Induced protein degradation: an emerging drug discovery paradigm

Nat Rev Drug Discov. 2017 Feb;16(2):101-114. doi: 10.1038/nrd.2016.211. Epub 2016 Nov 25.

Abstract

Small-molecule drug discovery has traditionally focused on occupancy of a binding site that directly affects protein function, and this approach typically precludes targeting proteins that lack such amenable sites. Furthermore, high systemic drug exposures may be needed to maintain sufficient target inhibition in vivo, increasing the risk of undesirable off-target effects. Induced protein degradation is an alternative approach that is event-driven: upon drug binding, the target protein is tagged for elimination. Emerging technologies based on proteolysis-targeting chimaeras (PROTACs) that exploit cellular quality control machinery to selectively degrade target proteins are attracting considerable attention in the pharmaceutical industry owing to the advantages they could offer over traditional small-molecule strategies. These advantages include the potential to reduce systemic drug exposure, the ability to counteract increased target protein expression that often accompanies inhibition of protein function and the potential ability to target proteins that are not currently therapeutically tractable, such as transcription factors, scaffolding and regulatory proteins.

Publication types

  • Review

MeSH terms

  • Animals
  • Drug Discovery / methods*
  • High-Throughput Screening Assays
  • Humans
  • Mutant Chimeric Proteins
  • Protein Binding / drug effects
  • Proteins / chemistry*
  • Proteins / drug effects
  • Proteomics
  • Small Molecule Libraries

Substances

  • Mutant Chimeric Proteins
  • Proteins
  • Small Molecule Libraries