Infant rhabdomyosarcoma — finding what drives it and new ways to target it
Official title: Understanding Infantile Rhabdomyosarcoma Biology and Therapeutic Targets
Funded at Research Inst Nationwide Children's Hosp · NIH-11211683 · Data updated 2026-11-12
Researchers will use animal models and patient tumor samples to find the molecular drivers and potential drug targets for infant rhabdomyosarcoma in young children.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Research Inst Nationwide Children's Hosp NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project start | September 23, 2022 |
| Project end | August 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $71,772 |
| NIH project number | 3R01CA272872-04S1 |
| Project ID | NIH-11211683 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be infants and very young children diagnosed with infantile rhabdomyosarcoma, especially those whose tumors carry VGLL2-NCOA2 or related gene fusions.
✕Less likely to help
Children with other RMS subtypes that do not share the same fusion-driven biology, or older patients with different tumor genetics, may not see direct benefit from this work.
What this research studies
This project combines zebrafish, mouse, cell culture models, and patient tumor samples to study infantile rhabdomyosarcoma driven by gene fusions like VGLL2-NCOA2. Researchers plan to create and use animal models and patient-derived materials because there are currently few models to study how these tumors grow and respond to drugs. The team will look at normal developmental programs and specific molecules such as ARF6 that the tumor may hijack to grow. Findings will be used to identify potential therapeutic targets that could be tested in future treatments.
Why it matters
Potential benefit: If successful, the work could point to targeted treatments that work better and cause fewer long-term side effects than current chemotherapy, surgery, and radiation.
How similar studies have gone: Researchers are applying methods that have found targets in other cancers, but targeted therapies for VGLL2-NCOA2-driven infantile RMS are largely untested and represent a novel area of study.
Where this research is happening
-
Research Inst Nationwide Children's Hosp
Columbus, United States Active
Researchers
- Principal investigator: Kendall, Genevieve Claire — Research Inst Nationwide Children's Hosp
- Study coordinator: Kendall, Genevieve Claire
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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.