Targeting a specific cancer driver in a type of childhood cancer
Official title: Targeting mTORC1 translational control in FOXO1 fusion positive rhabdomyosarcoma
Funded at University of California, San Francisco · NIH-10979766 · Data updated 2026-11-12
This study is looking at a type of childhood cancer called rhabdomyosarcoma, especially the cases caused by a specific gene change, and it's exploring how a new kind of medicine can help fight this cancer and improve treatment for kids who have it.
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 | 2024 |
| Award (this year) | $482,175 |
| NIH project number | 1R01CA285680-01A1 |
| Project ID | NIH-10979766 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children under 11 years old diagnosed with PAX3-FOXO1 fusion positive rhabdomyosarcoma.
✕Less likely to help
Patients with other types of rhabdomyosarcoma or those without the PAX3-FOXO1 fusion may not benefit from this research.
What this research studies
This research focuses on a specific type of childhood cancer called rhabdomyosarcoma, particularly those cases driven by a genetic fusion known as PAX3-FOXO1. The researchers are investigating how this fusion affects cancer cell behavior and how it can be targeted using a new class of drugs that inhibit a pathway called mTORC1. By understanding the molecular mechanisms involved, they aim to develop more effective treatments that could lead to better outcomes for affected children. The study will involve both laboratory experiments and analysis of patient-derived models to identify the best strategies for overcoming treatment resistance.
Why it matters
Potential benefit: If successful, this research could lead to more effective therapies for children with PAX3-FOXO1 fusion positive rhabdomyosarcoma, potentially improving survival rates.
How similar studies have gone: This approach is based on promising preliminary results from similar studies using mTORC1 inhibitors, indicating potential for success in this area.
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
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 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.