Investigating ways to reduce a cancer gene's impact in childhood muscle cancer.
Official title: Clinical & Mechanistic underpinnings to reducing PAX:FOXO1 for alveolar rhabdomyosarcoma
Funded at Children's Cancer Therapy Develop/inst · NIH-11136866 · Data updated 2026-11-12
This study is looking at a tough childhood muscle cancer called alveolar rhabdomyosarcoma and how a specific gene makes it harder to treat with chemotherapy; the researchers want to see if a drug called entinostat can help make chemotherapy work better by blocking that gene, which could lead to better outcomes for kids with this cancer.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Cancer Therapy Develop/inst NIH-funded |
| Lab location | 1 site (Beaverton, United States) |
| Project start | May 14, 2021 |
| Project end | April 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $303,780 |
| NIH project number | 5R01CA258720-05 |
| Project ID | NIH-11136866 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with alveolar rhabdomyosarcoma, particularly those whose cancer has shown resistance to standard chemotherapy.
✕Less likely to help
Patients with other types of cancer or those whose alveolar rhabdomyosarcoma is not driven by the PAX3:FOXO1 oncogene may not benefit from this research.
What this research studies
This research focuses on alveolar rhabdomyosarcoma (ARMS), a challenging childhood muscle cancer that often becomes resistant to chemotherapy due to the PAX3:FOXO1 oncogene. The team aims to understand how this gene contributes to treatment resistance and explore methods to counteract its effects, particularly through the use of a drug called entinostat. By silencing PAX3:FOXO1, they hope to enhance the effectiveness of chemotherapy and reduce the likelihood of cancer recurrence. The research involves detailed molecular studies to identify key regulatory pathways that could be targeted for improved treatment outcomes.
Why it matters
Potential benefit: If successful, this research could significantly improve survival rates for children with alveolar rhabdomyosarcoma by making their tumors more responsive to chemotherapy.
How similar studies have gone: Previous research has shown promise in targeting similar oncogenes and enhancing chemotherapy sensitivity, suggesting that this approach may be effective.
Where this research is happening
-
Children's Cancer Therapy Develop/inst
Beaverton, United States Active
Researchers
- Principal investigator: Keller, Charles — Children's Cancer Therapy Develop/inst
- Study coordinator: Keller, Charles
Related research
Other NIH-funded projects on the same topics.
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Targeting the PAX3-FOXO1 protein in a deadly childhood cancer
This study is looking at a specific protein that helps a tough childhood cancer called alveolar rhabdomyosarcoma, with the goal of finding new ways to treat kids who have this illness.
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Targeting how RMS cells fail to mature to slow or stop pediatric muscle cancer spread
A project to understand why a pediatric muscle cancer called rhabdomyosarcoma spreads, and to test ideas that could slow or stop it by helping cancer cells mature properly.
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A research project aims to create mouse models that mimic high-risk pediatric rhabdomyosarcoma and test therapies that target the NOTCH1 pathway to slow or stop tumor growth.
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Understanding infantile rhabdomyosarcoma biology and new treatment targets
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.