Targeting mutant-MYOD1 driven rhabdomyosarcoma in kids
Official title: Targeting mutant-MYOD1 driven rhabdomyosarcoma
Funded at Research Inst Nationwide Children's Hosp · NIH-11365879 · Data updated 2026-11-12
Aiming to find new treatments for a deadly childhood cancer driven by MYOD1 mutations by building a fast, collaborative pipeline from lab discoveries to patient care.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Research Inst Nationwide Children's Hosp NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project start | July 3, 2025 |
| Project end | June 30, 2029 |
| Funding year | 2026 |
| Award (this year) | $372,142 |
| NIH project number | 5U01CA298763-02 |
| Project ID | NIH-11365879 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and adolescents diagnosed with MYOD1-mutant rhabdomyosarcoma who could participate in preclinical testing and, later, clinical trials.
✕Less likely to help
Early-stage preclinical work or therapies not yet approved for RMS may not benefit current patients.
What this research studies
This project focuses on rhabdomyosarcoma, a pediatric cancer that is often aggressive and hard to treat. It will study how MYOD1 mutations create vulnerabilities that can be targeted with drugs, especially through PI3K-related pathways and chromatin accessibility. The team will develop preclinical models and integrate clinical data from the NIH Clinical Center to test therapies and understand which patients might benefit most. A bench-to-bedside approach will be used to move promising treatments into clinical trials with correlative studies to map genetic contexts to outcomes.
Why it matters
Potential benefit: If successful, patients with this lethal form of RMS could have more effective, tailored therapies and faster access to new treatment options.
How similar studies have gone: Prior studies show PI3K-related vulnerabilities in some RMS models, supporting a potentially fruitful translational path.
Where this research is happening
-
Research Inst Nationwide Children's Hosp
Columbus, United States Active
Researchers
- Principal investigator: Stanton, Benjamin — Research Inst Nationwide Children's Hosp
- Study coordinator: Stanton, Benjamin
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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.