Understanding and targeting a weakness in childhood rhabdomyosarcoma

EXPLORE AND TARGET THE EPIGENETIC VULNERABILITY OF PAX3-FOXO1-DRIVEN RHABDOMYOSARCOMA

NIH-funded research St. Jude Children's Research Hospital · NIH-11122258

This project aims to find new ways to treat a serious childhood cancer called rhabdomyosarcoma by understanding its unique weaknesses.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionSt. Jude Children's Research Hospital NIH-funded
Lab location1 site (Memphis, United States)
Project IDNIH-11122258 on NIH RePORTER

What this research studies

This project focuses on rhabdomyosarcoma, a challenging soft tissue cancer that affects children and young adults. We know that aggressive forms of this cancer, especially alveolar rhabdomyosarcoma, are caused by specific genetic changes. While current treatments have helped some patients, those with advanced disease still face poor outcomes, and existing therapies can have severe long-term side effects for children. Our goal is to discover new targets within these cancer cells that can be blocked, leading to more effective and gentler treatments. By understanding how these cancer cells become vulnerable, we hope to develop better options for young patients.

Who could benefit from this research

Good fit: This research is focused on understanding the biology of rhabdomyosarcoma, particularly the aggressive alveolar subtype, which primarily affects children and adolescent young adults.

Not a fit: Patients with other types of cancer or those not diagnosed with rhabdomyosarcoma would not directly benefit from this specific research.

Why it matters

Potential benefit: If successful, this work could lead to the development of new, more effective, and less toxic therapies for children and young adults with aggressive rhabdomyosarcoma.

How similar studies have performed: While current treatments for rhabdomyosarcoma have improved outcomes for some, new therapeutic targets are urgently needed for aggressive forms of the disease, making this approach novel and critical.

Where this research is happening

Memphis, 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.
Conditions Cancers
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.