Explore and target the epigenetic weak spots in PAX3-FOXO1 driven rhabdomyosarcoma
Official title: EXPLORE AND TARGET THE EPIGENETIC VULNERABILITY OF PAX3-FOXO1-DRIVEN RHABDOMYOSARCOMA
Funded at St. Jude Children's Research Hospital · NIH-11362175 · Data updated 2026-11-12
Researchers are studying how a specific cancer-causing fusion protein changes epigenetics in rhabdomyosarcoma to find new therapies for affected children and teens.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project start | July 1, 2022 |
| Project end | June 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $691,425 |
| NIH project number | 5R01CA266600-05 |
| Project ID | NIH-11362175 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children and adolescents with fusion-positive alveolar rhabdomyosarcoma or related RMS subtypes.
✕Less likely to help
Patients without this specific cancer subtype may not directly benefit from these targeted approaches.
What this research studies
This project looks at how the cancer-driving PAX3-FOXO1 fusion protein alters epigenetic regulators called KDMs in rhabdomyosarcoma, with a focus on identifying accessible targets to block the oncoprotein’s effects. By examining chromatin openness using ATAC sequencing and other epigenetic markers, the team hopes to uncover vulnerabilities unique to the tumor cells. The goal is to translate these insights into new treatments that spare healthy tissues and reduce long-term toxicity for pediatric and adolescent patients. The study is led by Jun Yang at St. Jude Children’s Research Hospital and spans collaboration across pediatric oncology researchers. Outcomes could pave the way for targeted therapies in a challenging cancer subtype."
Why it matters
Potential benefit: If successful, this research could lead to targeted therapies that improve survival and reduce treatment-related side effects for young people with alveolar rhabdomyosarcoma.
How similar studies have gone: Previous work in pediatric cancers shows promise when targeting epigenetic regulators, but this program addresses a high-need, specific fusion-driven RMS context.
Where this research is happening
-
St. Jude Children's Research Hospital
Memphis, United States Active
Researchers
- Principal investigator: Yang, Jun — St. Jude Children's Research Hospital
- Study coordinator: Yang, Jun
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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Genetically engineered mouse model to study and treat high-risk pediatric rhabdomyosarcoma
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.
-
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.