Improving Treatments for Childhood Rhabdomyosarcoma
Official title: Targeting mTORC1 translational control in FOXO1 fusion positive rhabdomyosarcoma
Funded at University of California, San Francisco · NIH-11126020 · Data updated 2026-11-12
This research looks for new ways to stop a type of childhood cancer called rhabdomyosarcoma from growing and becoming resistant to current medicines.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | July 1, 2024 |
| Project end | June 30, 2029 |
| Funding year | 2025 |
| Award (this year) | $482,283 |
| NIH project number | 5R01CA285680-02 |
| Project ID | NIH-11126020 on NIH RePORTER |
Who could benefit from this research
✓Could help
This research is relevant for children, specifically those aged 0-11 years, who have rhabdomyosarcoma with a FOXO1 gene fusion.
✕Less likely to help
Patients with other types of cancer or rhabdomyosarcoma without the FOXO1 gene fusion may not directly benefit from this specific research.
What this research studies
We are exploring how a specific type of childhood cancer, rhabdomyosarcoma with a FOXO1 gene fusion, relies on a process called mTORC1 for its growth. While new medicines that block mTORC1 show promise in initial tests, the cancer can sometimes return. Our goal is to understand why these cancers become resistant and find better ways to combine treatments to achieve lasting cures. We hope to provide information that helps doctors use these new medicines more effectively for children with this challenging cancer.
Why it matters
Potential benefit: If successful, this work could lead to more effective and lasting treatments for children with a difficult-to-treat form of rhabdomyosarcoma, potentially improving cure rates.
How similar studies have gone: New mTORC1 inhibitors are currently in early clinical testing and have shown strong results in laboratory models, but resistance remains a challenge that this work aims to address.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Sabnis, Amit J. — University of California, San Francisco
- Study coordinator: Sabnis, Amit J.
Related research
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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
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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.