Schubert Maria-Luisa, Hückelhoven Angela, Hoffmann Jean-Marc, Schmitt Anita, Wuchter Patrick, Sellner Leopold, Hofmann Susanne, Ho Anthony D, Dreger Peter, Schmitt Michael
Department of Internal Medicine V, Heidelberg University Hospital , Heidelberg, Germany .
Hum Gene Ther. 2016 Oct;27(10):758-771. doi: 10.1089/hum.2016.097. Epub 2016 Jul 31.
Novel therapies with chimeric antigen receptor (CAR)-transduced T cells (TCs) sparked new hope for patients with relapsed or refractory CD19-positive leukemia or lymphoma even after stem cell therapies. This review focuses on CARs recognizing the B cell antigen CD19. Both retroviral and lentiviral vectors are used, encoding various anti-CD19 CAR constructs comprising costimulatory molecules such as CD28, CD137/4-1BB, and OX40 either alone (second-generation CARs) or in combination (third-generation CARs). Current, up-to-date published studies on anti-CD19 CAR therapy for acute lymphoblastic leukemia (ALL), chronic lymphocytic leukemia (CLL), and non-Hodgkin lymphoma (NHL) with observed side effects are discussed and an outlook on 58 ongoing trials is given. Clinical responses were achieved in up to 81% of ALL, 50% of CLL, and 40% of NHL patients. Factors with potential influence on the clinical outcome might be the design of the vector, the preconditioning regimen, and the number and quality of transfused CAR TCs. The applicability of clinical CAR TC therapy might include relapse after allogeneic stem cell transplantation (alloSCT), and ineligibility for or "bridging" until alloSCT. In summary, CAR therapy represents a highly promising treatment option even in heavily pretreated patients.
嵌合抗原受体(CAR)转导的T细胞(TC)新型疗法为复发或难治性CD19阳性白血病或淋巴瘤患者带来了新希望,即便在接受干细胞治疗后亦是如此。本综述聚焦于识别B细胞抗原CD19的CAR。逆转录病毒载体和慢病毒载体均被使用,它们编码包含共刺激分子(如CD28、CD137/4-1BB和OX40)的各种抗CD19 CAR构建体,这些共刺激分子可单独使用(第二代CAR)或联合使用(第三代CAR)。本文讨论了目前已发表的关于抗CD19 CAR疗法治疗急性淋巴细胞白血病(ALL)、慢性淋巴细胞白血病(CLL)和非霍奇金淋巴瘤(NHL)以及观察到的副作用的最新研究,并对58项正在进行的试验进行了展望。高达81%的ALL患者、50%的CLL患者和40%的NHL患者实现了临床缓解。可能对临床结果产生潜在影响的因素包括载体设计、预处理方案以及输注的CAR TC数量和质量。临床CAR TC疗法的适用情况可能包括异基因干细胞移植(alloSCT)后复发,以及不适合alloSCT或在alloSCT之前的“桥接”治疗。总之即便对于经过大量预处理的患者,CAR疗法仍是一个极具前景的治疗选择。