Infantile rhabdomyosarcoma: causes and new treatment targets
Official title: Understanding Infantile Rhabdomyosarcoma Biology and Therapeutic Targets
Funded at Research Inst Nationwide Children's Hosp · NIH-11145032 · Data updated 2026-11-12
This project looks into the genes behind aggressive infant rhabdomyosarcoma to find possible drug targets for babies and 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) | $382,872 |
| NIH project number | 5R01CA272872-04 |
| Project ID | NIH-11145032 on NIH RePORTER |
Who could benefit from this research
✓Could help
Infants and very young children diagnosed with infantile rhabdomyosarcoma—particularly those whose tumors carry the VGLL2-NCOA2 fusion—or families willing to donate tumor tissue or clinical data are the most relevant candidates.
✕Less likely to help
Children without infantile RMS, those with different tumor subtypes, or patients unable or unwilling to provide samples may not directly benefit from this specific research effort.
What this research studies
As a parent or patient, you should know researchers are using tumor samples from children plus animal and cell models to learn what makes infantile RMS grow. They focus on a specific gene fusion called VGLL2-NCOA2 and related pathways like ARF6 that seem to drive the disease. Because there are few existing models, the team will build zebrafish, mouse, cell line, and patient-derived tumor models to compare biology across systems. The aim is to find shared genetic programs and drug targets that could lead to better, less toxic treatments for affected infants.
Why it matters
Potential benefit: If successful, this work could point to targeted therapies or drug candidates that are more effective and less harmful than current chemotherapy and radiation for infants with this cancer.
How similar studies have gone: Genomic sequencing has already identified the VGLL2-NCOA2 fusion, but there are few preclinical models or targeted treatments for infantile RMS, so this approach is relatively novel with limited prior clinical success.
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.