Tag Archive for: clinical trials.gov

Emerging AML Treatments: What Is Menin Inhibitor Therapy?

Emerging AML Treatments: What Is Menin Inhibitor Therapy? from Patient Empowerment Network on Vimeo.

How does menin inhibitor therapy work to treat acute myeloid leukemia (AML)? Dr. Sanam Loghavi discusses how this novel targeted therapy in clinical trials is showing promise for patients with the NPM1 mutation or the KMT2A mutation. 

Dr. Sanam Loghavi is a hematopathologist and molecular pathologist at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Loghavi.

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Katherine Banwell:

Let’s talk about this new groundbreaking menin inhibitor therapy. Can you go into more detail about what the therapy is and who it might be right for? 

Dr. Sanam Loghavi:

Sure. So, right now, the drug really has been tested in the setting of relapsed refractory disease, meaning for patients whose disease has already been treated but has relapsed. And there are certain genetic subtypes of acute myeloid leukemia that are eligible for this disease, or unamenable, sorry, to this targeted therapy. So, these include acute myeloid leukemias with NPM1 mutation or acute myeloid leukemias with KMT2A, or formerly known as the MLL gene-rearrangement. 

And the reason for this that these alterations, these genetic alterations lead to an apparent interaction of menin with KMT2A and the leukemia depends on this interaction. So, what the Menin inhibitor does, it eliminates this interaction and so it’s used for therapy in patients that have this genetic change. 

Katherine Banwell:

Are there other menin inhibitors in development? 

Dr. Sanam Loghavi:

There are. 

Katherine Banwell:

And what are they? 

Dr. Sanam Loghavi:

There are several specific ones that are being tested of different names. So, the one that MD Anderson just published on is revumenib, but there are several ones that are in development. 

Katherine Banwell:

And what about these other inhibitors are showing promise? 

Dr. Sanam Loghavi:

So, if you think about AML, in general, really the only curative therapy that we have, outside of the favorable risk disease, is hematopoietic stem cell transplant. 

And hematopoietic stem cell transplant is not a trivial treatment, it has a lot of side effects in and of itself. So, the goal really is to be able to treat patients with less intensive therapies. And the goal of these targeted therapies is to provide patients with less intensive therapies even compared with chemotherapy, with conventional chemotherapy that tends to be toxic. So, the goal is really to be smart about it and try to figure out how the pathogenesis of disease is developed and to try and eliminate the pathways that that cancer is using to proliferate. 

Katherine Banwell:

If patients are interested in this menin inhibitor therapy, where do they start? Are there trials outside of MD Anderson? 

Dr. Sanam Loghavi:

Yes. These are multi-institutional trials, and I will tell you that the best resource to identify clinical trials is essentially clinicaltrials.gov, dot G-O-V. So, you can go there and look up the active clinical trials by disease type, by location. So, that is the best resource to identify clinical trials.  

Expert Advice for Finding an MPN Clinical Trial

Expert Advice for Finding an MPN Clinical Trial from Patient Empowerment Network on Vimeo.

Dr. Andrew Kuykendall, an MPN specialist and researcher, shares tips for learning about available clinical trials. Dr. Kuykendall emphasizes the importance of seeking a consultation with a specialist and suggests questions to ask your provider about clinical trials.

Dr. Andrew Kuykendall is an Assistant Member at Moffitt Cancer Center in the Department of Malignant Hematology. Dr. Kuykendall’s clinical and research efforts focus on myeloproliferative neoplasms (MPNs), MDS/MPN overlap syndromes and systemic mastocytosis (SM). Learn more about Dr. Kuykendall, here.

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How can patients find out about clinical trials? Are there specific questions that they should be asking their doctors about to participate in a trial?

Dr. Kuykendall:

Yeah. I think it’s tough. One way – there are a few different tools that I would recommend. One, if you’re very interested in just what trials are going on you can go to this national cancer trials, or NCT, network and try to understand online what trials are available. Clinicaltrials.gov is the actual website but that’ll show you the ongoing clinical trials that are there.

You can type in a disease state, so you can type in polycythemia vera or myelofibrosis or essential thrombocythemia, and it’ll give you a huge list of all the trials that are there. It can be kind of overwhelming because it’ll list all of the trials that have ever been done, but there are different ways that you can stratify those results and look for trials that are just recruiting that are active and that’ll taper down that list. And when you click on those trials there usually is at the bottom a list of participating centers that are there. So, you can see the different centers that are there. Overall, I think that that is a very broad way of doing it and somewhat complicated.

What I would ask is – and one of the things that we always push for is – while most of these myeloproliferative neoplasms can be treated quite easily in the community, meaning that the actual mechanisms of what’s being provided is not something that requires a specialized center. I think the understanding of the disease really does. We always recommend having someone in your corner who’s an expert. They don’t have to be the one who is most involved in your care but having someone in your corner who’s an expert.

That’s the person who’s going to know what trials are going on, what trials may be coming down the pipeline, where those trials may be occurring, and they might also tell you “Okay, here are the things that would prompt you to maybe want a trial.” I had a lot of patients that were surprised to realize there were trials available just because they had – they were getting six or seven phlebotomies a year. They were complaining about that but they figured that was just the ways things were. Lo and behold, there was actually a trial that was ongoing that was trying to reduce the need for those phlebotomies in otherwise low-risk patients.

You can always go to clinicaltrials.gov but also try to ask your doctor about hey is there, if you haven’t seen an expert, is there someone close by an expert that I can see for a second opinion just to understand the disease and ask about trials. Usually everyone’s okay with that and when you do see an expert, say “Hey, first of all what trials are right for me now and what in the future might be reasonable and how am I going to know and how often should I check in to see what things are available?”