Investigating a fusion protein linked to a rare childhood cancer

Pilot Studies of PAX3-FOXO1 Fusions Proteins in Alveolar Rhabdomyosarcoma

NIH-funded research Pennsylvania State University, the · NIH-10726763

This study is looking at a special protein linked to a rare childhood cancer called alveolar rhabdomyosarcoma, with the goal of finding better treatments by understanding how this protein works.

Quick facts

Grant typeR03 grant
Study typeNIH-funded research
Funding institutionPennsylvania State University, the NIH-funded
Lab location1 site (University Park, United States)
Project IDNIH-10726763 on NIH RePORTER

What this research studies

This research focuses on understanding the PAX3-FOXO1 fusion protein, which is associated with alveolar rhabdomyosarcoma, a rare type of cancer primarily affecting children. The project aims to develop biochemical tools and assays to study the structure and function of this protein, which could lead to new drug development. By utilizing advanced techniques like nuclear magnetic resonance spectroscopy, the researchers hope to uncover how this fusion protein operates at a molecular level, ultimately paving the way for targeted therapies. Patients may benefit from insights gained that could lead to more effective treatments for this aggressive cancer.

Who could benefit from this research

Good fit: Ideal candidates for this research are children diagnosed with alveolar rhabdomyosarcoma or those with genetic predispositions to this condition.

Not a fit: Patients with other types of rhabdomyosarcoma or unrelated cancers may not receive direct benefits from this research.

Why it matters

Potential benefit: If successful, this research could lead to the development of targeted therapies for patients with alveolar rhabdomyosarcoma.

How similar studies have performed: While research on fusion proteins is ongoing, this specific investigation into PAX3-FOXO1 is relatively novel and aims to fill a significant gap in understanding this rare disease.

Where this research is happening

University Park, 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 Disease
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.