School of Pharmacy, Key Laboratory of Molecular Pharmacology and Drug Evaluation (Yantai University), Ministry of Education, China; College of Life Sciences and Medicine, Zhejiang Sci-Tech University, Hangzhou 310018; Collaborative Innovation Center of Advanced Drug Delivery System and Biotech Drugs in Universities of Shandong, Yantai University, Yantai 264005, China.
University of Chinese Academy of Sciences, 19 Yuquan Road, Beijing 100049, China; Drug Discovery and Design Center, State Key Laboratory of Drug Research, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Shanghai 201203, China.
Pharmacol Ther. 2019 Dec;204:107406. doi: 10.1016/j.pharmthera.2019.107406. Epub 2019 Sep 12.
Epigenetics has emerged as an extremely exciting fast-growing area of biomedical research in post genome era. Epigenetic dysfunction is tightly related with various diseases such as cancer and aging related degeneration, potentiating epigenetics modulators as important therapeutics targets. Indeed, inhibitors of histone deacetylase and DNA methyltransferase have been approved for treating blood tumor malignancies, whereas inhibitors of histone methyltransferase and histone acetyl-lysine recognizer bromodomain are in clinical stage. However, it remains a great challenge to discover potent and selective inhibitors by targeting catalytic site, as the same subfamily of epigenetic enzymes often share high sequence identity and very conserved catalytic core pocket. It is well known that epigenetic modifications are usually carried out by multi-protein complexes, and activation of catalytic subunit is often tightly regulated by other interactive protein component, especially in disease conditions. Therefore, it is not unusual that epigenetic complex machinery may exhibit allosteric regulation site induced by protein-protein interactions. Targeting allosteric site emerges as a compelling alternative strategy to develop epigenetic drugs with enhanced druggability and pharmacological profiles. In this review, we highlight recent progress in the development of allosteric inhibitors for epigenetic complexes through targeting protein-protein interactions. We also summarized the status of clinical applications of those inhibitors. Finally, we provide perspectives of future novel allosteric epigenetic machinery modulators emerging from otherwise undruggable single protein target.
表观遗传学是后基因组时代生物医学研究中一个令人兴奋的快速发展领域。表观遗传功能障碍与癌症和与衰老相关的退行性等各种疾病密切相关,使表观遗传调节剂成为重要的治疗靶点。事实上,组蛋白去乙酰化酶和 DNA 甲基转移酶抑制剂已被批准用于治疗血液肿瘤恶性肿瘤,而组蛋白甲基转移酶和组蛋白乙酰-赖氨酸识别溴结构域抑制剂处于临床阶段。然而,通过靶向催化位点发现有效和选择性抑制剂仍然是一个巨大的挑战,因为表观遗传酶的相同亚家族通常具有高度的序列同一性和非常保守的催化核心口袋。众所周知,表观遗传修饰通常由多蛋白复合物进行,催化亚基的激活通常受到其他相互作用蛋白成分的严格调节,尤其是在疾病状态下。因此,表观遗传复合物机械可能表现出由蛋白-蛋白相互作用诱导的别构调节位点并不罕见。靶向别构位点成为开发具有增强药物可及性和药理学特性的表观遗传药物的一种有吸引力的替代策略。在这篇综述中,我们强调了通过靶向蛋白-蛋白相互作用开发表观遗传复合物的别构抑制剂的最新进展。我们还总结了这些抑制剂的临床应用现状。最后,我们提供了从原本不可成药的单个蛋白靶标中出现的新型别构表观遗传机械调节剂的未来前景。