Discovery of a functionally selective serotonin receptor (5-HT<sub>1A</sub>R) agonist for the treatment of pain.

Ullrich, Annika; Schneider, Johannes; Braz, João M; Neu, Eduard; Staffen, Nico; Stanek, Markus; Bláhová, Jana; Hove, Tamsanqa et al. · Sci Adv · 2025

basic_science · Level V

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Abstract

The heterotrimeric G protein-coupled serotonin receptor 5-HT<sub>1A</sub> receptor (5-HT<sub>1A</sub>R) 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-HT<sub>1A</sub>R agonist that revealed highly potent and functionally selective G<sub>i/o</sub> signaling without G<sub>s</sub> activation and marginal β-arrestin recruitment. ST171 is effective in acute and chronic pain models. Cryo-electron microscopy structures of ST171 bound to 5-HT<sub>1A</sub>R in complex with the G<sub>i</sub> protein compared to the canonical agonist befiradol bound to complexes of 5-HT<sub>1A</sub>R with G<sub>i</sub> or G<sub>s</sub> 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.

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