How abnormal platelet‑making cells drive worsening primary myelofibrosis
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-11094801 · Data updated 2026-11-12
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.
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) | $468,639 |
| NIH project number | 5P01CA108671-17 |
| Project ID | NIH-11094801 on NIH RePORTER |
Who could benefit from this research
✓Could help
People diagnosed with primary myelofibrosis, including those in prefibrotic or early fibrotic stages and those with JAK2, MPL, or CALR mutations, would be the most relevant candidates for related clinical or sample‑donation opportunities.
✕Less likely to help
People without myelofibrosis or with unrelated blood disorders are unlikely to benefit directly, and those needing immediate therapy may not see immediate treatment effects from this lab‑focused work.
What this research studies
You would hear from researchers who study how certain bone marrow cells called megakaryocytes change as myelofibrosis moves from an early to a fibrotic stage. They are focusing on low GATA1 in these cells and on secreted factors like TGF‑β and IL‑13, plus changes in p53 and HIF‑1α that might harm normal blood stem cells. The team uses patient samples, cell and animal models, and molecular experiments to trace how these changes cause marrow scarring and increase risk of progression to acute leukemia. Their lab work aims to point to targets that could be turned into treatments to slow or stop disease progression.
Why it matters
Potential benefit: If successful, this work could identify new targets to reduce bone marrow fibrosis and lower the chance myelofibrosis turns into leukemia.
How similar studies have gone: Previous research supports a role for megakaryocyte‑derived TGF‑β in marrow fibrosis, but the specific contributions of IL‑13, p53 changes, and HIF‑1α to progression are less explored and more novel.
Where this research is happening
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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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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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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.
-
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.