Berlin Institute of Health Center for Regenerative Therapies, Charité-Universitatsmedizin Berlin, Berlin, Germany.
Center for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Lancet Oncol. 2020 Feb;21(2):e104-e116. doi: 10.1016/S1470-2045(19)30729-6.
Chimeric antigen receptor (CAR) T cells represent a potent new approach to treat haematological malignancies. Two CAR T-cell therapies, tisagenlecleucel and axicabtagene ciloleucel, have been approved in Europe and the USA, as well as several other countries, for the treatment of leukaemia and lymphoma. These approvals marked a major milestone in the field of cell and gene therapies. However, the clinical development and regulatory evaluation of these innovative therapies faced several challenges that are considered important lessons learned for future similar products. Here, we examine the products' non-clinical and clinical data packages to outline the challenges encountered during the regulatory evaluation process in Europe, and to provide an update on their performance after authorisation.
嵌合抗原受体 (CAR) T 细胞代表了一种治疗血液系统恶性肿瘤的有效新方法。两种 CAR T 细胞疗法，tisagenlecleucel 和 axicabtagene ciloleucel，已在欧洲和美国以及其他几个国家获得批准，用于治疗白血病和淋巴瘤。这些批准标志着细胞和基因治疗领域的一个重大里程碑。然而，这些创新疗法的临床开发和监管评估面临着一些挑战，这些挑战被认为是为未来类似产品提供了重要的经验教训。在这里，我们检查了产品的非临床和临床数据包，以概述在欧洲监管评估过程中遇到的挑战，并提供授权后的最新性能信息。
