Targeting a specific protein to improve treatment for high-risk rhabdomyosarcoma in children
Official title: Dissecting and targeting oncogenic functions of PAK4 in high-risk rhabdomyosarcoma
Funded at Emory University · NIH-10979166 · Data updated 2026-11-12
This study is looking at how a protein called PAK4 affects a type of cancer called rhabdomyosarcoma, which often affects kids and teens, to find better treatments that can help those whose cancer has come back or spread.
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 | 2025 |
| Award (this year) | $356,758 |
| NIH project number | 5R01CA277686-03 |
| Project ID | NIH-10979166 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children and adolescents diagnosed with high-risk rhabdomyosarcoma, particularly those with metastatic or relapsed disease.
✕Less likely to help
Patients with low-risk rhabdomyosarcoma or those whose cancer has not progressed may not receive benefit from this research.
What this research studies
This research focuses on understanding how the PAK4 protein contributes to the progression of high-risk rhabdomyosarcoma, a common soft tissue cancer in children and adolescents. By investigating the molecular mechanisms involved, the researchers aim to develop targeted therapies that can more effectively treat patients with metastatic or relapsed forms of this cancer. The approach includes using advanced techniques such as genomic analysis and drug screening to identify potential new treatment options. Patients may benefit from more effective therapies that specifically target the underlying causes of their cancer.
Why it matters
Potential benefit: If successful, this research could lead to improved treatment options and better survival rates for children with high-risk rhabdomyosarcoma.
How similar studies have gone: Other research has shown promise in targeting similar oncogenic pathways in various cancers, suggesting that this approach could be effective.
Where this research is happening
-
Emory University
Atlanta, United States Active
Researchers
- Principal investigator: Yustein, Jason — Emory University
- Study coordinator: Yustein, Jason
Related research
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A new grant is studying why infantile rhabdomyosarcoma develops and what drugs or strategies could specifically target it in young children.
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Targeting CLK2 to Fight Rhabdomyosarcoma
Aims to see if blocking CLK2 can weaken rhabdomyosarcoma cancer cells, potentially offering a new treatment for kids and young adults.
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Epitranscriptomic Crosstalk in Alveolar Rhabdomyosarcoma
A pediatric cancer study that looks at how chemical marks on messenger RNA may influence the behavior of alveolar rhabdomyosarcoma and point toward newer treatment strategies.
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.