Abdelfatah Eihab, Kerner Zachary, Nanda Nainika, Ahuja Nita
Department of Surgery, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Therap Adv Gastroenterol. 2016 Jul;9(4):560-79. doi: 10.1177/1756283X16644247. Epub 2016 May 1.
Epigenetics is a relatively recent field of molecular biology that has arisen over the past 25 years. Cancer is now understood to be a disease of widespread epigenetic dysregulation that interacts extensively with underlying genetic mutations. The development of drugs targeting these processes has rapidly progressed; with several drugs already FDA approved as first-line therapy in hematological malignancies. Gastrointestinal (GI) cancers possess high degrees of epigenetic dysregulation, exemplified by subtypes such as CpG island methylator phenotype (CIMP), and the potential benefit of epigenetic therapy in these cancers is evident. The application of epigenetic drugs in solid tumors, including GI cancers, is just emerging, with increased understanding of the cancer epigenome. In this review, we provide a brief overview of cancer epigenetics and the epigenetic targets of therapy including deoxyribonucleic acid (DNA) methylation, histone modifications, and chromatin remodeling. We discuss the epigenetic drugs currently in use, with a focus on DNA methyltransferase (DNMT) and histone deacetylase (HDAC) inhibitors, and explain the pharmacokinetic and mechanistic challenges in their application. We present the strategies employed in incorporating these drugs into the treatment of GI cancers, and explain the concept of the cancer stem cell in epigenetic reprogramming and reversal of chemo resistance. We discuss the most promising combination strategies in GI cancers including: (1) epigenetic sensitization to radiotherapy, (2) epigenetic sensitization to cytotoxic chemotherapy, and (3) epigenetic immune modulation and priming for immune therapy. Finally, we present preclinical and clinical trial data employing these strategies thus far in various GI cancers including colorectal, esophageal, gastric, and pancreatic cancer.
表观遗传学是分子生物学中一个相对较新的领域,在过去25年中兴起。现在人们认识到癌症是一种广泛的表观遗传失调疾病,与潜在的基因突变广泛相互作用。针对这些过程的药物开发进展迅速;已有几种药物获得美国食品药品监督管理局(FDA)批准,作为血液系统恶性肿瘤的一线治疗药物。胃肠道(GI)癌症存在高度的表观遗传失调,如CpG岛甲基化表型(CIMP)等亚型就是例证,表观遗传疗法在这些癌症中的潜在益处是显而易见的。随着对癌症表观基因组的认识不断增加,表观遗传药物在实体瘤(包括胃肠道癌症)中的应用才刚刚起步。在这篇综述中,我们简要概述了癌症表观遗传学以及治疗的表观遗传靶点,包括脱氧核糖核酸(DNA)甲基化、组蛋白修饰和染色质重塑。我们讨论了目前正在使用的表观遗传药物,重点是DNA甲基转移酶(DNMT)和组蛋白去乙酰化酶(HDAC)抑制剂,并解释了它们应用中的药代动力学和机制挑战。我们介绍了将这些药物纳入胃肠道癌症治疗的策略,并解释了癌症干细胞在表观遗传重编程和化疗耐药逆转中的概念。我们讨论了胃肠道癌症中最有前景的联合策略,包括:(1)对放疗的表观遗传致敏,(2)对细胞毒性化疗的表观遗传致敏,以及(3)表观遗传免疫调节和免疫治疗的启动。最后,我们展示了迄今为止在各种胃肠道癌症(包括结直肠癌、食管癌、胃癌和胰腺癌)中采用这些策略的临床前和临床试验数据。