Megakaryocyte changes in myelofibrosis and its progression to leukemia
Official title: Project 2: Defining the Role of Megakaryocyte Abnormalities in the Progression of Primary Myelofibrosis
Funded at Icahn School of Medicine at Mount Sinai · NIH-11399019 · Data updated 2026-11-12
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.
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 | 2026 |
| Award (this year) | $472,107 |
| NIH project number | 5P01CA108671-18 |
| Project ID | NIH-11399019 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with myelofibrosis or related myeloid diseases, and those whose disease involves abnormal megakaryocytes or progression toward leukemic transformation.
✕Less likely to help
Early-stage research without immediate clinical testing or enrollment opportunities may not directly benefit individual patients.
What this research studies
This NIH grant supports work at Icahn School of Medicine at Mount Sinai to understand how abnormal megakaryocytes contribute to myelofibrosis progression and the development of acute leukemia. Researchers will examine changes in the megakaryocyte lineage and how secreted factors such as TGF-β and IL-13 influence fibrosis and stem cell function. They will also explore the role of p53 activity in inhibiting normal hematopoiesis and in disease progression, aiming to identify targets for therapy. The project builds on prior work to test new strategies that could slow progression or improve symptoms for patients with myelofibrosis and related leukemias.
Why it matters
Potential benefit: If successful, findings could lead to new treatments that slow fibrosis, reduce symptom burden, and prevent progression to acute leukemia.
How similar studies have gone: Previous studies show that targeting megakaryocyte biology and cytokine signaling can affect disease features in myeloproliferative disorders, though progression to leukemia remains challenging and may require combination approaches.
Where this research is happening
-
Icahn School of Medicine at Mount Sinai
New York, United States Active
Researchers
- Principal investigator: Crispino, John D — Icahn School of Medicine at Mount Sinai
- Study coordinator: Crispino, John D
Related research
Other NIH-funded projects on the same topics.
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How abnormal platelet‑making cells drive worsening primary myelofibrosis
Looks at whether changes in megakaryocytes (the cells that make platelets) and the signals they release make primary myelofibrosis worse for people with the disease.
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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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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.
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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.