Society (LLS). We thank Astellas, Celgene Corporation, Novartis, Pfizer and Seattle Genetics for their support.

View more programs featuring , and

Produced in association with and

Transcript

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

Andrew Schorr:

We're going to meet the first of our experts now, and that is Lee Greenberger, who is the Chief Scientific Officer at our partner, The Leukemia & Lymphoma Society, and he joins us from Westchester County, New York, and he's the Vice President with the LLS as well.  Lee, thank you so much for being with us. 

Dr. Greenberger:

Pleasure to be here. 

Andrew Schorr:

And so, Lee, you have a lot of research underway that you helped guide and make grants from the LLS for.  AML is an important research area for the LLS, isn't it? 

Dr. Greenberger:

I'd say it's a major area of research.  So we have about 230 active grants placed globally, and about 30 percent of them are focused on new therapies for AML and understanding the basis of the disease. 

Andrew Schorr:

Okay. And understanding the basis, and that can vary, or the specific characteristics of AML can vary by patient, right, related to genetics?

Dr. Greenberger:

Yeah. So no question about it.  First of all, what we have found is that AML is just not one disease. 

It's probably about 20 or 30 diseases, and beyond that the way you classify AML used to be on looking at the cells and looking at the chromosomes.  Now we know that there are many mutations in this disease, and that further subdivides the patients based on prognosis, and ultimately therapies apply to the mutations that are observed. 

And this is happening right now.  I should add that some of this work goes back 20 years.  With the advancement of being able to find the mutations and then looking at all the genes and doing it quickly and economically, this has taken many, many years.  And then to develop the drugs that actually target those mutations also takes many years.  So what we're seeing is fort of a convergence of understanding the molecular basis of the disease, and then ultimately we hope to apply therapies to those molecular defects. 

Andrew Schorr:

Right. And that's starting to happen.  There were a number of approvals of drugs in spring of this year, 2017, and a lot of research underway. 

Well, let's meet somebody who's in the clinic, if you will, and that's Dr. Amit Verma, who joins us from Montefiore Medical Center in Bronx, New York, just down the road from where Dr. Greenberger is.  Dr. Verma, welcome to the program.  

Dr. Verma:

Thank you, Andrew. 

Andrew Schorr:

Dr. Verma, so you're the director of the MDS program in the Division of Hemato-Oncology there in Montefiore, and here we have Kuldip, who had MDS.  Just to understand, you have a lab as well as you see patients.  Is this testing now to understand what version of AML a patient has critical really for them to get the right treatment? 

Dr. Verma:

Yes. You know, Andrew, like Dr. Greenberger said, the knowledge of what mutations can cause MDS and AML has really transformed our field. 

Instead of just relying on physical appearance of how the cells look, how the bone marrow looks, and then we used to decide on which chemotherapy to give, how much to give.  We now can use knowledge of what mutations are seen exactly for each patient.  And for some of these mutations we do have medicines that are supposed to act specifically for these exact mutations.  So we have already started using some of these drugs.  There have been two approved this year, which are approved only for specific mutations that are seen in MDS and AML.  

Andrew Schorr:

Okay. And more in your researching others as well. 

Dr. Verma:

Absolutely. So the way this started was technology actually helped us a lot. 

We have thousands of genes in our body, and it was very tough, you know, a few years back to sequence all of them for a particular patient.  It used to take months and months and thousands of dollars.  Now we can do whole genome sequencing in a matter of days at a fraction of the cost, and when they did these sequencing projects they figured out that in MDS and AML there were about 20, 30 genes that were mutated most of the times. 

So now these have been used to build platforms, you know, by commercial companies and by academic hospitals, where as soon as we have suspicion for MDS and AML we send a sample for testing.  We get the results back in about five to seven days, and then we use it for approved medicines.  But as you rightly mentioned, we also can use these mutations to guide patients into clinical trials for drugs that are still in clinical research but are not yet FDA approved.  From a basic lab research point of view we are always trying to figure out how does a particular mutation lead to the causation of disease.  And we do it using human cells.  We use mouse mortals of leukemia.  We use, you know, all different kinds of models to test that—let's say that you discover a specific mutation that doesn't yet have a drug, but how can we use the existing library of drugs to kill these cells or to study how this mutation uses other parts of the body to cause disease. So a lot of research has come about after the discovery of these mutations.

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

Related Programs

Webinar Replay: AML Genetic Testing and Me

Watch the webinar replay of AML Genetic Testing and Me, as an expert panel discusses essential genetic testing for AML patients.

Published:

Webinar Replay: Finding the Right AML Treatment for Me

What is personalized medicine, and how can it help AML (acute myeloid leukemia) patients? At the 2017 ASH (American Society of Hematology), AML experts share an update.

Published:

Targeted Therapy: Finding the Right Treatment Option for Your AML

With several newly approved AML treatments, how do you know which is right for you? Dr. Michael Deininger discusses current AML medicines and ones on the horizon.

Published:

Advertisement
Join Our Community Register for Events Read Our Latest Blog
Advertisement

Page last updated on September 3, 2019