Liu T-C, Wang Y, Hallden G, Brooks G, Francis J, Lemoine N R, Kirn D
Viral and Genetic Therapy Program, Cancer Research UK Molecular Oncology Unit, Barts & The London School of Medicine and Imperial College Faculty of Medicine, Hammersmith Hospital, London, UK.
Gene Ther. 2005 Sep;12(17):1333-46. doi: 10.1038/sj.gt.3302555.
Replication-selective oncolytic adenoviruses hold promise, but novel mechanisms must be identified to maximize intratumoral virus persistence, spread and therapeutic transgene-carrying capacity while maintaining safety. One of the main approaches to engineering cancer-selectivity has been to delete a viral gene that is theoretically expendable in cancer cells. Results with this approach have been mixed, however, as evidenced by controversy over Onyx-015 (E1B-55kD(-)) selectivity. We hypothesized that the functional redundancy between viral gene products might limit selectivity and/or potency with this approach. Antiviral immune inducers of apoptosis (eg TNF-alpha) have not been thoroughly investigated in previous studies. We therefore explored whether deletion of functionally redundant viral genes, E1B-19kD and E3B, both independently antagonize TNF-alpha, could lead to enhanced oncolytic potency while maintaining selectivity. Since tumors have numerous blocks in apoptotic pathways, we hypothesized that deletion of one or both gene regions would result in cancer-selectivity in the presence of TNF-alpha. We have previously shown that the E1B-19kD deletion resulted in enhanced viral spread in vitro and in immunocompetent tumor models in vivo. In contrast, the impact of E3B deletion, especially its in vitro selectivity and potency, was not thoroughly characterized, although it resulted in rapid immune-mediated viral clearance in vivo. Furthermore, previous publications indicated that double-deleted mutants have selectivity but unsatisfactory efficacy. We compared the selectivity and potency of E1B-19kD(-), E3B(-) and E1B-19kD(-)/E3B(-) mutants to wild-type adenovirus. In cancer cells, the E1B-19kD(-) mutant had superior replication, spread and cytolysis (+) or (-) TNF-alpha; deletion of both E1B-19kD and E3B was relatively deleterious. In normal cells without TNF-alpha, similar results were obtained. In contrast, all three mutants were significantly inhibited in the presence of TNF-alpha. In immunocompetent mice, all three mutants were significantly inhibited in normal tissue. In tumors, only the E1B-19kD(-) mutant demonstrated enhanced replication, spread and antitumoral efficacy. Therefore, E1B-19kD deletion and E3B retention should be incorporated in oncolytic adenoviruses for enhanced safety and efficacy. In addition, functional redundant viral genes and their biological mediators/targets need to be carefully examined for the next generation of gene-deleted oncolytic viruses.
复制选择性溶瘤腺病毒具有应用前景,但必须确定新机制,以在保持安全性的同时,最大限度地提高肿瘤内病毒的持久性、传播能力和携带治疗性转基因的能力。构建癌症选择性的主要方法之一是删除一个理论上在癌细胞中可缺失的病毒基因。然而,这种方法的结果好坏参半,如安柯瑞(E1B - 55kD(-))的选择性存在争议就证明了这一点。我们推测病毒基因产物之间的功能冗余可能会限制这种方法的选择性和/或效力。以往研究尚未对凋亡的抗病毒免疫诱导剂(如肿瘤坏死因子-α)进行深入研究。因此,我们探讨了删除功能冗余的病毒基因E1B - 19kD和E3B(二者均能独立拮抗肿瘤坏死因子-α)是否能在保持选择性的同时提高溶瘤效力。由于肿瘤在凋亡途径中有众多阻碍,我们推测删除一个或两个基因区域会在肿瘤坏死因子-α存在的情况下产生癌症选择性。我们之前已表明,E1B - 19kD缺失会导致病毒在体外以及体内免疫健全的肿瘤模型中传播增强。相比之下,E3B缺失的影响,尤其是其体外选择性和效力,尚未得到充分表征,尽管它会导致体内免疫介导的病毒快速清除。此外,以往出版物表明双缺失突变体具有选择性,但疗效不尽人意。我们比较了E1B - 19kD(-)、E3B(-)和E1B - 19kD(-)/E3B(-)突变体与野生型腺病毒的选择性和效力。在癌细胞中,E1B - 19kD(-)突变体具有更好的复制、传播和细胞溶解能力(无论有无肿瘤坏死因子-α);E1B - 19kD和E3B二者均缺失相对有害。在无肿瘤坏死因子-α的正常细胞中,也得到了类似结果。相比之下,在肿瘤坏死因子-α存在的情况下,所有三种突变体均受到显著抑制。在免疫健全的小鼠中,所有三种突变体在正常组织中均受到显著抑制。在肿瘤中,只有E1B - 19kD(-)突变体表现出复制、传播增强以及抗肿瘤疗效增强。因此,应将E1B - 19kD缺失和E3B保留纳入溶瘤腺病毒中,以提高安全性和疗效。此外,对于下一代基因缺失的溶瘤病毒,需要仔细研究功能冗余的病毒基因及其生物学介质/靶点。