Using targeted therapy to treat a deadly brain tumor in children
Official title: Inducing H3F3A exon skipping with antisense oligonucleotides as an approach to treat diffuse intrinsic pontine glioma
Funded at State University New York Stony Brook · NIH-11099689 · Data updated 2026-11-12
This study is testing a new treatment for children with a tough brain tumor called DIPG, using special molecules to target a specific gene mutation in the tumor, with the hope of helping kids live longer and healthier lives.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | State University New York Stony Brook NIH-funded |
| Lab location | 1 site (Stony Brook, United States) |
| Project start | June 1, 2023 |
| Project end | February 28, 2027 |
| Funding year | 2025 |
| Award (this year) | $54,538 |
| NIH project number | 5F30CA281196-03 |
| Project ID | NIH-11099689 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with diffuse intrinsic pontine glioma, particularly those with the H3F3A mutation.
✕Less likely to help
Patients with other types of brain tumors or those without the specific H3F3A mutation may not benefit from this research.
What this research studies
This research focuses on developing a new treatment for diffuse intrinsic pontine glioma (DIPG), a highly aggressive brain tumor in children. The approach involves using antisense oligonucleotides (ASOs) to induce changes in the tumor's genetic material, specifically targeting a mutation in the H3F3A gene that is common in DIPG. By doing so, the researchers aim to promote neural differentiation and potentially improve survival rates for affected children. The study builds on previous findings that showed promise in using ASOs to combat this type of cancer.
Why it matters
Potential benefit: If successful, this research could lead to a novel treatment that significantly improves survival rates for children with DIPG.
How similar studies have gone: Previous research has shown promise in using antisense oligonucleotides for similar applications, indicating potential for success in this approach.
Where this research is happening
-
State University New York Stony Brook
Stony Brook, United States Active
Researchers
- Principal investigator: Yang, Lucia — State University New York Stony Brook
- Study coordinator: Yang, Lucia
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Targeting a specific mutation in brain tumors with RNA nanoparticles
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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.