• C Chabannon, R Bouabdallah, S Fürst, A Granata, C Saillard, N Vey, D Mokart, E Fougereau, C Lemarie, B Mfarrej, D Blaise, and B Calmels.
• Institut Paoli-Calmettes (IPC), 13009 Marseille, France; Aix-Marseille Université, 13007 Marseille, France; Inserm, Aix-Marseille Université, CNRS, Institut Paoli-Calmettes, 13009 Marseille, France; Inserm, Aix-Marseille Université, Institut Paoli-Calmettes, AP-HM, CBT-1409, centre d'Investigations Cliniques en Biothérapies, 13009 Marseille, France. Electronic address: chabannonc@ipc.unicancer.frchristian.
• Rev Med Interne. 2019 Aug 1; 40 (8): 545-552.

AbstractCAR-T cells are genetically modified human lymphocytes and gene therapy medicinal products. They are developed to treat cancers that express a membrane antigen targeted by the CAR. The FDA approved the two first-in-class medicinal products in 2017 and EMA in August 2018; both are autologous CAR-T cells targeting CD19 that is expressed at the surface of normal B-cells throughout their differentiation, and on B-cell lymphoid malignancies. Clinical efficacy was demonstrated for B-cell acute lymphoblastic leukemias, non-Hodgkin's lymphoma and chronic lymphocytic leukemia, although the marketing authorizations are less liberal in terms of indications. Manufacturing of these personalized treatments necessitates that a novel organization and supply chain be set in place, to ensure product preservation, patient safety and compliance with complex regulatory requirements. Side effects are commensurate with clinical efficacy and can be life-threatening: proper management imposes tight coordination between various specialists, particularly between hematologists and intensive care practitioners. High pricing for these treatments is part of a long-term trend for increasing costs of innovations in hematology and oncology; it questions the ability of healthcare systems to sustain their reimbursement.Copyright © 2018 Société Nationale Française de Médecine Interne (SNFMI). Published by Elsevier Masson SAS. All rights reserved.

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