Dual-Target CAR T-Cell Therapy Produces Deep Remissions in Pediatric B-ALL

Bicistronic CD19/CD22 chimeric antigen receptor (CAR) T-cell therapy produced high rates of deep remission in children with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL), according to a multicenter phase 2 nonrandomized clinical trial published in JAMA Oncology.

The open-label trial enrolled patients at 5 centers in China. Investigators evaluated a product engineered to target both CD19 and CD22, an approach intended to reduce relapse caused by loss of a single antigen after CD19-directed therapy. The analysis included 261 patients with relapsed or refractory disease; their mean age was 8.2 years.

Complete remission with negative minimal residual disease was achieved by 259 patients, or 99.2%. Event-free survival was 70.9% at 12 months, 63.2% at 24 months, and 61.7% at 36 months. Among selected patients, consolidative transplantation was associated with higher 24-month event-free survival than no transplant, 85.7% versus 57.9% (P = .004), although transplant assignment was not randomized.

Treatment-related toxic effects were common. Grade 3 or 4 cytokine release syndrome occurred in 49.4% of patients, and immune effector cell-associated neurotoxicity occurred in 13%. Because the trial was nonrandomized and lacked an active comparator, comparative effectiveness versus other CAR T-cell strategies remains uncertain.

The results support further prospective evaluation of dual-target CAR T-cell therapy as a strategy for achieving durable disease control in pediatric B-ALL.

Source: Wan X, Tang Y, Cai J, et al. Bicistronic CD19/CD22 CAR T-cell therapy in pediatric B-cell acute lymphoblastic leukemia: a nonrandomized clinical trial. JAMA Oncol. Published online September 3, 2026. doi:10.1001/jamaoncol.2026.3460