Discovery of a functionally selective ghrelin receptor (GHSR<sub>1a</sub>) ligand for modulating brain dopamine.

Gross, J D; Kim, D W; Zhou, Y; Jansen, D; Slosky, L M; Clark, N B; Ray, C R; Hu, X et al. · Proc Natl Acad Sci U S A · 2022

basic_science · Level V

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Abstract

SignificanceThe modulation of growth hormone secretagogue receptor-1a (GHSR<sub>1a</sub>) signaling is a promising strategy for treating brain conditions of metabolism, aging, and addiction. GHSR<sub>1a</sub> activation results in pleiotropic physiological outcomes through distinct and pharmacologically separable G protein- and β-arrestin (βarr)-dependent signaling pathways. Thus, pathway-selective modulation can enable improved pharmacotherapeutics that can promote therapeutic efficacy while mitigating side effects. Here, we describe the discovery of a brain-penetrant small molecule, N8279 (NCATS-SM8864), that biases GHSR<sub>1a</sub> conformations toward Gα<sub>q</sub> activation and reduces aberrant dopaminergic behavior in mice. N8279 represents a promising chemical scaffold to advance the development of better treatments for GHSR<sub>1a</sub>-related brain disorders involving the pathological dysregulation of dopamine.

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