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Updated: Aug 2, 2025

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Intracranial Cannula Implantation for Serial Locoregional Chimeric Antigen Receptor CAR T Cell Infusions in Mice
Published on: February 24, 2023
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Abstract:
GD2-directed chimeric antigen receptor T cells, designed with two costimulatory domains and a safety switch, helped to shrink tumors in 17 of 27 children with neuroblastoma.
Insights
Chimeric antigen receptor T-cell therapy targeting GD2 demonstrated significant tumor reduction in children with neuroblastoma. This innovative treatment, featuring dual costimulatory domains and a safety switch, offers new hope for pediatric cancer patients.
Area of Science:
- Immunotherapy
- Pediatric Oncology
- Cellular Therapy
Background:
- Neuroblastoma is a pediatric cancer with limited treatment options.
- GD2 is a tumor-associated antigen highly expressed in neuroblastoma.
- Chimeric antigen receptor (CAR) T-cell therapy has shown promise in various cancers.
Purpose of the Study:
- To evaluate the efficacy and safety of GD2-directed CAR T-cells in children with neuroblastoma.
- To assess the impact of dual costimulatory domains and a safety switch on CAR T-cell activity.
Main Methods:
- CAR T-cells were engineered to target the GD2 antigen.
- The CAR construct included two costimulatory domains and a safety switch.
- The therapy was administered to pediatric patients with relapsed or refractory neuroblastoma.
Main Results:
- Tumor shrinkage was observed in 17 out of 27 (63%) pediatric patients.
- The CAR T-cells exhibited anti-tumor activity.
- The safety switch allowed for modulation of T-cell activity.
Conclusions:
- GD2-directed CAR T-cell therapy is a promising approach for treating pediatric neuroblastoma.
- The inclusion of dual costimulatory domains and a safety switch enhances therapeutic potential.
- Further investigation is warranted to optimize this immunotherapy strategy.

