Long-term outcomes of a phase 1 trial of GD2-directed CAR-T cell therapy in patients with neuroblastoma

A phase 1 trial open to accrual from 2004 to 2009 at Baylor College of Medicine (Houston, TX) included 19 children with relapsed or refractory neuroblastoma, who were treated with EBV-specific T lymphocytes and CD2-activated T cells, each expressing CARs targeting GD2 but without embedded co-stimulatory sequence (first-generation CARs). Twelve patients died between 2 months and 7 years after treatment, owing to relapsed neuroblastoma. Of the remaining 7 patients, 5 continued in follow-up for at least 13 years after treatment. Three patients achieve complete response that was sustained in two patients, one for 8 year until loss to follow-up, and one for more than 18 years. The authors conclude that these data suggest that CAR-T cells have the potential to provide long-term benefits to patients with solid tumors.