Finding new treatment targets for certain childhood brain tumors
Identifying genetic vulnerabilities in KIAA1549-BRAF mutant pediatric low-grade gliomas
This study is looking for better and safer treatments for children with a specific type of brain tumor that has a genetic change called the KIAA1549-BRAF fusion, using new technology to find ways to help them feel better without the harsh side effects of current medicines.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Broad Institute, INC. NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project ID | NIH-10951512 on NIH RePORTER |
What this research studies
This research focuses on pediatric low-grade gliomas, a type of brain tumor that often has a specific genetic mutation involving the BRAF gene. The study aims to identify new therapeutic targets that could improve treatment outcomes for children with tumors that have the KIAA1549-BRAF fusion. Using advanced CRISPR technology, researchers will explore genetic vulnerabilities in these tumors to find alternatives to current BRAF inhibitors, which can have significant side effects. The goal is to develop safer and more effective treatment options for affected children.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with pediatric low-grade gliomas that harbor the KIAA1549-BRAF fusion mutation.
Not a fit: Patients with other types of brain tumors or those without the KIAA1549-BRAF fusion mutation may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to improved treatment strategies that minimize side effects and enhance the effectiveness of therapies for children with these brain tumors.
How similar studies have performed: Previous research has shown promise in targeting genetic vulnerabilities in cancer, suggesting that this approach could yield beneficial results.
Where this research is happening
Cambridge, United States
- Broad Institute, INC. — Cambridge, United States (Active)
Researchers
- Principal investigator: Misek, Sean Alexander — Broad Institute, INC.
- Study coordinator: Misek, Sean Alexander
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 below.