The New England Journal of MedicineGD2-CART01 for Relapsed or Refractory High-Risk Neuroblastoma

Twenty-seven (27) children with heavily pretreated neuroblastoma (12 with refractory disease, 14 with relapsed disease, and one with a complete response at the end of first-line therapy) were enrolled in this academic, phase 1-2 clinical trial to test autologous, third-generation GD2-CAR T cells expressing the inducible caspase 9 suicide gene (GD2-CART01). The use of GD2-CART01 in the treatment of high-risk neuroblastoma was feasible and safe. Treatment-related toxic effects emerged, and the suicide gene was activated to control side effects. GD2-CART01 may have long-term antitumor activity.