Hu Weilei, Wang Guosheng, Yarmus Lonny B, Wan Yuan
Institute of Translational Medicine, Zhejiang University, Hangzhou 310029, China.
Center for Disease Prevention Research and Department of Pharmacology & Toxicology, Medical College of Wisconsin, Milwaukee, WI 53226, USA.
Cancers (Basel). 2020 Sep 3;12(9):2496. doi: 10.3390/cancers12092496.
Immune checkpoint inhibitors (ICIs) targeting PD-1/PD-L1 have demonstrated remarkable treatment efficacy in advanced non-small cell lung cancer (NSCLC). However, low expression of programmed death-ligand 1 (PD-L1), epidermal growth factor receptor (EGFR) wild-type NSCLCs are refractory, and only few therapeutic options exist. Currently, combination therapy with ICIs is frequently used in order to enhance the treatment response rates. Yet, this regimen is still associated with poor treatment outcome. Therefore, identification of potential therapeutic targets for this subgroup of NSCLC is strongly desired. Here, we report the distinct methylation signatures of this special subgroup. Moreover, several druggable targets and relevant drugs for targeted therapy were incidentally identified. We found hypermethylated differentially methylated regions (DMRs) in three regions (TSS200, TSS1500, and gene body) are significantly higher than hypomethylated ones. Downregulated methylated genes were found to be involved in negative regulation of immune response and T cell-mediated immunity. Moreover, expression of four methylated genes (PLCXD3 (Phosphatidylinositol-Specific Phospholipase C, X Domain Containing 3), BAIAP2L2 (BAR/IMD Domain Containing Adaptor Protein 2 Like 2), NPR3 (Natriuretic Peptide Receptor 3), SNX10 (Sorting Nexin 10)) can influence patients' prognosis. Subsequently, based on DrugBank data, NetworkAnalyst 3.0 was used for protein-drug interaction analysis of up-regulated differentially methylated genes. Protein products of nine genes were identified as potential druggable targets, of which the tumorigenic potential of XDH (Xanthine Dehydrogenase), ATIC (5-Aminoimidazole-4-Carboxamide Ribonucleotide Formyltransferase/IMP Cyclohydrolase), CA9 (Carbonic Anhydrase 9), SLC7A11 (Solute Carrier Family 7 Member 11), and GAPDH (Glyceraldehyde-3-Phosphate Dehydrogenase) have been demonstrated in previous studies. Next, molecular docking and molecular dynamics simulation were performed to verify the structural basis of the therapeutic targets. It is noteworthy that the identified pemetrexed targeting ATIC has been recently approved for first-line use in combination with anti-PD1 inhibitors against lung cancer, irrespective of PD-L1 expression. In future work, a pivotal clinical study will be initiated to further validate our findings.
靶向程序性死亡蛋白 1(PD-1)/程序性死亡配体 1(PD-L1)的免疫检查点抑制剂(ICIs)已在晚期非小细胞肺癌(NSCLC)中显示出显著的治疗效果。然而,程序性死亡配体 1(PD-L1)低表达、表皮生长因子受体(EGFR)野生型的 NSCLC 患者难治,且治疗选择有限。目前,为提高治疗反应率,常采用 ICIs 联合治疗。然而,该方案的治疗效果仍然不佳。因此,迫切需要为这一 NSCLC 亚组确定潜在的治疗靶点。在此,我们报告了这一特殊亚组独特的甲基化特征。此外,还意外发现了几个可成药靶点及相关的靶向治疗药物。我们发现三个区域(转录起始位点上游 200bp(TSS200)、转录起始位点上游 1500bp(TSS1500)和基因体)的高甲基化差异甲基化区域(DMRs)显著多于低甲基化区域。发现甲基化下调的基因参与免疫反应的负调控和 T 细胞介导的免疫。此外,四个甲基化基因(磷脂酰肌醇特异性磷脂酶 C、X 结构域包含 3(PLCXD(_3))、含 BAR/IMD 结构域的衔接蛋白 2 样蛋白 2(BAIAP2L2)、利钠肽受体 3(NPR3)、分选连接蛋白 10(SNX10))的表达可影响患者预后。随后,基于药物银行(DrugBank)数据,使用 NetworkAnalyst 3.0 对上调的差异甲基化基因进行蛋白-药物相互作用分析。九个基因的蛋白产物被鉴定为潜在的可成药靶点,其中黄嘌呤脱氢酶(XDH)、5-氨基咪唑-4-甲酰胺核苷酸甲酰基转移酶/肌苷酸环化水解酶(ATIC)、碳酸酐酶 9(CA9)、溶质载体家族 7 成员 11(SLC7A11)和甘油醛-3-磷酸脱氢酶(GAPDH)的致瘤潜力在先前的研究中已得到证实。接下来,进行分子对接和分子动力学模拟以验证治疗靶点的结构基础。值得注意的是,已证实靶向 ATIC 的培美曲塞最近被批准与抗 PD-1 抑制剂联合用于肺癌一线治疗,无论 PD-L1 表达情况如何。在未来的工作中,将启动一项关键的临床研究以进一步验证我们的发现。