Creating a bone marrow model to study a genetic platelet disorder and its cancer risks
Official title: Development of Bone Marrow Organoid System to Study RUNX1-Familial Platelet Disorder with Associated Myeloid Malignancy (RUNX1-FPD/MM)
Funded at Boston Children's Hospital · NIH-11011813 · Data updated 2026-11-12
This study is looking at a rare genetic condition that affects blood platelets and can increase the risk of blood cancers, and it's for people with this condition; the researchers want to create a mini version of bone marrow to better understand the disease and find new ways to help reduce the risk of leukemia.
Quick facts
| Grant type | R03 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | June 15, 2025 |
| Project end | June 14, 2026 |
| Funding year | 2025 |
| Award (this year) | $178,000 |
| NIH project number | 1R03TR004848-01A1 |
| Project ID | NIH-11011813 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with RUNX1 Familial Platelet Disorder, particularly those with a family history of the condition.
✕Less likely to help
Patients without RUNX1-FPD/MM or those with other unrelated hematological disorders may not benefit from this research.
What this research studies
This research focuses on a rare genetic condition known as RUNX1 Familial Platelet Disorder with Propensity to Develop Myeloid Malignancy (RUNX1-FPD/MM), which leads to low platelet counts and an increased risk of blood cancers. The researchers aim to develop a bone marrow organoid system that mimics human disease, allowing for better understanding of the disorder and testing potential treatments. By using advanced techniques like CRISPR gene editing, they hope to create models that accurately reflect the genetic mutations involved. This could ultimately lead to identifying interventions that may reduce the risk of leukemia in affected individuals.
Why it matters
Potential benefit: If successful, this research could lead to new strategies for preventing leukemia in patients with RUNX1-FPD/MM.
How similar studies have gone: While there have been attempts to study similar genetic disorders, the specific approach of using bone marrow organoids for RUNX1-FPD/MM is relatively novel and has not been extensively tested.
Where this research is happening
-
Boston Children's Hospital
Boston, United States Active
Researchers
- Principal investigator: Cantor, Alan B. — Boston Children's Hospital
- Study coordinator: Cantor, Alan B.
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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.