Phase 1-2 study of anti-CLL based CAR-T therapy for relapsed or refractory AML in children

C-type lectin-like molecule-1 (CLL1) is preferentially expressed on acute myeloid leukemia (AML) stem cells and AML blasts, and can be considered as AML-associated antigen. Anti-CLL1 based CAR-T cells exhibited effective tumor-killing capacity in vitro and in an AML-bearing mouse model. A multicenter phase 1-2 study in China evaluated autologous anti-CLL1 CAR-T cell immunotherapy for relapsed or refractory AML in children. The treatment was well-tolerated and resulted in morphologic leukemia-free state and minimal residual disease negativity in 4 of 8 patients. The authors conclude that anti-CLL1 based CAR-T cell immunotherapy was an effective option for R/R AML in children.