Current Pharmacology Reports

, Volume 3, Issue 5, pp 268–285 | Cite as

Novel Drugs Targeting the Epigenome

  • Zhuo Chen
  • Honglin LiEmail author
Epigenetics ( ATY Lau, Section Editor)
Part of the following topical collections:
  1. Topical Collection on Epigenetics


Epigenetic drug discovery has its beginning in the cancer research arena, focusing first on DNA methylation and histone deacetylation. There are currently two DNA methyltransferase (DNMT) inhibitors and four histone deacetylase (HDAC) inhibitors approved by the US Food and Drug Administration (FDA) during the past 13 years. Over the past few years, breakthrough discoveries of chromatin-modifying enzymes and associated mechanisms have exploded, providing new insights into the role of epigenetic control in gene regulation and leading to the discovery of a variety of new and specific drug targets. Among them, epigenetic “reader”—bromodomain and extra-terminal protein (BET), “writers”—disruptor of telomeric silencing 1-like (DOT1L), enhancer of zeste homolog 2 (EZH2), and protein arginine methyltransferase 5 (PRMT5), and “erasers”—lysine-specific histone demethylase 1 (LSD1) as well as isocitrate dehydrogenase (IDH) attract greater attention due to the ongoing clinical trials. This article provides a brief overview of new drugs modulating the above epigenetic targets, including their indication, mechanism of action, and disclosed chemical structures. The trend of epigenetic drug approval in the following few years is expectable, at least partially, from current clinical trials summarized in this review.


Epigenetic Clinical trials Inhibitors Cancer Cardiovascular 



Angioimmunoblastic T-cell lymphoma


Anaplastic large cell lymphoma


Acute myeloid leukemia


Atypical teratoid rhabdoid tumor


Bromodomain and extra-terminal protein


Bristol-Myers Squibb




Coronary artery disease


Castration-resistant prostate cancer


Cardiovascular disease


Diffuse large B-cell lymphoma


Diabetes mellitus


DNA methyltransferase


Disruptor of telomeric silencing 1-like


Estrogen receptor


Enhancer of zeste homolog 2


Food and Drug Administration




Histone deacetylase


Histone methyltransferase


Isocitrate dehydrogenase


Lysine-specific histone demethylase 1


Mantle cell lymphoma


Myelodysplastic syndrome


Multiple myeloma


Malignant rhabdoid tumor


Non-Hodgkin lymphoma


Non-small cell lung cancer


NUT midline carcinoma


Protein arginine methyltransferase


Rhabdoid tumors of the kidney


Small cell lung cancer


Triple-negative breast cancer



This work was supported by the National Key R&D Program of China (grant 2016YFA0502304) and the National Natural Science Foundation of China (grants 81230076).

Compliance with Ethical Standards

Conflict of Interest

The authors indicate no conflict of interest with the subject matter of this review.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the authors.


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Copyright information

© Springer International Publishing AG 2017

Authors and Affiliations

  1. 1.State Key Laboratory of Bioreactor Engineering, Shanghai Key Laboratory of New Drug Design, School of PharmacyEast China University of Science and TechnologyShanghaiChina

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