Vickram Sundaram, Infant Shofia Saghya, Sivasubramanian Manikandan, Anbalagan Saravanan, Marimuthu Mathan Muthu Chinnakannu, Chopra Hitesh
Department of Biotechnology, Saveetha School of Engineering, Saveetha Institute of Medical and Technical Sciences, Chennai, 602105, India.
Centre for Research Impact & Outcome, Chitkara College of Pharmacy, Chitkara University, Rajpura, 140401, India.
Pharmacol Rep. 2025 Sep 2. doi: 10.1007/s43440-025-00770-1.
Epigenetic modulation has emerged as a central strategy that can change the fate of tumour cells to offer more rational and precise approaches by modulating reversible changes in chromatin structure, regulating gene expression without altering DNA sequence. Many reports have indicated the contributions of abnormal epigenetic alterations, particularly DNA methylation and histone modification patterns, as well as their association with non-coding RNA interactions during cancer emergence, development or resistance to standard therapies. Ongoing studies on various inhibitors also demonstrate encouraging preclinical results and potent inhibitory activity. Furthermore, combining epigenetic medicines with conventional treatment approaches such as chemotherapy and radiotherapy is proven to improve therapeutic efficacy in resistant cases of various malignancies. This article also briefly reviews RNA modifications (epitranscriptomics, such as m6A and m5C), novel acetylation modifications, chromosomal interaction studies, and the role of AlphaFold. The present review further illustrates these translational challenges and future opportunities in epigenetic drug development, while shedding light on the necessity of developing predictive biomarkers capable of informing personalized therapies to reduce off-target effects. The ability to target epigenetic modulators has the potential to improve patient outcomes and increase treatment options when coupled with traditional guidelines, as evidenced by on-going clinical trials and FDA approvals.
表观遗传调控已成为一种核心策略,它可以通过调节染色质结构的可逆变化、在不改变DNA序列的情况下调控基因表达,从而改变肿瘤细胞的命运,提供更合理、精确的方法。许多报告指出了异常表观遗传改变的作用,特别是DNA甲基化和组蛋白修饰模式,以及它们在癌症发生、发展或对标准疗法的耐药性过程中与非编码RNA相互作用的关联。对各种抑制剂的持续研究也显示出令人鼓舞的临床前结果和强大的抑制活性。此外,将表观遗传药物与化疗和放疗等传统治疗方法相结合,已被证明可以提高各种恶性肿瘤耐药病例的治疗效果。本文还简要回顾了RNA修饰(表观转录组学,如m6A和m5C)、新型乙酰化修饰、染色体相互作用研究以及AlphaFold的作用。本综述进一步阐述了表观遗传药物开发中的这些转化挑战和未来机遇,同时阐明了开发能够指导个性化治疗以减少脱靶效应的预测性生物标志物的必要性。靶向表观遗传调节剂的能力有可能改善患者预后,并在与传统指南结合时增加治疗选择,正在进行的临床试验和FDA批准证明了这一点。