Molecular causes and new treatments for myelofibrosis
Official title: Project 1: Molecular Pathogenesis and Therapy of Myelofibrosis
Funded at Icahn School of Medicine at Mount Sinai · NIH-11094800 · Data updated 2026-11-12
This project is developing new treatments that target gene changes, inflammation, and epigenetic pathways to help people with myelofibrosis and related blood cancers.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Icahn School of Medicine at Mount Sinai NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | July 1, 2006 |
| Project end | May 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $453,221 |
| NIH project number | 5P01CA108671-17 |
| Project ID | NIH-11094800 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are people with primary myelofibrosis or those with polycythemia vera or essential thrombocytosis who are progressing toward myelofibrosis, especially if they have JAK2, MPL, or CALR mutations or worsening symptoms.
✕Less likely to help
Patients with unrelated blood conditions or those with stable, well-controlled disorders not driven by the pathways under study are unlikely to benefit directly from this project.
What this research studies
Researchers will study the common gene changes seen in myelofibrosis (like JAK2, MPL, and CALR) and how abnormal inflammation and epigenetic changes drive disease. They will use patient samples alongside lab and animal models to link those molecular findings to what happens in people. The team plans to test therapeutic strategies that go beyond current JAK2 inhibitors to try to reduce fibrosis, symptoms, and progression to acute leukemia. Successful lab findings could be moved into clinical trials to offer new treatment options.
Why it matters
Potential benefit: If successful, this work could lead to treatments that reduce spleen size and symptoms, slow or reverse bone marrow fibrosis, and lower the chance of progression to acute leukemia.
How similar studies have gone: Current JAK2 inhibitors help symptoms but seldom change disease course, and targeting epigenetic or inflammatory pathways is a newer approach with encouraging lab data but limited proven success in patients so far.
Where this research is happening
-
Icahn School of Medicine at Mount Sinai
New York, United States Active
Researchers
- Principal investigator: Levine, Ross L — Icahn School of Medicine at Mount Sinai
- Study coordinator: Levine, Ross L
Related research
Other NIH-funded projects on the same topics.
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Strategies to deplete myelofibrosis stem cells using HDM2/p53 pathway
A patient-focused program tests a therapy that may selectively reduce disease-causing blood stem cells in people with myelofibrosis.
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Megakaryocyte changes in myelofibrosis and its progression to leukemia
A scientist-guided project will study how abnormal megakaryocytes and signals like TGF-β and IL-13 may drive progression from myelofibrosis toward acute leukemia, with potential implications for future treatments.
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Molecular clues and new therapies for myelofibrosis and related blood cancers
This grant supports research to understand what drives myelofibrosis and related disorders and to develop new treatment ideas for patients.
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Myelofibrosis and the bone marrow environment: how inflammation changes blood production
A doctor-researcher is studying how inflammation in the bone marrow affects the blood-forming system in people with myeloproliferative neoplasms (MPNs) to find ways to prevent scarring and leukemia.
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Early detection of treatment response in myelofibrosis using a new imaging technology
A new, quick imaging method could tell patients with myelofibrosis early whether a treatment is working.
-
MYC-driven vulnerability in myelofibrosis could guide new therapies
A new project is exploring how the MYC protein contributes to myelofibrosis and whether it can be targeted to help people with this blood cancer.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.