Targeted drug strategies for RAS-driven childhood rhabdomyosarcoma
Improving Therapeutic Approaches for RAS-driven Rhabdomyosarcoma
This project will try combinations of drugs that block RAF-MEK signaling to treat children with RAS-mutant embryonal rhabdomyosarcoma.
Quick facts
| Grant type | R37 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Oregon Health & Science University NIH-funded |
| Lab location | 1 site (Portland, United States) |
| Project ID | NIH-11303331 on NIH RePORTER |
What this research studies
From a patient's perspective, researchers are developing less-damaging, targeted treatments for high-risk childhood rhabdomyosarcoma that has RAS mutations. They use patient-derived tumor models (PDX/CDX) and laboratory tests to see how new RAF inhibitors combined with MEK inhibitors shrink tumors. The team is also studying how tumors become resistant to these drug combinations so they can design better, longer-lasting treatments. Results are intended to guide safer, more effective clinical therapies for children in the future.
Who could benefit from this research
Good fit: Children (typically infants to preteens) with embryonal rhabdomyosarcoma that carries RAS mutations, especially advanced or high-risk cases, would be the primary candidates for the therapies this project targets.
Not a fit: Patients without RAS mutations, with different sarcoma subtypes, or adults with unrelated cancers are unlikely to benefit directly from these specific approaches.
Why it matters
Potential benefit: If successful, this work could lead to more effective, less-genotoxic treatments that improve survival and reduce lifelong side effects for children with RAS-mutant ERMS.
How similar studies have performed: Preclinical work using RAF plus MEK inhibitors has produced dramatic tumor regressions in models, but these combinations have not yet been curative and clinical evidence remains limited.
Where this research is happening
Portland, United States
- Oregon Health & Science University — Portland, United States (Active)
Researchers
- Principal investigator: Vaseva, Angelina V — Oregon Health & Science University
- Study coordinator: Vaseva, Angelina V
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 below.