Targeting the PAX3-FOXO1 protein in a deadly childhood cancer

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

NIH-funded research Duke University · NIH-9833682

This study is looking at a specific protein that helps a tough childhood cancer called alveolar rhabdomyosarcoma, with the goal of finding new ways to treat kids who have this illness.

Quick facts

Grant typeNIH-funded research
Study typeNIH-funded research
Funding institutionDuke University NIH-funded
Lab location1 site (Durham, United States)
Project IDNIH-9833682 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 alveolar rhabdomyosarcoma, a severe form of childhood cancer. The project aims to identify the molecular vulnerabilities associated with this protein to develop new therapeutic strategies. By employing advanced biological and clinical techniques, the research seeks to uncover the mechanisms that drive tumor growth and find ways to disrupt them. This comprehensive approach could lead to more effective treatments for affected children.

Who could benefit from this research

Good fit: Ideal candidates for this research are children diagnosed with PAX3-FOXO1-positive alveolar 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 significantly improve survival rates and treatment options for children diagnosed with alveolar rhabdomyosarcoma.

How similar studies have performed: While the PAX3-FOXO1 fusion protein has been identified for some time, this research represents a novel and comprehensive approach to targeting it, as previous strategies have been limited and largely untested.

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 2026-06-10 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.