Targeted therapy for myeloid blood cancers with splicing factor mutations
Official title: Project 3: Targeted therapy for splicing factor mutant myeloid malignancies
Funded at Washington University · NIH-11195604 · Data updated 2026-11-12
This project explores whether combining two targeted drugs that block ATR and PARP can better treat adults with myeloid blood cancers that carry splicing factor mutations.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project start | September 3, 2013 |
| Project end | June 30, 2029 |
| Funding year | 2025 |
| Award (this year) | $356,195 |
| NIH project number | 5P50CA171963-12 |
| Project ID | NIH-11195604 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults (21+) with myelodysplastic syndromes or acute myeloid leukemia whose tumors harbor splicing factor mutations, especially those who have relapsed or are refractory to front-line therapy, are the ideal candidates.
✕Less likely to help
Patients without splicing factor mutations or with other unrelated types of leukemia are unlikely to benefit from this specific ATR+PARP targeted approach.
What this research studies
If you have MDS or AML with a splicing factor mutation, researchers are studying how those mutations create DNA:RNA hybrid structures called R loops that make cancer cells dependent on certain repair pathways. They plan to optimize a drug combination that blocks ATR and PARP, because lab studies show the two drugs work together to kill these cancer cells. The team will use multiple preclinical models and build on earlier patient experience with an ATR inhibitor to guide safer, more effective combinations. The goal is to move promising combinations toward clinical testing for adults whose disease has relapsed or not responded to initial therapy.
Why it matters
Potential benefit: If successful, this approach could produce more effective, targeted treatment options for adults with MDS/AML driven by splicing factor mutations and potentially improve remission duration and survival.
How similar studies have gone: A phase II trial showed activity of an ATR inhibitor alone in relapsed/refractory MDS and preclinical studies report strong synergy for ATR plus PARP inhibition, but combination clinical data are still limited.
Where this research is happening
-
Washington University
Saint Louis, United States Active
Researchers
- Principal investigator: Walter, Matthew J — Washington University
- Study coordinator: Walter, Matthew J
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Targeted therapy for splicing-factor mutant myeloid cancers
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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.