CNS Endothelial Cell Activation Emerges as a Driver of CAR T Cell-Associated Neurotoxicity

Central nervous system (CNS) toxicity associated with chimeric antigen receptor-based therapeutics has emerged as a significant cause of morbidity and mortality, and insights into the pathophysiology of this syndrome have been lacking. A new study provides evidence that cytokine-induced CNS endothelial cell activation leading to disruption of the blood-brain barrier plays an early and critical role in this phenomenon. These insights provide new opportunities for targeted therapeutic interventions to modulate endothelial cell activation. (C) 2017 AACR.