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CTL019 (tisagenlecleucel): CAR-T therapy for relapsed and refractory B-cell acute lymphoblastic leukemia

Authors Vairy S, Lopes Garcia J, Teira P, Bittencourt H

Received 12 April 2018

Accepted for publication 5 September 2018

Published 12 November 2018 Volume 2018:12 Pages 3885—3898


Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 3

Editor who approved publication: Dr Sukesh Voruganti

Stephanie Vairy,* Julia Lopes Garcia,* Pierre Teira, Henrique Bittencourt

Division of Haematology and Oncology, Department of Pediatrics, CHU Sainte-Justine, Université de Montréal, Montréal, QC, Canada

*These authors contributed equally to this work

Over the past decades, survival of patients with acute lymphoblastic leukemia (ALL) has dramatically improved, but the subgroup of patients with relapsed/refractory ALL still continues to have dismal prognosis. As an emerging therapeutic approach, chimeric antigen receptor-modified T-cells (CAR-T) represent one of the few practice-changing therapies for this subgroup of patients. Originally conceived and built in Philadelphia (University of Pennsylvania), CTL019 or tisagenlecleucel, the first CAR-T approved by the US Food and Drug Administration, showed impressive results in refractory/relapsed ALL since the publication on two pediatric patients in 2013. It is in this context that we provide a review of this product in terms of manufacturing, pharmacology, toxicity, and efficacy studies. Evaluation and management of toxicities, particularly cytokine release syndrome and neurotoxicity, is recognized as an essential part of the patient treatment with broader use of IL-6 receptor inhibitor. An under-assessed aspect, the quality of life of patients entering CAR-T cells treatment, will also be reviewed. By their unique nature, CAR-T cells such as tisagenlecleucel operate in a different way than typical drugs, but also provide unique hope for B-cell malignancies.

Keywords: CTL019, tisagenlecleucel, B-cell acute lymphoblastic leukemia

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