Discovery of a functionally selective serotonin receptor (5-HT1AR) agonist for the treatment of pain

Sci Adv. 2025 Jun 20;11(25):eadv9267. doi: 10.1126/sciadv.adv9267. Epub 2025 Jun 18.

Abstract

The heterotrimeric G protein-coupled serotonin receptor 5-HT1A receptor (5-HT1AR) mediates antinociception and may serve as a valuable target for the treatment of pain. Starting from a chemical library, we evolved ST171, a bitopic 5-HT1AR agonist that revealed highly potent and functionally selective Gi/o signaling without Gs activation and marginal β-arrestin recruitment. ST171 is effective in acute and chronic pain models. Cryo-electron microscopy structures of ST171 bound to 5-HT1AR in complex with the Gi protein compared to the canonical agonist befiradol bound to complexes of 5-HT1AR with Gi or Gs revealed that the ligands occupy different exo-sites. The individual binding poses are associated with ligand-specific receptor conformations that were further studied by molecular dynamics simulations, allowing us to better understand ligand bias, a phenomenon that may be crucial to the discovery of more effective and safe G protein-coupled receptor drugs.

MeSH terms

  • Animals
  • Cryoelectron Microscopy
  • Drug Discovery
  • HEK293 Cells
  • Humans
  • Ligands
  • Mice
  • Molecular Dynamics Simulation
  • Pain* / drug therapy
  • Pain* / metabolism
  • Protein Binding
  • Receptor, Serotonin, 5-HT1A* / chemistry
  • Receptor, Serotonin, 5-HT1A* / metabolism
  • Serotonin 5-HT1 Receptor Agonists* / chemistry
  • Serotonin 5-HT1 Receptor Agonists* / pharmacology
  • Serotonin 5-HT1 Receptor Agonists* / therapeutic use

Substances

  • Receptor, Serotonin, 5-HT1A
  • Serotonin 5-HT1 Receptor Agonists
  • Ligands