Targeting PAK4 to treat high-risk rhabdomyosarcoma in children and teens
Official title: Dissecting and targeting oncogenic functions of PAK4 in high-risk rhabdomyosarcoma
Funded at Emory University · NIH-11233151 · Data updated 2026-11-12
This project looks at whether blocking a protein called PAK4 can help existing drugs work better for children and adolescents with aggressive rhabdomyosarcoma.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Emory University NIH-funded |
| Lab location | 1 site (Atlanta, United States) |
| Project start | December 6, 2022 |
| Project end | November 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $356,480 |
| NIH project number | 5R01CA277686-04 |
| Project ID | NIH-11233151 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and adolescents with metastatic, relapsed, or otherwise high-risk rhabdomyosarcoma, especially those whose tumors show RAS/NF1 or RTK/PI3K pathway alterations, would be the most relevant candidates.
✕Less likely to help
Patients with low-risk, localized rhabdomyosarcoma who are well controlled by standard treatments, or tumors lacking PAK4-driven signaling, may not benefit from these approaches.
What this research studies
Researchers will study how PAK4 helps rhabdomyosarcoma tumors grow and resist treatment by examining tumor samples and laboratory models. They will use computer analyses, genetic and biochemical experiments, and high-throughput drug combination screens to find drugs that work with PAK4 inhibition. The team will also explore how PAK4 affects RNA splicing and immune signals that let tumors survive. Promising combinations could be advanced toward patient-focused testing and future clinical trials.
Why it matters
Potential benefit: If successful, this work could lead to new targeted drug combinations that improve survival and reduce relapse in high-risk pediatric rhabdomyosarcoma.
How similar studies have gone: Drugs targeting PAK family proteins have shown promise in lab models of several cancers but remain largely preclinical for rhabdomyosarcoma, so the approach is promising but not yet proven in patients.
Where this research is happening
-
Emory University
Atlanta, United States Active
Researchers
- Principal investigator: Yustein, Jason — Emory University
- Study coordinator: Yustein, Jason
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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.