Creating a mouse model to study and treat high-risk rhabdomyosarcoma in children

Development of a Clinically Relevant Genetically Engineered Mouse Model for Human High-Risk Rhabdomyosarcoma

['FUNDING_R21'] · SANFORD RESEARCH/USD · NIH-11044502

This study is creating special mice that have a type of cancer similar to what some kids get, called rhabdomyosarcoma, to help scientists learn more about what causes it and how it grows, with the hope of finding better treatments for children facing this tough illness.

Quick facts

Phase['FUNDING_R21']
Study typeNih_funding
SexAll
SponsorSANFORD RESEARCH/USD (nih funded)
Locations1 site (SIOUX FALLS, UNITED STATES)
Trial IDNIH-11044502 on ClinicalTrials.gov

What this research studies

This research focuses on developing genetically engineered mouse models that mimic human high-risk rhabdomyosarcoma, a common and aggressive cancer in children. By studying these models, researchers aim to understand the genetic factors that drive this cancer and how it progresses. The project will specifically investigate the NOTCH1 pathway, which is linked to poorer survival outcomes in rhabdomyosarcoma, to identify potential therapeutic targets. The ultimate goal is to evaluate new treatment strategies that could improve outcomes for affected children.

Who could benefit from this research

Good fit: Ideal candidates for this research are children under 11 years old diagnosed with high-risk rhabdomyosarcoma.

Not a fit: Patients with other types of cancer or those over the age of 11 may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to more effective therapies for children with high-risk rhabdomyosarcoma, potentially improving survival rates.

How similar studies have performed: Previous research using genetically engineered mouse models has shown promise in understanding and treating various cancers, suggesting that this approach could be effective for rhabdomyosarcoma as well.

Where this research is happening

SIOUX FALLS, 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.

View on NIH RePORTER →

Conditions: anti-cancer therapy, Cancer Biology, Cancer Genes, cancer genomics

Last reviewed 2026-05-15 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.