Tag Archive for: FL3 mutation

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.
 
 
Related Resources:
AML Therapy | Emerging Treatments and Clinical Trials

AML Therapy | Emerging Treatments and Clinical Trials

An Overview of Current AML Treatment Types

An Overview of Current AML Treatment Types

AML Treatment Planning | Key Questions to Ask You Doctor

AML Treatment Planning | Key Questions to Ask You Doctor

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 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:

AML Gene Mutations | Emerging Targeted Therapies in Development

AML Gene Mutations | Emerging Targeted Therapies in Development

An Overview of Current AML Treatment Types

An Overview of Current AML Treatment Types

AML Treatment Planning | Key Questions to Ask You Doctor

AML Treatment Planning | Key Questions to Ask You Doctor

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.