Myelofibrosis Clinical Consortium
Project 4: MPN Clinical Consortium
This project tests new treatments that aim to remove the abnormal blood-forming stem cells and repair the bone marrow environment in people with myelofibrosis.
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-11094808 on NIH RePORTER |
What this research studies
This effort runs clinical trials through a multi-center myelofibrosis consortium to try therapies that selectively deplete the disease-causing hematopoietic stem cells. Doctors will collect blood and bone marrow samples and use biomarkers to track how well the stem cell pool is reduced and how patients respond or develop resistance. The trials build on laboratory findings from other consortium projects and use Mount Sinai's clinical network to enroll patients and coordinate correlative lab studies. Findings will help identify which approaches best slow or reverse disease progression.
Who could benefit from this research
Good fit: Adults with Philadelphia chromosome–negative myelofibrosis, especially those with progressive disease or inadequate response to current treatments, would be the most likely candidates.
Not a fit: People without myelofibrosis, patients with other blood cancers, or those who are too frail or otherwise ineligible for clinical trials are unlikely to benefit directly from this project.
Why it matters
Potential benefit: If successful, these approaches could slow or stop disease progression, reduce spleen size and symptoms, and improve survival for people with myelofibrosis.
How similar studies have performed: Existing treatments like JAK inhibitors help symptoms but do not halt progression, while newer therapies that target disease-initiating stem cells have shown promise in early work but are still being tested clinically.
Where this research is happening
New York, United States
- Icahn School of Medicine at Mount Sinai — New York, United States (Active)
Researchers
- Principal investigator: Mascarenhas, John — Icahn School of Medicine at Mount Sinai
- Study coordinator: Mascarenhas, John
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.