Targeting a specific mutation in brain tumors with RNA nanoparticles
Official title: H3K27M as a Target for RNA-nanoparticles in the Treatment of Diffuse Intrinsic Pontine Glioma
Funded at University of Florida · NIH-10951747 · Data updated 2026-11-12
This study is testing a new treatment for kids with Diffuse Intrinsic Pontine Glioma (DIPG), a tough-to-treat brain tumor, by using special mRNA vaccines that help the immune system fight the cancer, and it hopes to eventually help children with this condition.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project start | July 15, 2019 |
| Project end | June 30, 2025 |
| Funding year | 2024 |
| Award (this year) | $75,099 |
| NIH project number | 3R25NS108939-05S2 |
| Project ID | NIH-10951747 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are pediatric patients diagnosed with Diffuse Intrinsic Pontine Glioma, particularly those with the H3K27M mutation.
✕Less likely to help
Patients with other types of brain tumors or those without the H3K27M mutation may not benefit from this research.
What this research studies
This research focuses on developing a novel treatment for Diffuse Intrinsic Pontine Glioma (DIPG), an inoperable pediatric brain tumor. The approach involves using mRNA vaccines that contain tumor-derived antigens encapsulated in lipid nanoparticles, which can stimulate the immune system to attack the tumor. The study aims to evaluate the safety, feasibility, and effectiveness of these RNA nanoparticles in animal models, with the hope of translating these findings to human patients. The research also investigates the specific H3K27M mutation found in DIPG, which serves as a target for this innovative therapy.
Why it matters
Potential benefit: If successful, this research could provide a new, effective treatment option for children suffering from DIPG and potentially other difficult-to-treat cancers.
How similar studies have gone: Previous studies have shown promising results using similar RNA nanoparticle approaches in animal models, indicating potential for success in human applications.
Where this research is happening
-
University of Florida
Gainesville, United States Active
Researchers
- Principal investigator: Okun, Michael S — University of Florida
- Study coordinator: Okun, Michael S
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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.