Finding new treatments for aggressive brain tumors in children with a specific genetic mutation
Development of A High-Throughput Screen for Identification of Targeted Therapies in Brainstem Tumors with the H3K27M Mutation
This study is looking for new treatments for kids with aggressive brain tumors that have a specific genetic change, hoping to find better options to help them fight their illness.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mayo Clinic Rochester NIH-funded |
| Lab location | 1 site (Rochester, United States) |
| Project ID | NIH-10704481 on NIH RePORTER |
What this research studies
This research focuses on developing a high-throughput screening method to identify targeted therapies for pediatric brain tumors, particularly those with the H3K27M mutation. These tumors, including Diffuse Intrinsic Pontine Gliomas (DIPG), are highly aggressive and currently have no effective treatments. By utilizing advanced techniques, including CRISPR, the research aims to understand how genetic changes affect tumor behavior and to discover new therapeutic options. The ultimate goal is to improve treatment outcomes for affected children by identifying drugs that can specifically target the underlying genetic alterations.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with high-grade gliomas, particularly those with the H3K27M mutation.
Not a fit: Patients with brain tumors that do not have the H3K27M mutation may not benefit from this specific research.
Why it matters
Potential benefit: If successful, this research could lead to the development of effective therapies that significantly improve survival rates and quality of life for children with these aggressive brain tumors.
How similar studies have performed: While there have been numerous clinical trials for pediatric brain tumors, this approach utilizing high-throughput screening and CRISPR technology is relatively novel and has not been extensively tested in this context.
Where this research is happening
Rochester, United States
- Mayo Clinic Rochester — Rochester, United States (Active)
Researchers
- Principal investigator: Daniels, David — Mayo Clinic Rochester
- Study coordinator: Daniels, David
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.