Finding ways to target a specific protein in a childhood cancer
Official title: Chemical Approaches to Modulate PAX3-FOXO1 in Fusion-Positive Alveolar Rhabdomyosarcoma
Funded at Massachusetts Institute of Technology · NIH-10987609 · Data updated 2026-11-12
This study is looking for new ways to help kids with a tough type of cancer called fusion-positive alveolar rhabdomyosarcoma by finding special chemicals that can block a protein that helps the cancer grow.
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 | 2024 |
| Award (this year) | $2,326,552 |
| NIH project number | 1UM1CA294108-01 |
| Project ID | NIH-10987609 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with fusion-positive alveolar rhabdomyosarcoma.
✕Less likely to help
Patients with other types of rhabdomyosarcoma or those without the PAX3-FOXO1 fusion may not benefit from this research.
What this research studies
This research focuses on fusion-positive alveolar rhabdomyosarcoma, a severe childhood cancer driven by the PAX3-FOXO1 protein. The team aims to develop chemical agents that can inhibit this protein's function, which is crucial for the cancer's progression. By utilizing advanced techniques in chemistry and biology, they will explore various methods to disrupt the activity of PAX3-FOXO1 and its interactions with other proteins. This coordinated approach involves a collaboration of experts in high-throughput screening and medicinal chemistry to identify potential therapeutic compounds.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that significantly improve outcomes for children with this aggressive cancer.
How similar studies have gone: While targeting fusion proteins in cancers is a challenging area, there have been promising advancements in similar approaches, indicating potential for success.
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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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.