Department of Lymphoma and Myeloma, University of Texas M. D. Anderson Cancer Center, Houston, TX, USA.
Adv Exp Med Biol. 2020;1244:215-233. doi: 10.1007/978-3-030-41008-7_10.
CAR-T (chimeric antigens receptor-T) cell therapy is a breakthrough therapy of the twenty-first century for the management of different malignancies including lymphomas and leukemias. Numeral trials are underway to understand the optimal CAR-T cell design and dose to maximize efficacy and mitigate toxicity. Currently two CAR-T cell therapy products, axicabtagene ciloleucel and tisagenlecleucel, are approved by the US Food and Drug Administration, which have shown excellent responses in otherwise poor prognostic lymphomas and leukemias. The favorable outcomes achieved of this therapy were noted to be durable during long-term follow-up. Understanding the challenges associated with manufacturing and the reasons for T cell failure including poor T cell expansion, persistence, and tumor resistance are critical for its wide-scale application in order to attain the full potential of this novel therapy. Here we review the salient features of the different CAR-T products and discuss the pivotal trials that led to its approval.
嵌合抗原受体 T 细胞（CAR-T）疗法是 21 世纪治疗不同恶性肿瘤（包括淋巴瘤和白血病）的突破性疗法。目前正在进行大量试验以了解最佳的 CAR-T 细胞设计和剂量，以最大程度地提高疗效并减轻毒性。目前，两种 CAR-T 细胞疗法产品，axicabtagene ciloleucel 和 tisagenlecleucel，已获得美国食品和药物管理局（FDA）的批准，它们在其他预后不良的淋巴瘤和白血病中显示出了极好的疗效。在长期随访中，观察到这种疗法的良好效果具有持久性。为了广泛应用这种疗法以充分发挥其潜力，了解与制造相关的挑战以及 T 细胞衰竭的原因（包括 T 细胞扩增、持久性和肿瘤耐药性差）至关重要。在这里，我们回顾了不同 CAR-T 产品的显著特征，并讨论了导致其批准的关键试验。
