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.
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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.
