Targeted therapy for splicing-factor mutant myeloid cancers
Official title: Project 3: Targeted therapy for splicing factor mutant myeloid malignancies
Funded at Washington University · NIH-11423682 · Data updated 2026-11-12
Aims to test a dual drug strategy targeting ATR and PARP pathways in adults with splicing-factor mutant acute myeloid leukemia or related myeloid diseases.
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 | 2026 |
| Award (this year) | $436,552 |
| NIH project number | 5P50CA171963-13 |
| Project ID | NIH-11423682 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with myeloid malignancies carrying spliceosome mutations (e.g., AF9/AML contexts) who can participate in preclinical-in-vitro/in-vivo studies or future clinical trials.
✕Less likely to help
Patients without spliceosome mutations or those not eligible for the proposed preclinical models may not benefit from this approach.
What this research studies
This grant focuses on how mutations in splicing factors make myeloid cancers like AML more vulnerable to combined ATR and PARP inhibitors. Researchers will use preclinical models to optimize the ATR plus PARP1 inhibitor combination and evaluate tolerability and effectiveness. If successful, this work could guide future trials offering a targeted treatment option for patients whose disease carries these specific genetic changes.
Why it matters
Potential benefit: If successful, patients with splicing-factor mutant myeloid malignancies could receive a new combination therapy that better controls the disease.
How similar studies have gone: Preclinical models show synergy between ATR and PARP inhibitors in related contexts, with ongoing clinical interest in dual inhibition.
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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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.