NIH-funded research R01 grant

Targeting a new cancer gene in rhabdomyosarcoma

Official title: Targeting AVIL, a novel oncogene in rhabdomyosarcoma

Funded at University of Virginia · NIH-11001485 · Data updated 2026-11-12

This study is looking at a new way to treat rhabdomyosarcoma, a common soft-tissue cancer in kids, by targeting a gene called AVIL that helps the cancer grow, with the hope of finding better treatments for children facing this tough diagnosis.

More Rhabdomyosarcoma research and trials

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of Virginia
NIH-funded
Lab location1 site (Charlottesville, United States)
Project startJanuary 1, 2023
Project endDecember 31, 2027
Funding year2025
Award (this year)$503,649
NIH project number5R01CA269594-03
Project IDNIH-11001485 on NIH RePORTER

Who could benefit from this research

Could help

Ideal candidates for this research are children and young adults under 20 years old diagnosed with rhabdomyosarcoma.

Less likely to help

Patients with rhabdomyosarcoma who do not express the AVIL oncogene may not benefit from this targeted approach.

What this research studies

This research focuses on rhabdomyosarcoma (RMS), the most common soft-tissue cancer in children, which currently lacks effective targeted therapies. The team has identified a novel oncogene called AVIL that is overexpressed in many RMS cases and plays a critical role in the cancer's growth and spread. By investigating the effects of targeting AVIL, the researchers aim to develop new treatment strategies that could significantly improve outcomes for patients with advanced RMS. The study will involve laboratory experiments to assess the effectiveness of small molecules that inhibit AVIL in RMS cells.

Why it matters

Potential benefit: If successful, this research could lead to the development of targeted therapies that significantly improve survival rates for children with advanced rhabdomyosarcoma.

How similar studies have gone: Previous research has shown promising results in targeting oncogenes in other cancers, suggesting that this approach could be effective, although targeting AVIL specifically is a novel endeavor.

Where this research is happening

  • University of Virginia
    Charlottesville, United States
    Active

Researchers

  • Principal investigator: Li, Hui — University of Virginia
  • Study coordinator: Li, Hui

Related research

Other NIH-funded projects on the same topics.

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.
Last reviewed 2026-11-14 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.