Targeting the PAX3-FOXO1 protein in a childhood cancer

Defining and targeting the molecular vulnerabilities of the PAX3-FOXO1 protein in rhabdomyosarcoma

NIH-funded research Duke University · NIH-10221081

This study is looking at a specific protein that helps a tough childhood cancer called rhabdomyosarcoma grow, and the goal is to find new ways to treat this cancer better for kids who are affected by it.

Quick facts

Grant typeNIH-funded research
Study typeNIH-funded research
Funding institutionDuke University NIH-funded
Lab location1 site (Durham, United States)
Project IDNIH-10221081 on NIH RePORTER

What this research studies

This research focuses on understanding and targeting the PAX3-FOXO1 fusion protein, which plays a critical role in a severe type of childhood cancer called rhabdomyosarcoma. The team aims to identify the proteins and regulatory elements that support the tumor's growth, using advanced biological and technological methods. By comprehensively studying these molecular vulnerabilities, the research seeks to develop new therapeutic strategies that could improve treatment outcomes for affected children.

Who could benefit from this research

Good fit: Ideal candidates for this research are children diagnosed with PAX3-FOXO1-positive rhabdomyosarcoma.

Not a fit: Patients with other types of rhabdomyosarcoma or those without the PAX3-FOXO1 fusion protein may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to more effective treatments for children with rhabdomyosarcoma, potentially increasing survival rates.

How similar studies have performed: While the approach to directly target the PAX3-FOXO1 protein is innovative, similar research efforts in targeting oncogenic proteins have shown promise in other cancers.

Where this research is happening

Durham, 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 2026-06-13 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.