Department of Oncology, University of Padova, Padova, Italy.
Cancer Res. 2009 Dec 15;69(24):9385-94. doi: 10.1158/0008-5472.CAN-09-0494.
T-cell receptor (TCR) gene transfer for cancer immunotherapy is limited by the availability of large numbers of tumor-specific T cells. TCR alpha and beta chains were isolated from a highly lytic HLA-A2-restricted cytotoxic T lymphocyte (CTL) clone recognizing the melanoma-associated Melan-A/MART-1 antigen and inserted into a lentiviral vector carrying a bidirectional promoter capable of robust and coordinated expression of the two transgenes. Lentiviral vector-based gene delivery systems have shown increased transfer efficiency and transgene expression compared with the widely used gamma-retroviral vectors. This vector performed more efficiently than a gamma-retrovirus-based vector containing the same expression cassette, resulting in a T-cell population with 60% to 80% of transgenic TCR expression with mainly CD8(+) intermediate effector phenotype. Transgenic T cells specifically produced cytokine in response to and killed antigen-expressing melanoma cells, retained an overlapping functional avidity in comparison with the TCR donor CTL clone, and exerted significant therapeutic effects in vivo upon adoptive transfer in melanoma-bearing severe combined immunodeficient mice. Optical imaging showed their accumulation in the tumor site. Overall, our results indicate that lentiviral vectors represent a valid tool for stable and high-intensity expression of transgenic TCR and support clinical exploitation of this approach for therapeutic application.
T 细胞受体(TCR)基因转移用于癌症免疫治疗受到大量肿瘤特异性 T 细胞的限制。TCRα和β链从一个高度溶瘤 HLA-A2 限制性细胞毒性 T 淋巴细胞(CTL)克隆中分离出来,该克隆识别黑色素瘤相关的 Melan-A/MART-1 抗原,并插入到携带双向启动子的慢病毒载体中,该启动子能够强大且协调地表达两个转基因。与广泛使用的γ逆转录病毒载体相比,基于慢病毒载体的基因传递系统显示出更高的转移效率和转基因表达。与包含相同表达盒的γ逆转录病毒载体相比,该载体的性能更好,导致转导 T 细胞群体中 60%至 80%的转基因 TCR 表达主要为 CD8(+)中间效应表型。转基因 T 细胞特异性地产生细胞因子以响应并杀伤表达抗原的黑色素瘤细胞,与 TCR 供体 CTL 克隆相比保持重叠的功能亲和力,并在荷黑色素瘤的严重联合免疫缺陷小鼠中进行过继转移后发挥显著的治疗效果。光学成像显示它们在肿瘤部位聚集。总体而言,我们的结果表明,慢病毒载体代表了稳定和高强度表达转基因 TCR 的有效工具,并支持该方法的临床应用,用于治疗应用。