Rizzo Angela, Iachettini Sara, Zizza Pasquale, Cingolani Chiara, Porru Manuela, Artuso Simona, Stevens Malcolm, Hummersone Marc, Biroccio Annamaria, Salvati Erica, Leonetti Carlo
Experimental Chemotherapy Laboratory, Regina Elena National Cancer Institute, via delle Messi d'Oro 156, 00158, Rome, Italy.
School of Pharmacy, University of Nottingham, Nottingham, NG7 2RD, UK.
J Exp Clin Cancer Res. 2014 Oct 6;33(1):81. doi: 10.1186/s13046-014-0081-x.
The pentacyclic acridinium salt RHPS4 (3,11-difluoro-6,8,13-trimethyl-8H-quino [4,3,2-kl] acridinium methosulfate, compound 1) is one of the most interesting DNA G-quadruplex binding molecules due to its high efficacy in tumor cell growth inhibition both in in vitro models and in vivo against human tumor xenografts in combination with conventional chemotherapeutics. Despite compound 1 having desirable chemical and pharmaceutical properties, its potential as a therapeutic agent is compromised by off-target effects on cardiovascular physiology. In this paper we report a new series of structurally-related compounds which were developed in an attempt to minimize its off-target profile, but maintaining the same favorable chemical and pharmacological features of the lead compound. By performing a comparative analysis it was possible to identify which derivatives had the following properties: (i) to show a reduced capacity in respect to compound 1 to inhibit the hERG tail current tested in a patch clamp assay and/or to interact with the human recombinant β2 receptor; (ii) to maintain both a good G4-binding affinity and cancer cell selectivity; and (iii) to trigger DNA damage with specific telomere uncapping. These studies allowed us to identify a novel G4-stabilizing molecule, compound 8, being characterized by reduced off-target effects and potent telomere on-target properties compared to the prototypic compound 1. Moreover, compound 8 shares with compound 1 the same molecular mode of action and an anti-tumour activity specifically restricted to replicating cells, as evident with its particularly efficient activity in combination therapy with a topoisomerase I inhibitor. In conclusion, we have identified a new pentacyclic derivative 8 having suitable properties to be the focus of further investigations as a clinical candidate for cancer therapy.
五环吖啶鎓盐RHPS4(3,11 - 二氟 - 6,8,13 - 三甲基 - 8H - 喹啉并[4,3,2 - kl]吖啶鎓甲磺酸盐,化合物1)是最有趣的DNA G - 四链体结合分子之一,因为它在体外模型和体内对人肿瘤异种移植瘤与传统化疗药物联合使用时,对肿瘤细胞生长具有高效抑制作用。尽管化合物1具有理想的化学和药物性质,但其作为治疗剂的潜力因对心血管生理的脱靶效应而受到损害。在本文中,我们报道了一系列新的结构相关化合物,这些化合物是为了尽量减少其脱靶特性而开发的,同时保持先导化合物相同的良好化学和药理特性。通过进行比较分析,可以确定哪些衍生物具有以下性质:(i)与化合物1相比,在膜片钳试验中抑制hERG尾电流和/或与人重组β2受体相互作用的能力降低;(ii)保持良好的G4结合亲和力和癌细胞选择性;(iii)通过特异性端粒解盖引发DNA损伤。这些研究使我们能够鉴定出一种新型的G4稳定分子,化合物8,与原型化合物1相比,其脱靶效应降低,端粒靶向特性更强。此外,化合物8与化合物1具有相同的分子作用模式,并且抗肿瘤活性特别限于复制细胞,这在其与拓扑异构酶I抑制剂联合治疗中的特别有效活性中很明显。总之,我们已经鉴定出一种新的五环衍生物8,它具有合适的性质,可作为癌症治疗临床候选药物进行进一步研究。