Blocking the AVIL gene in rhabdomyosarcoma
Official title: Targeting AVIL, a novel oncogene in rhabdomyosarcoma
Funded at University of Virginia · NIH-11258871 · Data updated 2026-11-12
Using new drugs that block the AVIL cancer gene to try to stop rhabdomyosarcoma growth in children, adolescents, and young adults.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Virginia NIH-funded |
| Lab location | 1 site (Charlottesville, United States) |
| Project start | January 1, 2023 |
| Project end | December 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $503,649 |
| NIH project number | 5R01CA269594-04 |
| Project ID | NIH-11258871 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with rhabdomyosarcoma—particularly children, adolescents, and young adults—whose tumors show high AVIL expression or the MARS-AVIL fusion are the most likely candidates for AVIL-targeted therapy.
✕Less likely to help
Patients whose tumors do not overexpress AVIL or lack the MARS-AVIL fusion may not benefit from AVIL-targeted treatments.
What this research studies
Researchers discovered that the AVIL gene is often abnormally active in rhabdomyosarcoma and can drive tumor growth. They will test small molecules that block AVIL in lab-grown tumor cells and in animal models to see if cancer cells die or tumors shrink. The team will also measure AVIL levels and related changes in patient tumor samples to understand who might benefit. The goal is to use these results to support future clinical testing of AVIL-targeted treatments.
Why it matters
Potential benefit: If successful, this work could lead to a targeted drug that shrinks tumors or improves outcomes for patients with AVIL-driven rhabdomyosarcoma.
How similar studies have gone: Preclinical data reported by the team show that silencing AVIL or using AVIL-inhibiting molecules killed RMS cells and slowed tumor xenografts, but AVIL-targeting therapies have not yet been proven in human patients.
Where this research is happening
-
University of Virginia
Charlottesville, United States Active
Researchers
- Principal investigator: Li, Hui — University of Virginia
- Study coordinator: Li, Hui
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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.