Approved CAR T cell therapies: ice bucket challenges on glaring safety risks and long-term impacts

• DOI: 10.1016/j.drudis.2018.02.012
• Publication Date: 2018-03-01T20:10:46Z
• Authors: Ping-Pin Zheng; Johan M. Kros; Jin Li
• Keywords: 0303 health sciences; 03 medical and health sciences; Treatment Outcome; Drug Resistance, Neoplasm; Precursor B-Cell Lymphoblastic Leukemia-Lymphoma; EMC OR-01; Antigens, CD19; Quality of Life; Receptors, Antigen, T-Cell; Humans; 3. Good health

Two autologous chimeric antigen receptor (CAR) T cell therapies (Kymriah™ and Yescarta™) were recently approved by the FDA. Kymriah™ is for the treatment of pediatric patients and young adults with refractory or relapse (R/R) B cell precursor acute lymphoblastic leukemia and Yescarta™ is for the treatment of adult patients with R/R large B cell lymphoma. In common, both are CD19-specific CAR T cell therapies lysing CD19-positive targets. Their dramatic efficacy in the short term has been highlighted by many media reports. By contrast, their glaring safety gaps behind the miracles remain much less addressed. Here, we focus on addressing the crucial challenges in relation to the gaps.