How abnormal platelet‑making cells drive worsening primary myelofibrosis
Project 2: Defining the Role of Megakaryocyte Abnormalities in the Progression of 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.
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-11094801 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: 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.
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 performed: 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
New York, United States
- 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
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.