Multicenter phase 1-2 trial of GD2-CART01 for relapsed or refractory high-risk neuroblastoma

Children with relapsed or refractory neuroblastoma after first-line therapy have a dismal prognosis, with long-term survival in less than 10% of patients. Immunotherapy with CAR T cells targeting the disialogangloside GD2 on tumor cells is under study as a potential therapy for children with high-risk neuroblastoma. A multicenter phase 1-2 trial in Italy evaluated third-generation GD2-directed CAR T cells (GD2-CART01) in patients with relapsed or refractory high-risk neuroblastoma. Cytokine release syndrome occurred in 74% of the patients and was mild in all but one. Central neurotoxic effects did not develop in any of the patients. Response was seen in 63% of the patients and complete response in 33%. More than half of the patients with complete response maintained response during median follow-up of 1.7 years. Three year event-free survival rate was 36% and three year overall survival rate was 60%. The authors conclude that the use of GD2-CART01 was feasible and safe in treating high-risk neuroblastoma. GD2-CART01 may have a sustained antitumor effect (quick take video summary) .