Molecular causes and new treatments for myelofibrosis
Project 1: Molecular Pathogenesis and Therapy of Myelofibrosis
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 ID | NIH-11094800 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: 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.
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 performed: 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
New York, United States
- 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
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 below.