New mouse models that mirror high-risk childhood rhabdomyosarcoma
Official title: Development of a Clinically Relevant Genetically Engineered Mouse Model for Human High-Risk Rhabdomyosarcoma
Funded at Sanford Research/usd · NIH-11247133 · Data updated 2026-11-12
Researchers are creating mouse versions of high-risk childhood rhabdomyosarcoma to test whether blocking the NOTCH1 pathway can slow tumor growth.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Sanford Research/usd NIH-funded |
| Lab location | 1 site (Sioux Falls, United States) |
| Project start | December 11, 2024 |
| Project end | November 30, 2026 |
| Funding year | 2026 |
| Award (this year) | $232,815 |
| NIH project number | 5R21CA286286-02 |
| Project ID | NIH-11247133 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with high-risk or treatment-resistant rhabdomyosarcoma would be the most likely candidates for future trials informed by this work.
✕Less likely to help
Patients with other cancer types or children whose rhabdomyosarcoma is low-risk and already responsive to standard therapy may not directly benefit from this mouse-model research.
What this research studies
I read that scientists are building genetically engineered mouse models that closely copy high-risk rhabdomyosarcoma in children so they can study how these tumors start and behave. They will turn on the NOTCH1 pathway in the mice because NOTCH1 is linked to worse outcomes in many patient tumors. Using these mice, the team will try drugs that block NOTCH1 and explore whether combining treatments works better than single drugs. The goal is to find promising targeted therapies in the lab before moving them toward trials that could help kids with resistant disease.
Why it matters
Potential benefit: If successful, this work could point to new targeted treatments that slow or stop high-risk rhabdomyosarcoma in children.
How similar studies have gone: Laboratory studies and patient tumor analyses have previously linked NOTCH1 to poorer RMS outcomes and some preclinical drug work shows promise, but faithful genetically engineered mouse models and combination therapy testing remain relatively new.
Where this research is happening
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Sanford Research/usd
Sioux Falls, United States Active
Researchers
- Principal investigator: Tao, Jianning — Sanford Research/usd
- Study coordinator: Tao, Jianning
Related research
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Epitranscriptomic Crosstalk in Alveolar Rhabdomyosarcoma
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.