What does the future of myeloproliferative neoplasm (MPN) care look like for patients? Expert Dr. Idoroenyi Amanam from City of Hope explains how MPN treatments have changed in recent decades, symptoms that are relieved with treatments, and how treatments of the future may help patients.
Dr. Amanam, what promising treatments are available for newly diagnosed MPN patients, and what questions should patients be asking? They come into your office scared to death and not even knowing what to ask. Do you have any suggestions for what those patients should be asking when they go in as a newly diagnosed patient?
Dr. Indoroenyi Amanam:
Right, right. Twenty years ago, we really didn’t have any therapies for most MPN patients, aside from performing phlebotomy and using non-specific therapies to try to help control their counts and therefore reduce their risk of clotting and stroke. We are getting to a point which is really exciting, where we actually are treating the underlying disease, meaning that the cells that are causing this cancer, we have been able to identify targets that will help eradicate those cells and, therefore, get rid of the cancer. And so we’re getting there. Unfortunately, we still are not there yet, and so when we look at the FDA-approved drugs in this space, really, they help control symptoms, they help control some of the associated complications with the disease, mainly when your spleen’s enlarged, and that potentially may affect your quality of life, mainly your nutritional status and your physical status, and so we do have drugs that are able to do that, that are FDA-approved right now.
I think in the next three to five years, we’re going to have drugs that are going to actually be able to treat the underlying disease before it gets to a point where you may need more aggressive therapy. Currently, the only defined curative therapy that we have, when I say defined, meaning that we have multiple studies that have shown that that’s the case, is bone marrow transplant. I’m a bone marrow transplanter, I do treat some of my MPN patients with bone marrow transplant to get rid of the underlying…those underlying cells that are driving this disease. But that’s a very intense therapy and it has its own associated complications. But we are…will be having other drugs that potentially we would be able to offer that are not as intense as bone marrow transplant.
And those include immunotherapy, other drugs that can target the signals that drive these cells to divide and multiply. Also there are within the bone marrow for patients that have myelofibrosis, which is one of the MPNs, we will be able to target the environment that allows for these cells to persist and grow. And so it’s exciting where we’re going, and I think the questions that as a patient that I would ask are, because of the fact that we only have few FDA-approved therapies, are there any clinical trials that are able to target the underlying disease as opposed to just treating the symptoms? I think that’s very important for the patients to ask, especially in this space now.