Department of Obstetrics, Gynecology, and Reproductive Sciences, Yale University School of Medicine, New Haven, CT, 06510, USA.
Department of Pediatrics, Emory University School of Medicine, Atlanta, GA, 30322, USA.
Sci Rep. 2021 Apr 13;11(1):8042. doi: 10.1038/s41598-021-87325-5.
Poly ADP-ribose polymerase (PARP) inhibitors are promising targeted therapy for epithelial ovarian cancer (EOC) with BRCA mutations or defective homologous recombination (HR) repair. However, reversion of BRCA mutation and restoration of HR repair in EOC lead to PARP inhibitor resistance and reduced clinical efficacy of PARP inhibitors. We have previously shown that triapine, a small molecule inhibitor of ribonucleotide reductase (RNR), impaired HR repair and sensitized HR repair-proficient EOC to PARP inhibitors. In this study, we performed in silico screening of small molecule libraries to identify novel compounds that bind to the triapine-binding pocket on the R2 subunit of RNR and inhibit RNR in EOC cells. Following experimental validation of selected top-ranking in silico hits for inhibition of dNTP and DNA synthesis, we identified, DB4, a putative RNR pocket-binding inhibitor markedly abrogated HR repair and sensitized BRCA-wild-type EOC cells to the PARP inhibitor olaparib. Furthermore, we demonstrated that the combination of DB4 and olaparib deterred the progression of BRCA-wild type EOC xenografts and significantly prolonged the survival time of tumor-bearing mice. Herein we report the discovery of a putative small molecule inhibitor of RNR and HR repair for combination with PARP inhibitors to treat PARP inhibitor-resistant and HR repair-proficient EOC.
聚 ADP-核糖聚合酶 (PARP) 抑制剂是一种有前途的靶向治疗方法,可用于治疗具有 BRCA 突变或同源重组 (HR) 修复缺陷的上皮性卵巢癌 (EOC)。然而,EOC 中 BRCA 突变的逆转和 HR 修复的恢复导致了 PARP 抑制剂耐药和 PARP 抑制剂临床疗效的降低。我们之前曾表明,三嗪,一种核糖核苷酸还原酶 (RNR) 的小分子抑制剂,可损害 HR 修复,并使 HR 修复功能正常的 EOC 对 PARP 抑制剂敏感。在这项研究中,我们对小分子文库进行了计算机筛选,以鉴定出能与 RNR 的 R2 亚基上的三嗪结合口袋结合并抑制 EOC 细胞中 RNR 的新型化合物。在对抑制 dNTP 和 DNA 合成的选定排名靠前的计算机命中进行实验验证后,我们确定了 DB4,一种假定的 RNR 口袋结合抑制剂,可显著阻断 HR 修复,并使 BRCA 野生型 EOC 细胞对 PARP 抑制剂奥拉帕利敏感。此外,我们证明 DB4 和奥拉帕利的联合使用阻止了 BRCA 野生型 EOC 异种移植物的进展,并显著延长了荷瘤小鼠的存活时间。在此,我们报告了一种 RNR 和 HR 修复的假定小分子抑制剂的发现,可与 PARP 抑制剂联合用于治疗 PARP 抑制剂耐药和 HR 修复功能正常的 EOC。