靶向MYC的抗肿瘤药物研究进展  

Research Progress of Antitumor Drugs Targeting MYC

作　　者：胡慧慧 田新磊 张小猛 袁胜涛[1] HU Huihui;TIAN Xinlei;ZHANG Xiaomeng;YUAN Shengtao(New Drug Screening and Pharmacodynamic Evaluation Center,State Key Laboratory of Natural Medicines,China Pharmaceutical University,Nanjing 210009,China;Nanjing Sanhome Pharmaceutical Co.,Ltd.,Nanjing 210038,China)

机构地区：[1]中国药科大学多靶标天然药物全国重点实验室新药筛选与药效评价中心,江苏南京210009 [2]南京圣和药业股份有限公司,江苏南京210038

出　　处：《药学进展》2025年第4期311-320,共10页Progress in Pharmaceutical Sciences

摘　　要：骨髓细胞瘤病毒癌基因同源物(myelocytomatosis viral oncogene homolog,MYC)是人类癌症中最常失调的驱动基因之一,其表达失调与多种肿瘤的发生和发展密切相关,其编码的MYC蛋白主要作为转录因子发挥作用,参与调节多种细胞过程,包括细胞生长、分裂、分化、代谢和凋亡,因此,靶向MYC蛋白是治疗肿瘤的潜在策略。然而,由于MYC缺乏合适的结合口袋与小分子化合物结合,MYC一直被认为是“不可成药”靶点。概述MYC的结构与功能,汇总分析现阶段正在研发的直接或间接靶向MYC的抗肿瘤药物,为靶向MYC的药物开发提供参考。The myelocytomatosis viral oncogene homolog(MYC)is one of the most frequently dysregulated driver genes in human cancers.The dysregulation of its expression is closely associated with the occurrence and progression of various tumors.The MYC protein encoded by it mainly functions as a transcription factor and is involved in regulating multiple cellular processes,including cell growth,division,differentiation,metabolism,and apoptosis.Therefore,targeting MYC protein is a potential strategy for tumor treatment.However,due to the lack of a suitable binding pocket in MYC for binding to small-molecule compounds,MYC has long been considered an“undruggable”target.This article outlines the structure and function of MYC,and comprehensively analyzes the currently developed antitumor drugs directly or indirectly targeting MYC,aiming to provide some reference for the development of drugs targeting MYC.

关 键 词：MYC 癌症 MYC相关因子X 临床应用 

分 类 号：R966[医药卫生—药理学] R979.1[医药卫生—药学]