Structure-based discovery of opioid analgesics with reduced side effects

Nature. 2016 Sep 8;537(7619):185-190. doi: 10.1038/nature19112. Epub 2016 Aug 17.

Abstract

Morphine is an alkaloid from the opium poppy used to treat pain. The potentially lethal side effects of morphine and related opioids-which include fatal respiratory depression-are thought to be mediated by μ-opioid-receptor (μOR) signalling through the β-arrestin pathway or by actions at other receptors. Conversely, G-protein μOR signalling is thought to confer analgesia. Here we computationally dock over 3 million molecules against the μOR structure and identify new scaffolds unrelated to known opioids. Structure-based optimization yields PZM21-a potent Gi activator with exceptional selectivity for μOR and minimal β-arrestin-2 recruitment. Unlike morphine, PZM21 is more efficacious for the affective component of analgesia versus the reflexive component and is devoid of both respiratory depression and morphine-like reinforcing activity in mice at equi-analgesic doses. PZM21 thus serves as both a probe to disentangle μOR signalling and a therapeutic lead that is devoid of many of the side effects of current opioids.

MeSH terms

  • Analgesia / methods
  • Analgesics, Opioid / adverse effects*
  • Analgesics, Opioid / chemistry*
  • Analgesics, Opioid / pharmacology
  • Animals
  • Drug Discovery*
  • GTP-Binding Protein alpha Subunits, Gi-Go / metabolism
  • HEK293 Cells
  • Humans
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Molecular Docking Simulation
  • Pain / drug therapy
  • Receptors, Opioid, mu / agonists*
  • Receptors, Opioid, mu / deficiency
  • Receptors, Opioid, mu / genetics
  • Receptors, Opioid, mu / metabolism
  • Spiro Compounds / pharmacology
  • Structure-Activity Relationship
  • Thiophenes / adverse effects
  • Thiophenes / chemistry*
  • Thiophenes / pharmacology*
  • Urea / adverse effects
  • Urea / analogs & derivatives*
  • Urea / chemistry
  • Urea / pharmacology

Substances

  • ((3-methoxythiophen-2-yl)methyl)((2-(9-(pyridin-2-yl)-6-oxaspiro(4.5)decan-9-yl)ethyl))amine
  • Analgesics, Opioid
  • PZM21 compound
  • Receptors, Opioid, mu
  • Spiro Compounds
  • Thiophenes
  • Urea
  • GTP-Binding Protein alpha Subunits, Gi-Go