The evasive nature of drug efficacy: implications for drug discovery

Trends Pharmacol Sci. 2007 Aug;28(8):423-30. doi: 10.1016/j.tips.2007.06.005. Epub 2007 Jul 19.

Abstract

The efficacy of a drug is generally determined by the drug's ability to promote a quantifiable biological response. In the context of the classical receptor-occupancy theory, the efficacy is considered an intrinsic property of the ligand/receptor pair, and it is often assumed to be the same for all the responses evoked by this pair. The recognition that a single receptor can engage different signalling pathways and that various drugs binding to this receptor might differentially influence each of these pathways led to the reassessment of the efficacy concept. Of particular notice is the fact that ligands that behave as agonists toward a given signalling pathway can act, through the same receptor, as antagonists or even inverse agonists on a different pathway in the same cell. These observations, variously referred to as 'ligand-directed trafficking of receptor signalling' (LDTRS), 'functional selectivity', 'biased agonism', 'ligand-biased efficacy', 'collateral efficacy' or 'pluridimensional efficacy', have important implications for the molecular definition of efficacy and the process of drug discovery.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Drug Design*
  • Humans
  • Models, Biological
  • Pharmaceutical Preparations / administration & dosage
  • Pharmaceutical Preparations / metabolism*
  • Protein Binding
  • Receptors, G-Protein-Coupled / agonists
  • Receptors, G-Protein-Coupled / antagonists & inhibitors
  • Receptors, G-Protein-Coupled / metabolism*
  • Signal Transduction / drug effects

Substances

  • Pharmaceutical Preparations
  • Receptors, G-Protein-Coupled