Chemical ways to block PAX3-FOXO1 in a dangerous childhood cancer
Official title: Chemical Approaches to Modulate PAX3-FOXO1 in Fusion-Positive Alveolar Rhabdomyosarcoma
Funded at Massachusetts Institute of Technology · NIH-11401672 · Data updated 2026-11-12
A study to find small molecules that can interfere with the cancer-driving PAX3-FOXO1 fusion protein in alveolar rhabdomyosarcoma, aiming to slow or stop tumor growth in affected children.
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 | 2026 |
| Award (this year) | $2,262,989 |
| NIH project number | 5UM1CA294108-03 |
| Project ID | NIH-11401672 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and young adults diagnosed with fusion-positive alveolar rhabdomyosarcoma or their families may benefit from future trials or related therapies derived from this work.
✕Less likely to help
People without FP-RMS or those who cannot participate in future translational studies arising from this work may not benefit directly.
What this research studies
This project brings together chemists, biologists, and clinicians to screen and study compounds that might directly bind PAX3-FOXO1 or nearby proteins. The team will use high-throughput screening, chemoproteomics, and molecular interaction analyses to understand how potential drugs interact with the fusion protein and its partners. The goal is to identify drug-like molecules and understand multiple ways they could block the fusion protein’s cancer-driving activity. If successful, findings could guide the development of therapies for FP-RMS, a childhood cancer with urgent unmet medical need.
Why it matters
Potential benefit: If successful, the research could lead to new drugs that specifically disrupt the PAX3-FOXO1 fusion, offering a targeted treatment option for FP-RMS.
How similar studies have gone: Previous efforts in fusion oncoproteins have shown that small molecules can modulate such proteins, but success with FP-RMS is still emerging and this program aims to build on that foundation.
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
Other NIH-funded projects on the same topics.
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Targeting the PAX3-FOXO1 protein in a deadly childhood cancer
This study is looking at a specific protein that helps a tough childhood cancer called alveolar rhabdomyosarcoma, with the goal of finding new ways to treat kids who have this illness.
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A project to understand why a pediatric muscle cancer called rhabdomyosarcoma spreads, and to test ideas that could slow or stop it by helping cancer cells mature properly.
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A research project aims to create mouse models that mimic high-risk pediatric rhabdomyosarcoma and test therapies that target the NOTCH1 pathway to slow or stop tumor growth.
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Understanding infantile rhabdomyosarcoma biology and new treatment targets
A new grant is studying why infantile rhabdomyosarcoma develops and what drugs or strategies could specifically target it in young children.
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Targeting CLK2 to Fight Rhabdomyosarcoma
Aims to see if blocking CLK2 can weaken rhabdomyosarcoma cancer cells, potentially offering a new treatment for kids and young adults.
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Epitranscriptomic Crosstalk in Alveolar Rhabdomyosarcoma
A pediatric cancer study that looks at how chemical marks on messenger RNA may influence the behavior of alveolar rhabdomyosarcoma and point toward newer treatment strategies.
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.