What are some promising treatment options or clinical trials for AML patients?
A lot of them are things that are built on top of hypomethylating agents, right, the hypomethylating agent. And a standard one is called azacitidine, and then you can put other medicines on top of that. One that's approved now is something called venetoclax. So people oftentimes get a combination of both azacitidine and venetoclax that works much better than existing by itself. So there are a lot of other things that people are thinking of that modulate the immune system or regulate how genes are turned off or on. Those things are combined with the hypomethylating agent, and that's a big area. The other is a big area of like immunotherapy. Can we use the immune system to try and attack the MDS, just like we do in allogeneic bone marrow transplant, just like we do with CAR-T cell therapy for lymphoma or myeloma? It's been very difficult to find good immunotherapies for MDS. And part of it is that the immune system doesn't recognize MDS versus the rest of your normal bone marrow. And so if we get something that kills all the MDS, we're probably going to kill all the normal cells that make blood. So that's the side effect, and that is potentially lethal. So that's the biggest challenge in developing immunotherapies for AML or MDS is that we need something to hit the cancer but leave the normal ones behind. In an allogeneic transplant, it doesn't matter because we're giving you new cells. So we're replacing it. But in us just giving you something to stimulate your immune system, that has been very difficult. But there are a lot of promising things that people are looking at.
There are a number of promising clinical trials for patients with AML. We divide AML into patients who are younger and able to get intensive chemotherapy and those who are older. And we have to give a little bit more of a gentler regimen, too. Recently, in the last five years, the treatment of older AML patients has been revolutionized by the adoption and approval of venetoclax, which is an inhibitor of a molecule that helps prevent cells from dying. And that has greatly increased the responses of patients who have AML. In patients who receive venetoclax in combination with the hypomethylating agent, which is usually azacitidine or decitabine, there are now several clinical trials of the addition of a third drug to try and improve the response rates and survival time even further. In patients who are younger with AML, we are looking at several drugs in addition to the standard strong chemotherapy that we give to try and improve outcomes.
HealthTree • September 30, 2024
