儿童嵌合抗原受体T细胞疗法的发展——全面概述

Development of CAR T Cell Therapy in Children-A Comprehensive Overview.

作者信息

Boettcher Michael, Joechner Alexander, Li Ziduo, Yang Sile Fiona, Schlegel Patrick

机构信息

Department of Pediatric Surgery, University Medical Centre Mannheim, University of Heidelberg, 69117 Heidelberg, Germany.

School of Medical Sciences, Faculty of Medicine and Health, University of Sydney, Sydney 2006, Australia.

出版信息

J Clin Med. 2022 Apr 12;11(8):2158. doi: 10.3390/jcm11082158.

Abstract

CAR T cell therapy has revolutionized immunotherapy in the last decade with the successful establishment of chimeric antigen receptor (CAR)-expressing cellular therapies as an alternative treatment in relapsed and refractory CD19-positive leukemias and lymphomas. There are fundamental reasons why CAR T cell therapy has been approved by the Food and Drug administration and the European Medicines Agency for pediatric and young adult patients first. Commonly, novel therapies are developed for adult patients and then adapted for pediatric use, due to regulatory and commercial reasons. Both strategic and biological factors have supported the success of CAR T cell therapy in children. Since there is an urgent need for more potent and specific therapies in childhood malignancies, efforts should also include the development of CAR therapeutics and expand applicability by introducing new technologies. Basic aspects, the evolution and the drawbacks of childhood CAR T cell therapy are discussed as along with the latest clinically relevant information.

摘要

嵌合抗原受体(CAR)T细胞疗法在过去十年中彻底改变了免疫疗法,成功建立了表达CAR的细胞疗法,作为复发和难治性CD19阳性白血病和淋巴瘤的替代治疗方法。CAR T细胞疗法首先获得美国食品药品监督管理局和欧洲药品管理局批准用于儿科和年轻成人患者有其根本原因。通常,由于监管和商业原因,新型疗法先为成人患者开发,然后再适用于儿科。战略和生物学因素都支持了CAR T细胞疗法在儿童中的成功。由于儿童恶性肿瘤迫切需要更有效和特异的疗法,努力还应包括开发CAR疗法,并通过引入新技术扩大其适用性。本文将讨论儿童CAR T细胞疗法的基本方面、发展历程和缺点,以及最新的临床相关信息。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f1e3/9024694/3975aba176b9/jcm-11-02158-g001.jpg

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