Why MYOD1‑mutant rhabdomyosarcoma acts more aggressively
Official title: Mechanisms of aggressive Rhabdomyosarcoma.
Funded at Massachusetts General Hospital · NIH-11286812 · Data updated 2026-11-12
This project looks at whether a specific MYOD1 gene change (L122R) makes some children's rhabdomyosarcoma grow faster and resist treatment.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | March 1, 2023 |
| Project end | February 29, 2028 |
| Funding year | 2026 |
| Award (this year) | $539,997 |
| NIH project number | 5R01CA276116-04 |
| Project ID | NIH-11286812 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children or young people with spindle/sclerosing rhabdomyosarcoma who have the MYOD1 L122R mutation, or families willing to provide tumor samples for research, would be the most relevant participants.
✕Less likely to help
Patients without the MYOD1 L122R mutation or those needing immediate clinical treatment are unlikely to get direct therapeutic benefit from this preclinical laboratory research.
What this research studies
Researchers will create lab-grown tumor cells and genetically engineered models that carry the MYOD1 L122R change to mimic the aggressive tumor subtype. They will map how this mutation alters gene control and chromatin accessibility using methods like ATAC‑seq and other molecular assays. The team will search for pathways that drive therapy resistance and cancer stem cells and test candidate drugs in those models. Results will guide new targets to develop treatments for this high‑risk pediatric sarcoma.
Why it matters
Potential benefit: If successful, this work could reveal drug targets and strategies to improve outcomes for children with MYOD1 L122R–mutant rhabdomyosarcoma.
How similar studies have gone: Related lab approaches (genetically engineered models and chromatin profiling) have found useful targets in other cancers, but direct mechanistic work on MYOD1 L122R in rhabdomyosarcoma is largely new.
Where this research is happening
-
Massachusetts General Hospital
Boston, United States Active
Researchers
- Principal investigator: Langenau, David Michael — Massachusetts General Hospital
- Study coordinator: Langenau, David Michael
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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.