A phase I clinical trial demonstrated successful long-term responses in pediatric patients with neuroblastoma treated with first-generation chimeric antigen receptor (CAR) T-cell therapy. The study showed a sustained complete remission lasting more than 18 years post-infusion in one patient, indicating the potential of GD2 CAR-ATC/VST therapy in achieving long-term disease control. Despite the absence of costimulatory domains in the vectors used, the therapy was found to be safe and effective. Adverse events primarily manifested as sensorineural hearing loss. This research demonstrates the potential of CAR T-cell therapy for improving outcomes in children with refractory/relapsed neuroblastoma.
18-Year Neuroblastoma Remission in a Patient After GD2 CAR T-Cell Therapy
Conexiant
February 20, 2025