New CAR T-cell treatment targeting GPC2 for childhood medulloblastoma
Next Generation GPC2-CARs for Medulloblastoma
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 ID | NIH-11192835 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: Patients whose tumors do not express GPC2 or who have other types of brain tumors are unlikely to benefit from this specific approach.
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 performed: 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, United States
- Stanford University — Stanford, United States (Active)
Researchers
- Principal investigator: Mackall, Crystal — Stanford University
- Study coordinator: Mackall, Crystal
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 below.