What are the latest research advances for essential thrombocytosis (ET), polycythemia vera (PV), and myelofibrosis (MF)? Myeloproliferative neoplasm (MPN) expert Dr. Shivani Handa reviews emerging therapies and innovative research that is opening the door to more effective, potentially disease-modifying treatments.
Dr. Shivani Handa is a hematologist-oncologist at the Ohio State University Comprehensive Cancer Center. Dr. Handa specializes in myeloid malignancies, which includes myeloproliferative neoplasms, myelodysplastic syndrome, and acute leukemias. Learn more about Dr. Handa.
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Transcript
Katherine Banwell:
Dr. Handa, what are the latest advances in treatment and research for essential thrombocythemia?
Dr. Shivani Handa:
So, one results from the SURPASS-ET study which looked at ropeginterferon alfa-1a (Besremi), which is a molecule that’s already been approved for polycythemia vera patients.
And this was a Phase III study that showed that – it basically compared interferon with anagrelide which is another antiplatelet therapy used for essential thrombocytosis, and it far outperformed anagrelide. So, it’s being currently reviewed by the FDA, and hopefully we’ll give an expanded label to interferon use in essential thrombocytosis. The other agent that’s being looked at is an LSD1 inhibitor called bomedemstat. That’s in clinical trials for ET and showing promising results. There is also the CALR antibody and vaccine and T-cell redirecting approaches which are all looking at both ET and myelofibrosis. But up to a third of ET patients can have CALR mutations, so the hope is that this could be something disease modifying for ET.
Katherine Banwell:
Dr. Handa, what are the latest research updates for polycythemia vera?
Dr. Shivani Handa:
So, in polycythemia vera, the most exciting update recently has been this emergence of a new class of drugs called hepcidin mimetics. And these drugs, they regulate the iron pathway, like how our body uses iron. So, in PV, it’s trying to increase the production of this or have a molecule that mimics this protein called hepcidin that our body normally produces that decreases iron absorption so we could limit the iron that’s taken up by the red blood cells, so eventually the red blood cell production will go down and more iron would be available for other body functions.
And that’s actually been shown to essentially eliminate the need for phlebotomies in PV patients and also improve quality of life and improve symptoms like fatigue and brain fog, which PV patients really suffer with.
Katherine Banwell:
Dr. Handa, are there recent advances in myelofibrosis treatment that patients should be aware of?
Dr. Shivani Handa:
Yes. So, in myelofibrosis I think we’re really trying to move beyond just JAK inhibitors and move into a combination era. So, there have been several trials recently looking at combination of different class of agents with JAK inhibitors. So, there was an update at the recent ASH meeting from the combination study of pelabresib with ruxolitinib (Jakafi). And that showed really durable spleen responses and very impressive deep spleen responses, much higher compared to just ruxolitinib by itself.
There’s also the combination of selinexor (Xpovio) and ruxolitinib, that’s being studied in a Phase III trial upfront. There is combination of a PIM1 kinase inhibitor, nuvisertib (TP-3654) that’s being looked at combination with another JAK inhibitor called momelotinib (Ojjaara). And it has shown really impressive spleen symptom responses but also anemia responses, which is something that myelofibrosis patients really suffer from and there are limited treatment options for. And in terms of anemia improvement, there’s also two other agents that are being looked at. There’s elritercept that’s being studied.
And there’s another hepcidin agent called an anti-hemojuvelin antibody that’s being developed by Disc Medicine. These are all going to be options for anemia management, so I think that’s really key.
Katherine Banwell:
Dr. Handa, how can patients stay up to date as MPN research evolves?
Dr. Shivani Handa:
Yeah. Great question. You know, there’s so much happening in the field right now that I think MPN patients should be seen at a specialized MPN center. So, I know most of cancer care happens in the community, and if somebody is not on active treatment, it may seem less important to be seen by a specialist. But by how nuanced this disease space is becoming, I think every patient should be referred to a specialized MPN center to have discussion and a more personalized treatment approach.
They can also stay informed by connecting with trusted organizations like the MPN Research Foundation is a great resource. The Patient Empowerment Network that you guys are leading, I think this is a great initiative for patients to get trusted information from experts in the field. There’s also a program that Incyte leads called Voices of MPN that’s a nonbranded, patient centric education that hosts online seminars and meet-ups. So, I think these are all great sources.