Understanding infantile rhabdomyosarcoma biology and new treatment targets
Official title: Understanding Infantile Rhabdomyosarcoma Biology and Therapeutic Targets
Funded at Research Inst Nationwide Children's Hosp · NIH-11411145 · Data updated 2026-11-12
A study to learn why a rare cancer in very young children grows and to find ways to stop it with targeted therapies.
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 | 2026 |
| Award (this year) | $382,872 |
| NIH project number | 5R01CA272872-05 |
| Project ID | NIH-11411145 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children under 12 with infantile rhabdomyosarcoma or related RMS subtypes, whose tumors may be driven by VGLL2-NCOA2 fusions.
✕Less likely to help
Patients with RMS not driven by VGLL2-NCOA2 or whose cancer biology is unrelated to the studied pathways may experience no direct benefit.
What this research studies
This project aims to uncover the biology of infantile rhabdomyosarcoma and identify actionable targets by studying gene fusions like VGLL2-NCOA2 and the role of ARF6. It plans to use zebrafish, mouse, cell culture models, and patient samples to map cancer-driving programs and compare infantile RMS with other RMS subtypes. The ultimate goal is to translate these biological insights into therapies that specifically target the cancer’s genetics and developmental pathways. The research emphasizes creating and using diverse model systems to understand tumor behavior and test potential treatments.
Why it matters
Potential benefit: If successful, the work could lead to more effective, less toxic therapies for young children with infantile RMS.
How similar studies have gone: Past efforts have started to unveil fusion-driven RMS biology, and this project builds on that by integrating multiple models to identify conserved therapeutic targets.
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.