Preventing leukemia in patients with RUNX1-FPD using tyrosine kinase inhibitors
Official title: Study of tyrosine kinase inhibitors as preventive therapy in RUNX1-FPD
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11027130 · Data updated 2026-11-12
This study is looking at whether a medication called imatinib can help prevent blood cancers in people with Familial Platelet Disorder by improving how their bodies fix DNA damage caused by a gene mutation.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project start | May 1, 2025 |
| Project end | April 30, 2030 |
| Funding year | 2025 |
| Award (this year) | $485,790 |
| NIH project number | 1U01CA296350-01 |
| Project ID | NIH-11027130 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Familial Platelet Disorder who carry mutations in the RUNX1 gene.
✕Less likely to help
Patients without RUNX1 mutations or those who do not have Familial Platelet Disorder may not benefit from this research.
What this research studies
This research investigates the use of tyrosine kinase inhibitors, specifically imatinib, as a preventive treatment for patients with Familial Platelet Disorder (FPD), a genetic condition that increases the risk of developing blood cancers. The study aims to understand how RUNX1 gene mutations affect the body's ability to repair DNA damage and promote the development of leukemia. By restoring the DNA damage repair response in pre-malignant cells, the researchers hope to reduce the risk of disease onset in these patients. The approach involves both laboratory experiments and potential clinical applications to evaluate the effectiveness of the treatment.
Why it matters
Potential benefit: If successful, this research could significantly lower the risk of leukemia in patients with RUNX1-FPD, potentially extending their healthy lifespan.
How similar studies have gone: Previous research has shown promise in using tyrosine kinase inhibitors for similar hematologic conditions, suggesting a potential for success in this novel application.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Castilla, Lucio H. — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Castilla, Lucio H.
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.