Emerging Therapeutic Strategies Across Myeloproliferative Neoplasms

At the 2026 LL&M Winter Symposium in Amelia Island, Florida, Lindsay Rein, MD, Duke Cancer Institute, Durham, North Carolina, discusses emerging therapeutic strategies across myeloproliferative neoplasms, including polycythemia vera, essential thrombocythemia, and myelofibrosis. 

Dr Rein highlights novel agents such as hepcidin mimetics, LSD1 inhibitors, and evolving combination approaches. Advances in mutation-specific and immune-based therapies, including those targeting calreticulin, are also shaping future treatment strategies.

Transcript:

We had an opportunity to consider some of the novel things that are happening within the world of MPNs. As a clinician and a researcher, I'm really excited to be a part of this because there really is so much excitement, so much research that's being done. 

We talked about polycythemia vera (PV) and some of the novel agents that are being studied within the context of this group of diseases. We discussed hepcidin mimetics and then we also discussed some of the novel LSD1 inhibitor data looking at BOMEDEMSTAT in patients with PV. 

We went on to think about essential thrombocytosis (ET) as part of a spectrum of disease, and we discussed the history of some of ET-directed therapeutics and the progress that's being made to move the field forward, considering the biology of the disease as we think about what particular agents are coming up the pipeline for new therapeutics.

We briefly thought about myelofibrosis and the current landscape of clinical trials within this space. This was enough to really fill probably an entire afternoon of discussion, but we briefly thought about the different combination strategies that are being studied now for patients with myelofibrosis. 

Lastly, we took a few minutes to think about calreticulin mutation-specific therapies and some of the novel immune-based strategies that we're implementing for that particular driver mutation and how that has really impacted that subset of patients.

Source: 

Rein L. Updates in myeloproliferative neoplasms: Translating emerging evidence into practice. Presented at Lymphoma, Leukemia & Myeloma Winter Symposium; January 30 - February 1, 2026. Amelia Island, Florida.