Tang Meiyue, Gong Ming, Liu Xiaoliang, Zhang Tiantian, Liu Zhenghua, Song Dandan
Department of Oncology, Shengjing Hospital of China Medical University, Shenyang, 110004, China.
Department of Radiation Oncology, The First Hospital of China Medical University, Shenyang, 110001, China.
Eur J Med Chem. 2025 Dec 5;299:118106. doi: 10.1016/j.ejmech.2025.118106. Epub 2025 Aug 28.
Enhancer of zeste homolog 2 (EZH2), the catalytic subunit of the Polycomb Repressive Complex 2 (PRC2), plays a pivotal role in epigenetic regulation by catalyzing the trimethylation of histone H3 at lysine 27 (H3K27me3), leading to transcriptional repression of target genes. Dysregulation of EZH2 has been implicated in various cancers, including lymphomas, prostate, and breast cancers, by promoting oncogenic transformation and tumor progression. Targeting EZH2 has been regarded as a promising strategy for cancer therapy. Over the past decade, significant progress has been made in the development of EZH2 inhibitors and degraders, several of which have been approved or are undergoing various clinical trials. These agents function by competing with the cofactor S-adenosyl-l-methionine (SAM) at the active site within the SET domain of EZH2, thereby preventing the methylation of H3K27 and reactivating silenced tumor suppressor genes. In addition, next-generation strategies, including dual EZH1/EZH2 inhibitors, PROTAC-based degraders, and combination regimens with immunotherapy or hormonal therapy, are being actively explored to enhance therapeutic benefit and overcome resistance mechanisms that limit monotherapy efficacy. This review provides an overview of the current landscape of EZH2-targeted therapies since 2020 and future directions for optimizing their application.
zeste 同源物 2 增强子(EZH2)是多梳抑制复合物 2(PRC2)的催化亚基,通过催化组蛋白 H3 赖氨酸 27 位点的三甲基化(H3K27me3)在表观遗传调控中起关键作用,导致靶基因的转录抑制。EZH2 的失调与包括淋巴瘤、前列腺癌和乳腺癌在内的多种癌症有关,它通过促进致癌转化和肿瘤进展发挥作用。靶向 EZH2 被认为是一种有前景的癌症治疗策略。在过去十年中,EZH2 抑制剂和降解剂的开发取得了重大进展,其中几种已获批准或正在进行各种临床试验。这些药物通过在 EZH2 的 SET 结构域内的活性位点与辅因子 S-腺苷-L-甲硫氨酸(SAM)竞争发挥作用,从而阻止 H3K27 的甲基化并重新激活沉默的肿瘤抑制基因。此外,包括双 EZH1/EZH2 抑制剂、基于 PROTAC 的降解剂以及与免疫疗法或激素疗法的联合方案在内的下一代策略正在积极探索中,以提高治疗效果并克服限制单一疗法疗效的耐药机制。本综述概述了自 2020 年以来 EZH2 靶向治疗的现状以及优化其应用的未来方向。