Discovery and Development of Small-Molecule Inhibitors of Glycogen Synthase

Journal of Medicinal Chemistry
2020.0

Abstract

The overaccumulation of glycogen appears as a hallmark in various glycogen storage diseases (GSDs), including Pompe, Cori, Andersen, and Lafora disease. Accumulating evidence suggests that suppression of glycogen accumulation represents a potential therapeutic approach for treating these GSDs. Using a fluorescence polarization assay designed to screen for inhibitors of the key glycogen synthetic enzyme, glycogen synthase (GS), we identified a substituted imidazole, (rac)-2-methoxy-4-(1-(2-(1-methylpyrrolidin-2-yl)ethyl)-4-phenyl-1H-imidazol-5-yl)phenol (H23), as a first-in-class inhibitor for yeast GS 2 (yGsy2p). Data from X-ray crystallography at 2.85 Å, as well as kinetic data, revealed that H23 bound within the uridine diphosphate glucose binding pocket of yGsy2p. The high conservation of residues between human and yeast GS in direct contact with H23 informed the development of around 500 H23 analogs. These analogs produced a structure-activity relationship profile that led to the identification of a substituted pyrazole, 4-(4-(4-hydroxyphenyl)-3-(trifluoromethyl)-1H-pyrazol-5-yl)pyrogallol, with a 300-fold improved potency against human GS. These substituted pyrazoles possess a promising scaffold for drug development efforts targeting GS activity in GSDs associated with excess glycogen accumulation.

Knowledge Graph

Similar Paper

Discovery and Development of Small-Molecule Inhibitors of Glycogen Synthase
Journal of Medicinal Chemistry 2020.0
Substituted 3-Imidazo[1,2-a]pyridin-3-yl- 4-(1,2,3,4-tetrahydro-[1,4]diazepino- [6,7,1-hi]indol-7-yl)pyrrole-2,5-diones as Highly Selective and Potent Inhibitors of Glycogen Synthase Kinase-3
Journal of Medicinal Chemistry 2004.0
Synthesis, Binding Mode, and Antihyperglycemic Activity of Potent and Selective (5-Imidazol-2-yl-4-phenylpyrimidin-2-yl)[2-(2-pyridylamino)ethyl]amine Inhibitors of Glycogen Synthase Kinase 3
Journal of Medicinal Chemistry 2017.0
Synthesis, screening and docking of small heterocycles as Glycogen Phosphorylase inhibitors
European Journal of Medicinal Chemistry 2014.0
Pyridinylimidazoles as dual glycogen synthase kinase 3β/p38α mitogen-activated protein kinase inhibitors
European Journal of Medicinal Chemistry 2019.0
5-Imino-1,2,4-Thiadiazoles: First Small Molecules As Substrate Competitive Inhibitors of Glycogen Synthase Kinase 3
Journal of Medicinal Chemistry 2012.0
2-Oxo-1,2-dihydropyridinyl-3-yl amide-based GPa inhibitors: Design, synthesis and structure-activity relationship study
European Journal of Medicinal Chemistry 2016.0
Novel 8-arylated purines as inhibitors of glycogen synthase kinase
European Journal of Medicinal Chemistry 2010.0
Polyhydroxylated azetidine iminosugars: Synthesis, glycosidase inhibitory activity and molecular docking studies
Bioorganic & Medicinal Chemistry Letters 2017.0
Glycogen phosphorylase inhibitory effects of 2-oxo-1,2-dihydropyridin-3-yl amide derivatives
Bioorganic & Medicinal Chemistry 2009.0