Intent-to-treat leukemia remission by CD19 CAR T cells of defined formulation and dose in children and young adults.

ABSTRACT: Transitioning CD19-directed chimeric antigen receptor (CAR) T cells from early-phase trials in relapsed patients to a viable therapeutic approach with predictable efficacy and low toxicity for broad application among patients with high unmet need is currently complicated by product heterogeneity resulting from transduction of undefined T-cell mixtures, variability of transgene expression, and terminal differentiation of cells at the end of culture. A phase 1 trial of 45 children and young adults with relapsed or refractory B-lineage acute lymphoblastic leukemia was conducted using a CD19 CAR product of defined CD4/CD8 composition, uniform CAR expression, and limited effector differentiation. Products meeting all defined specifications occurred in 93% of enrolled patients. The maximum toler

SUBMITTER: Gardner RA 

PROVIDER: S-EPMC5482103 | biostudies-literature | 2017 Jun

REPOSITORIES: biostudies-literature