What emerging myeloproliferative neoplasm (MPN) therapies are in the research pipeline? Expert Dr. Idoroenyi Amanam from City of Hope discusses MPN treatments that are under study, what the therapies target in MPN patients, and the outlook for the future of MPN care.
Dr. Amanam, can you speak to any exciting new developments in MPN care or trials that you see moving forward with great progress?
Dr. Indoroenyi Amanam:
Yeah. I think for MPNs and namely really the classic BCR-able or Philadelphia chromosome-negative MPNs, which include essential thrombocythemia, polycythemia vera, and myelofibrosis. I think we have a lot of exciting therapies that are going to be possibly FDA-approved in the next couple of years. So currently, for essential thrombocythemia, really the dogma therapy is related to keeping the counts under control and giving a therapy to reduce the risk of having a blood clot or stroke. We actually are in a space where we have therapies that are going to be targeting the underlying clone or basically the cells that are driving the proliferation of these platelets that lead to high platelet counts. And so I think that’s exciting.
So we do know that, in MPN there is an overexpression of Bcl-xL, and there’s a drug that targets Bcl-xL. And we’ve seen really great responses in essential thrombocythemia. And as a segue, this drug also targets the same cells and polycythemia vera and myelofibrosis, and we’ve seen really great responses in those patients. We also have had difficulty in managing patients who have myelofibrosis, but have very low counts. And typically the FDA-approved drugs that we’ve been using actually make the counts worse.
And so there are multiple drugs that are in the pipeline that are helping patients with low blood counts. And what they do is they help increase your red blood cells and reduce your requirements for red blood cell transfusions.
And one of the drugs helps stimulate erythropoiesis, and it’s an injection. And we’ve seen really good results in reducing the risk of…or reducing the amount of transfusions that patients receive. And then another one of these drugs targets ACVR1, which we understand that in myelofibrosis, you have overproduction of hepcidin, which leads to worsening anemia. And so by targeting ACVR1, it helps control this hepcidin. And by doing that these patients have improved red blood cell counts. And so that’s another drug that likely will be coming…that will be FDA-approved very soon, and I think will help patients in this space.
We also are interested in immunotherapy. And I think in other cancers, immunotherapy has been very successful in eradicating those cancer cells and curing some patients. And so there are clinical trials looking at a vaccine which targets certain mutations that are relevant to MPN patients. And also we are interested in actually using other types of immunotherapy namely, CAR T, which really helps connect your own immune cells to these cancer cells to help clear them out. And so I think over in the next five to 10 years, there’re going to be a lot of drugs and a lot of therapies that are going to really help patients who have MPNs.