Strategies to deplete myelofibrosis stem cells using HDM2/p53 pathway
Official title: Project 3: Development of Strategies to Deplete Myelofibrosis Stem Cells
Funded at Icahn School of Medicine at Mount Sinai · NIH-11399028 · Data updated 2026-11-12
A patient-focused program tests a therapy that may selectively reduce disease-causing blood stem cells 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 start | July 1, 2006 |
| Project end | May 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $594,320 |
| NIH project number | 5P01CA108671-18 |
| Project ID | NIH-11399028 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with myelofibrosis, especially those at risk for progression to blast phase, who are eligible for experimental therapies.
✕Less likely to help
Patients whose disease biology does not respond to HDM2-p53 modulation or who cannot tolerate the therapy may not benefit.
What this research studies
This grant focuses on a targeted approach to reduce abnormal blood stem cells in myelofibrosis by boosting the tumor suppressor p53 through HDM2 blockade. It builds on prior work showing that HDM2 antagonists can deplete diseased stem cells while sparing healthy ones. The research combines molecular studies with early therapeutic exploration to see if this strategy can slow progression and improve marrow function. If successful, it could translate into more effective treatments for patients with myelofibrosis who are at risk of progression to blast phase. The project is led by Ps Ronald Hoffman at Icahn School of Medicine at Mount Sinai and runs over many years, seeking to move from concept to potential clinical use.
Why it matters
Potential benefit: If this approach works, patients with myelofibrosis could experience slower disease progression and improved blood counts.
How similar studies have gone: Early studies suggest HDM2 antagonists can reduce disease-driving mutations and marrow fibrosis in myelofibrosis, supporting further development.
Where this research is happening
-
Icahn School of Medicine at Mount Sinai
New York, United States Active
Researchers
- Principal investigator: Hoffman, Ronald — Icahn School of Medicine at Mount Sinai
- Study coordinator: Hoffman, Ronald
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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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.
-
MRI markers to monitor bone marrow in myelofibrosis
Advanced MRI scans will be used to see bone marrow changes in people with myelofibrosis so doctors can tell sooner whether new targeted drugs are working.
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.