GD2-CART01 for relapsed or refractory high-risk neuroblastoma

Third-generation GD2-CAR T cells expressing the inducible caspase 9 suicide gene (G2-CART01) was found to be safe for treatment of patients with high-risk neuroblastoma. This phase I/II clinical trial found that no dose-limiting toxicities were recorded for the 27 children and young adults (aged 1-25 years) in the study, and the recommended dose was 10x106 CAR-positive T cells/kg. Cytokine release syndrome was seen in 20 of 27 patients. Complete response was seen in 9 patients, partial response in 8, and a response was seen in 17. Among the children who received the recommended dose, the 3-year overall survival rate was 60%, and the event-free survival rate was 36%.