Improving Treatments for Childhood Rhabdomyosarcoma

Targeting mTORC1 translational control in FOXO1 fusion positive rhabdomyosarcoma

NIH-funded research University of California, San Francisco · NIH-11126020

This research looks for new ways to stop a type of childhood cancer called rhabdomyosarcoma from growing and becoming resistant to current medicines.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of California, San Francisco NIH-funded
Lab location1 site (San Francisco, United States)
Project IDNIH-11126020 on NIH RePORTER

What this research studies

We are exploring how a specific type of childhood cancer, rhabdomyosarcoma with a FOXO1 gene fusion, relies on a process called mTORC1 for its growth. While new medicines that block mTORC1 show promise in initial tests, the cancer can sometimes return. Our goal is to understand why these cancers become resistant and find better ways to combine treatments to achieve lasting cures. We hope to provide information that helps doctors use these new medicines more effectively for children with this challenging cancer.

Who could benefit from this research

Good fit: This research is relevant for children, specifically those aged 0-11 years, who have rhabdomyosarcoma with a FOXO1 gene fusion.

Not a fit: Patients with other types of cancer or rhabdomyosarcoma without the FOXO1 gene fusion may not directly benefit from this specific research.

Why it matters

Potential benefit: If successful, this work could lead to more effective and lasting treatments for children with a difficult-to-treat form of rhabdomyosarcoma, potentially improving cure rates.

How similar studies have performed: New mTORC1 inhibitors are currently in early clinical testing and have shown strong results in laboratory models, but resistance remains a challenge that this work aims to address.

Where this research is happening

San Francisco, 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.