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-10221086

This study is looking at a specific protein that helps a serious childhood cancer called alveolar rhabdomyosarcoma grow, with the hope of finding new ways to treat this cancer and help kids live healthier lives.

Quick facts

Grant typeNIH-funded research
Study typeNIH-funded research
Funding institutionDuke University NIH-funded
Lab location1 site (Durham, United States)
Project IDNIH-10221086 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 the development of alveolar rhabdomyosarcoma (ARMS), 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 a combination of advanced biological techniques and data analysis, the research seeks to uncover the mechanisms that allow this protein to promote tumor growth. Ultimately, the goal is to create effective treatments that can improve survival rates for affected children.

Who could benefit from this research

Good fit: Ideal candidates for this research are children diagnosed with alveolar rhabdomyosarcoma, particularly those with the PAX3-FOXO1 fusion protein.

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 new, targeted therapies that significantly improve survival rates for children with PAX3-FOXO1-positive rhabdomyosarcoma.

How similar studies have performed: While the specific targeting of the PAX3-FOXO1 fusion protein is a novel approach, similar strategies 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.