Understanding and targeting a weakness in childhood rhabdomyosarcoma
Official title: EXPLORE AND TARGET THE EPIGENETIC VULNERABILITY OF PAX3-FOXO1-DRIVEN RHABDOMYOSARCOMA
Funded at St. Jude Children's Research Hospital · NIH-11122258 · Data updated 2026-11-12
This project aims to find new ways to treat a serious childhood cancer called rhabdomyosarcoma by understanding its unique weaknesses.
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 | 2025 |
| Award (this year) | $691,425 |
| NIH project number | 5R01CA266600-04 |
| Project ID | NIH-11122258 on NIH RePORTER |
Who could benefit from this research
✓Could help
This research is focused on understanding the biology of rhabdomyosarcoma, particularly the aggressive alveolar subtype, which primarily affects children and adolescent young adults.
✕Less likely to help
Patients with other types of cancer or those not diagnosed with rhabdomyosarcoma would not directly benefit from this specific research.
What this research studies
This project focuses on rhabdomyosarcoma, a challenging soft tissue cancer that affects children and young adults. We know that aggressive forms of this cancer, especially alveolar rhabdomyosarcoma, are caused by specific genetic changes. While current treatments have helped some patients, those with advanced disease still face poor outcomes, and existing therapies can have severe long-term side effects for children. Our goal is to discover new targets within these cancer cells that can be blocked, leading to more effective and gentler treatments. By understanding how these cancer cells become vulnerable, we hope to develop better options for young patients.
Why it matters
Potential benefit: If successful, this work could lead to the development of new, more effective, and less toxic therapies for children and young adults with aggressive rhabdomyosarcoma.
How similar studies have gone: While current treatments for rhabdomyosarcoma have improved outcomes for some, new therapeutic targets are urgently needed for aggressive forms of the disease, making this approach novel and critical.
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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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.
-
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.