Investigating the biology and treatment targets of infantile rhabdomyosarcoma

Understanding Infantile Rhabdomyosarcoma Biology and Therapeutic Targets

NIH-funded research Research Inst Nationwide Children's Hosp · NIH-11065573

This study is looking into a type of cancer called infantile rhabdomyosarcoma that affects young kids, and it's trying to find out how certain genes, especially one called VGLL2-NCOA2, might help us discover better treatments beyond just chemotherapy and surgery.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionResearch Inst Nationwide Children's Hosp NIH-funded
Lab location1 site (Columbus, United States)
Project IDNIH-11065573 on NIH RePORTER

What this research studies

This research focuses on understanding infantile rhabdomyosarcoma (RMS), an aggressive cancer affecting young children. The study aims to explore the genetic factors that contribute to this cancer, particularly a specific gene fusion known as VGLL2-NCOA2, which plays a role in muscle development. By utilizing various models, including zebrafish and mice, along with patient samples, the research seeks to identify new therapeutic targets that could lead to more effective treatments. The ultimate goal is to improve treatment options beyond the current reliance on general chemotherapy and surgery.

Who could benefit from this research

Good fit: Ideal candidates for this research are children under 11 years old diagnosed with infantile rhabdomyosarcoma.

Not a fit: Patients with other types of cancers or those older than 11 years may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to more targeted and effective treatments for infantile rhabdomyosarcoma, reducing the need for harsh chemotherapy and improving outcomes for young patients.

How similar studies have performed: While there has been limited research specifically targeting infantile rhabdomyosarcoma, similar approaches in understanding cancer biology and targeting genetic factors have shown promise in other cancer types.

Where this research is happening

Columbus, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.