基因修饰 T 细胞免疫疗法:限制副作用带来新挑战。

Immunotherapy with gene-modified T cells: limiting side effects provides new challenges.

机构信息

Department of Immunology, Institute of Immunity and Transplantation, Royal Free Hospital, University College London, London, UK.

出版信息

Gene Ther. 2013 Nov;20(11):1029-32. doi: 10.1038/gt.2013.34. Epub 2013 Jun 27.

Abstract

Genetic tools have been developed to efficiently engineer T-cell specificity and enhance T-cell function. Chimeric antigen receptors (CAR) use the antibody variable segments to direct specificity against cell surface molecules. T-cell receptors (TCR) can redirect T cells to intracellular target proteins, fragments of which are presented in the peptide-binding groove of HLA molecules. A recent clinical trial with CAR-modified T cells redirected against the B-cell lineage antigen CD19 showed dramatic clinical benefit in chronic lymphocytic leukaemia patients. Similarly, impressive clinical responses were seen in melanoma and synovial cell carcinoma with TCR-modified T cells redirected against the melanocyte lineage antigen MART-1 and the testis-cancer antigen NY-ESO-1. However, on and off-target toxicity was associated with most of these clinical responses, and fatal complications have been observed in some patients treated with gene modified T cells. This review will discuss factors that might contribute to toxic side effects of therapy with gene modified T cells, and outline potential strategies to retain anticancer activity while reducing unwanted side effects.

摘要

已经开发出遗传工具来有效地设计 T 细胞特异性并增强 T 细胞功能。嵌合抗原受体 (CAR) 使用抗体可变片段来针对细胞表面分子进行特异性导向。T 细胞受体 (TCR) 可以将 T 细胞重新定向到细胞内靶蛋白,其片段呈现在 HLA 分子的肽结合槽中。最近的一项临床试验表明,CAR 修饰的 T 细胞针对 B 细胞谱系抗原 CD19 的重新定向在慢性淋巴细胞白血病患者中显示出显著的临床获益。同样,针对黑素细胞谱系抗原 MART-1 和睾丸癌抗原 NY-ESO-1 的 TCR 修饰的 T 细胞在黑色素瘤和滑膜细胞癌中也观察到了令人印象深刻的临床反应。然而,大多数这些临床反应都伴随着脱靶和靶外毒性,并且在一些接受基因修饰 T 细胞治疗的患者中观察到了致命的并发症。这篇综述将讨论可能导致基因修饰 T 细胞治疗产生毒性副作用的因素,并概述保留抗癌活性同时减少不良反应的潜在策略。

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