Two experimental CD19-CAR T-cell products for treatment of R/R infant B-ALL
Annesley C, Lamble AJ, Summers C, et al. Feasibility and Favorable Responses Following Investigational CAR T-Cell Therapy for Relapsed and Refractory Infant ALL. Blood Advances. 2024; (doi: 10.1182/bloodadvances.2024012638).
Early evidence supports a deeper look into candidate chimeric antigen receptor (CAR) T-cell therapies for the treatment of relapsed and refractory B-cell acute lymphoblastic leukemia (B-ALL) in babies. Two experimental CD19-CAR T-cell products, SCRI-CAR19 or SCRI-CAR19x22, were administered to patients with a median age of 22.5 months at enrollment across three clinical studies. Among the 17 treated patients, 16 reached full remission with no detectable minimal residual disease. At a median follow-up of 35.8 months, the study authors recorded 1-year leukemia-free survival and 1-year overall survival at rates of 75.0% and 76.5%, respectively. Investigators were further encouraged by the safety findings, which confirmed neurotoxicity in only two infants and at ≤Grade 2 for both. Cytokine release syndrome was more common, affecting 14 patients; however, only one case was Grade 3. The results highlight the feasibility of manufacturing and infusing CAR-T cell products in the setting of R/R infant B-ALL, with promising outcomes.