Finding ways to target a specific protein in a childhood cancer

Chemical Approaches to Modulate PAX3-FOXO1 in Fusion-Positive Alveolar Rhabdomyosarcoma

NIH-funded research Massachusetts Institute of Technology · NIH-10987609

This study is looking for new ways to help kids with a tough type of cancer called fusion-positive alveolar rhabdomyosarcoma by finding special chemicals that can block a protein that helps the cancer grow.

Quick facts

Grant typeNIH-funded research
Study typeNIH-funded research
Funding institutionMassachusetts Institute of Technology NIH-funded
Lab location1 site (Cambridge, United States)
Project IDNIH-10987609 on NIH RePORTER

What this research studies

This research focuses on fusion-positive alveolar rhabdomyosarcoma, a severe childhood cancer driven by the PAX3-FOXO1 protein. The team aims to develop chemical agents that can inhibit this protein's function, which is crucial for the cancer's progression. By utilizing advanced techniques in chemistry and biology, they will explore various methods to disrupt the activity of PAX3-FOXO1 and its interactions with other proteins. This coordinated approach involves a collaboration of experts in high-throughput screening and medicinal chemistry to identify potential therapeutic compounds.

Who could benefit from this research

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

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

Why it matters

Potential benefit: If successful, this research could lead to new treatments that significantly improve outcomes for children with this aggressive cancer.

How similar studies have performed: While targeting fusion proteins in cancers is a challenging area, there have been promising advancements in similar approaches, indicating potential for success.

Where this research is happening

Cambridge, 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.