New CAR T cell therapy for treating childhood brain tumors
Official title: Next Generation GPC2-CARs for Medulloblastoma
Funded at Stanford University · NIH-11051634 · Data updated 2026-11-12
This study is testing a new type of treatment for children with medulloblastoma, a common brain tumor, by using specially designed immune cells to target the cancer while being kinder to their healthy cells.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project start | September 13, 2024 |
| Project end | August 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $1,390,720 |
| NIH project number | 1UG3CA297747-01 |
| Project ID | NIH-11051634 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children aged 0-11 years diagnosed with medulloblastoma, particularly those with high-risk or recurrent forms of the disease.
✕Less likely to help
Patients with non-cancerous conditions or those diagnosed with other types of brain tumors not expressing GPC2 may not benefit from this research.
What this research studies
This research focuses on developing a novel CAR T cell therapy targeting GPC2, a protein overexpressed in medulloblastoma, the most common malignant brain tumor in children. By using a specialized approach that enhances the effectiveness of CAR T cells, the research aims to create a targeted treatment that minimizes toxicity compared to traditional therapies. The methodology involves optimizing CAR T cells to specifically attack cancer cells while sparing normal tissues, potentially leading to better outcomes for young patients with high-risk tumors.
Why it matters
Potential benefit: If successful, this research could provide a safer and more effective treatment option for children with medulloblastoma.
How similar studies have gone: Previous research has shown promising results with CAR T cell therapies in pediatric brain tumors, indicating potential for success with this novel approach.
Where this research is happening
-
Stanford University
Stanford, United States Active
Researchers
- Principal investigator: Mackall, Crystal — Stanford University
- Study coordinator: Mackall, Crystal
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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Next generation GPC2 CARs to treat pediatric medulloblastoma
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Targeting medulloblastoma with multispecific T-cell engagers
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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.
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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.