Exploring the landscape of post-translational modification in drug discovery

Pharmacol Ther. 2025 Jan:265:108749. doi: 10.1016/j.pharmthera.2024.108749. Epub 2024 Nov 16.

Abstract

Post-translational modifications (PTMs) play a crucial role in regulating protein function, and their dysregulation is frequently associated with various diseases. The emergence of epigenetic drugs targeting factors such as histone deacetylases (HDACs) and histone methyltransferase enhancers of zeste homolog 2 (EZH2) has led to a significant shift towards precision medicine, offering new possibilities to overcome the limitations of traditional therapeutics. In this review, we aim to systematically explore how small molecules modulate PTMs. We discuss the direct targeting of enzymes involved in PTM pathways, the modulation of substrate proteins, and the disruption of protein-enzyme interactions that govern PTM processes. Additionally, we delve into the emerging strategy of employing multifunctional molecules to precisely regulate the modification levels of proteins of interest (POIs). Furthermore, we examine the specific characteristics of these molecules, evaluating their therapeutic benefits and potential drawbacks. The goal of this review is to provide a comprehensive understanding of PTM-targeting strategies and their potential for personalized medicine, offering a forward-looking perspective on the evolution of precision therapeutics.

Keywords: Cancer; Inhibitors; Innovative drugs; Multifunctional molecules; Post-translational modifications; Precision medicine; Proteomics; Rational drug design.

Publication types

  • Review

MeSH terms

  • Animals
  • Drug Discovery* / methods
  • Epigenesis, Genetic
  • Histone Deacetylases / metabolism
  • Humans
  • Precision Medicine / methods
  • Protein Processing, Post-Translational*

Substances

  • Histone Deacetylases