New drugs to block the PAX3‑FOXO1 driver in fusion‑positive alveolar rhabdomyosarcoma
Official title: Chemical Approaches to Modulate PAX3-FOXO1 in Fusion-Positive Alveolar Rhabdomyosarcoma
Funded at Massachusetts Institute of Technology · NIH-11187191 · Data updated 2026-11-12
Small‑molecule approaches aimed at stopping the PAX3‑FOXO1 cancer driver for children with fusion‑positive alveolar rhabdomyosarcoma.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts Institute of Technology NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project start | September 6, 2024 |
| Project end | August 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $2,271,313 |
| NIH project number | 5UM1CA294108-02 |
| Project ID | NIH-11187191 on NIH RePORTER |
Who could benefit from this research
✓Could help
The eventual candidates would be patients (typically children and adolescents) whose tumors are driven by the PAX3‑FOXO1 fusion.
✕Less likely to help
Patients whose tumors lack the PAX3‑FOXO1 fusion, or people with other cancer types, are unlikely to benefit directly from these specific approaches.
What this research studies
This project seeks small molecules that bind the PAX3‑FOXO1 fusion protein or its close partners to stop the fusion from driving tumor growth. The team will run high‑throughput screening, use chemoproteomics and interaction mapping to find where compounds bind, and study how hits change gene activity and chromatin in lab models. Medicinal chemistry and pharmacology work will optimize promising compounds and test them in preclinical models as a step toward therapies for patients.
Why it matters
Potential benefit: If successful, the work could produce targeted drugs that slow or stop tumor growth in children with PAX3‑FOXO1 fusion‑positive alveolar rhabdomyosarcoma.
How similar studies have gone: Directly targeting oncogenic fusion transcription factors with small molecules is relatively new and largely preclinical, though chemoproteomic and degrader strategies have shown promising lab and animal results.
Where this research is happening
-
Massachusetts Institute of Technology
Cambridge, United States Active
Researchers
- Principal investigator: Koehler, Angela Nicole — Massachusetts Institute of Technology
- Study coordinator: Koehler, Angela Nicole
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.