New CAR T-cell treatment targeting GPC2 for childhood medulloblastoma
Official title: Next Generation GPC2-CARs for Medulloblastoma
Funded at Stanford University · NIH-11192835 · Data updated 2026-11-12
Engineered immune cells called CAR T cells will be directed at a protein named GPC2 to try to treat children with medulloblastoma.
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 | 2025 |
| Award (this year) | $1,390,720 |
| NIH project number | 5UG3CA297747-02 |
| Project ID | NIH-11192835 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with medulloblastoma, particularly those with high-risk, metastatic, recurrent, or refractory disease whose tumors express the GPC2 protein, would be the most likely candidates.
✕Less likely to help
Patients whose tumors do not express GPC2 or who have other types of brain tumors are unlikely to benefit from this specific approach.
What this research studies
If your child has medulloblastoma, this project is developing next-generation CAR T cells that target GPC2, a protein found on many medulloblastoma cells but not on normal postnatal tissues. The team at Stanford is optimizing CAR design in the lab and in animal models to make the cells stronger and longer-lasting. They are specifically working to prevent tumors from escaping treatment by lowering or losing GPC2 on their surface. Successful preclinical work could pave the way for a future clinical trial for children whose tumors express GPC2.
Why it matters
Potential benefit: If successful, this approach could offer a more targeted therapy for high-risk or recurrent pediatric medulloblastoma with less of the lifelong toxicity seen with conventional chemotherapy and radiation.
How similar studies have gone: CAR T-cell therapy has been very successful in certain blood cancers and has shown early promising signals in some pediatric solid brain tumors, but GPC2-directed CARs are largely at the preclinical stage with prior work showing tumor recurrence due to antigen loss.
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.
-
Glial origins and signaling in childhood brain cancer (medulloblastoma)
A new study looks at how glial cells may drive relapse and growth in childhood medulloblastoma, with the goal of finding targeted treatments.
-
Next generation GPC2 CARs to treat pediatric medulloblastoma
A new CAR T-cell therapy approach targets a protein common on child brain tumor cells to improve outcomes for kids with medulloblastoma.
-
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.