Understanding the causes of a specific type of childhood brain cancer.
Official title: Oncogenic mechanisms underlying GLI2-amplified medulloblastoma
Funded at Children's Research Institute · NIH-11042848 · Data updated 2026-11-12
This study is looking at a type of brain tumor called medulloblastoma, which is common in kids and can be really tough to treat, especially the GLI2-amplified kind; researchers want to understand how this tumor grows so they can find better ways to help children who have it.
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 | 2025 |
| Award (this year) | $372,698 |
| NIH project number | 5R01NS129653-03 |
| Project ID | NIH-11042848 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with GLI2-amplified medulloblastoma, particularly those under 11 years old.
✕Less likely to help
Patients with other subtypes of medulloblastoma or those who are older than 11 years may not benefit from this specific research.
What this research studies
This research focuses on medulloblastoma, the most common malignant brain tumor in children, particularly the GLI2-amplified subtype which has a very poor prognosis. The study aims to uncover the cellular and molecular mechanisms that drive this type of tumor, using an engineered mouse model to explore how GLI2 influences tumor development. By identifying the specific cells involved and the pathways activated in these tumors, the research seeks to find new therapeutic targets that could lead to more effective treatments for affected children.
Why it matters
Potential benefit: If successful, this research could lead to the development of new treatments for children with GLI2-amplified medulloblastoma, significantly improving survival rates.
How similar studies have gone: Other research has shown promise in targeting specific pathways in similar types of tumors, indicating that this approach could lead to significant advancements in treatment.
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
Related research
Other NIH-funded projects on the same topics.
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Glial origins and signaling in childhood brain cancer (medulloblastoma)
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-
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-
Targeting medulloblastoma with multispecific T-cell engagers
A pediatric brain cancer treatment study tests a new multi-targeted T-cell engager designed to reach brain tumors and help the immune system kill cancer cells.
-
Efflux-aware brain barrier model to guide pediatric medulloblastoma drug discovery
A study using a pediatric brain cancer model to test how radiation-activated digoxin-like compounds could safely reach brain tumors in children.
-
Overcoming medulloblastoma resistance in kids using in vivo perturb-seq and preclinical therapies
A patient-focused study aiming to find new, less toxic ways to treat pediatric medulloblastoma by using advanced genetic screening to identify effective drug targets in preclinical models.
-
Dysregulation of a key gene complex and its role in Group 4 medulloblastoma
A study explores how disruption of a core gene complex in brain development may drive Group 4 medulloblastoma and seeks to understand new ways to help affected children.
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.