How GLI2-driven medulloblastoma starts and grows in children
Official title: Oncogenic mechanisms underlying GLI2-amplified medulloblastoma
Funded at Children's Research Institute · NIH-11300978 · Data updated 2026-11-12
Looking at whether blocking a cancer growth pathway called MAPK can slow or stop aggressive GLI2-driven medulloblastoma in children.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Research Institute NIH-funded |
| Lab location | 1 site (Washington, United States) |
| Project start | March 16, 2023 |
| Project end | February 29, 2028 |
| Funding year | 2026 |
| Award (this year) | $1 |
| NIH project number | 5R01NS129653-04 |
| Project ID | NIH-11300978 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children diagnosed with SHH‑subtype medulloblastoma whose tumors show GLI2 amplification would be the most relevant candidates for future trials based on this work.
✕Less likely to help
Children with medulloblastoma that lack GLI2 amplification or patients with other brain tumor types are unlikely to benefit directly from findings specific to GLI2-driven disease.
What this research studies
Researchers use a genetically engineered mouse model and analyses of human tumor samples to find why GLI2-amplified SHH-medulloblastoma forms and grows. Single-cell RNA sequencing showed a MAPK (MEK/ERK) pathway is active in the tumor’s cells of origin, and researchers will follow up on those findings. They test MEK/ERK inhibitors in animals to see if blocking that pathway slows tumor growth. The goal is to identify drug targets that could be moved toward treatments for children with this aggressive tumor type.
Why it matters
Potential benefit: Could point to new targeted therapies that slow tumor growth and improve survival for children with GLI2-amplified SHH‑medulloblastoma.
How similar studies have gone: Preclinical data reported here showed that MEK/ERK inhibition delayed tumor growth in mouse models and MAPK targeting has worked in other cancers, but applying it specifically to GLI2‑amplified medulloblastoma is relatively new.
Where this research is happening
-
Children's Research Institute
Washington, United States Active
Researchers
- Principal investigator: Pei, Yanxin — Children's Research Institute
- Study coordinator: Pei, Yanxin
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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.