诊所的CAR-T：小心驾驶

StephenW

Editorial | Published: 01 July 2018

CAR-T in the clinic: drive with care

    Rafael J. Yáñez-Muñoz & Stephan A. Grupp

Gene Therapyvolume 25, pages157–161 (2018) | Download Citation

Cell and gene therapies are in the limelight after a bit of a bumpy ride. Several marketing authorisations have been granted by health authorities around the world, but perhaps the ones attracting most attention are the first USA Food and Drug Administration (FDA) gene therapy approvals for chimeric antigen receptor T-cells (CAR-T), indicated for selected cancers of the haematopoietic system. The approved CAR-T drugs are CD19-directed genetically modified autologous T-cell immunotherapies. Novartis’ Kymriah (tisagenlecleucel) was approved on 30th August 2017, for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse and was the first ever US-approved gene therapy [1, 2]. Kite Pharma/Gilead’s Yescarta (axicabtagene ciloleucel) was approved on 18th October 2017, indicated for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma [3]. Most recently, on 1st May 2018, Kymriah was also approved for the B-cell lymphoma indication [4]. Both drugs have been granted Priority Medicines (PRIME) regulatory support and are undergoing accelerated assessment by the European Medicines Agency (EMA).

StephenW

编辑|发布时间：2018年7月1日

诊所的CAR-T：小心驾驶

    RafaelJ.Yáñez-Muñoz和Stephan A. Grupp

Gene Therapyvolume 25，pages 157-161（2018）|下载引文

经过一段颠簸之后，细胞和基因疗法成为众人瞩目的焦点。世界各地的卫生当局已经授予了几项营销许可，但最受关注的可能是美国食品和药物管理局（FDA）批准的嵌合抗原受体T细胞（CAR-T）的首批基因治疗批准。造血系统的癌症。批准的CAR-T药物是CD19指导的遗传修饰的自体T细胞免疫疗法。 Novartis的Kymriah（tisagenlecleucel）于2017年8月30日获批用于治疗25岁以下患有B细胞前体急性淋巴细胞白血病（ALL）的患者，该患者难治性或在第二次或以后复发，并且是第一个美国 - 批准的基因治疗[1,2]。风筝制药/吉利德的Yescarta（axicabtagene ciloleucel）于2017年10月18日获批，用于治疗两次或更多次全身治疗后复发或难治性大B细胞淋巴瘤的成人患者，包括弥漫性大B细胞淋巴瘤（DLBCL） ）没有特别说明，原发性纵隔大B细胞淋巴瘤，高级别B细胞淋巴瘤和滤泡性淋巴瘤引起的DLBCL [3]。最近，在2018年5月1日，Kymriah也被批准用于B细胞淋巴瘤适应症[4]。这两种药物均已获得优先药物（PRIME）监管支持，并正在接受欧洲药品管理局（EMA）的加速评估。

StephenW

https://www.nature.com/articles/ ... Id=MTQ0MDIwMTg2NQS2