What we learned from bench to bedside with the neuroblastoma targeting CD171-specific CAR

Introduction: Despite efficacy of chimeric antigen receptor (CAR) T cell therapy in leukemia and lymphoma patients, similar clinical responses in solid tumor patients is an unrealized objective. We developed a CAR specific for CD171, an antigen expressed in several solid tumors including neuroblastoma. Methods: Since CD171 is also expressed in healthy tissues, we performed a safety study in non-human primates. Further, we analyzed influence of CAR extracellular spacer and cytoplasmic signaling domain variants on magnitude of cytotoxic CD8+T lymphocyte (CTL) activation for tumor cell cytolysis and cytokine secret…