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Mechanism and cellular actions of the potent AMPK inhibitor BAY-3827

Title: Mechanism and cellular actions of the potent AMPK inhibitor BAY-3827
Authors: Bringas, Conchita Fraguas; Ahangar, Mohd Syed; Cuenco, Joyceline; Liu, Hongling; Addinsall, Alex; Lindahl, Maria; Ovens, Ashley; Febbraio, Mark; Foretz, Marc; Göransson, Olga; Scott, John; Zeqiraj, Elton; Sakamoto, Kei
Contributors: Novo Nordisk Foundation Center for Basic Metabolic Research (CBMR); Faculty of Health and Medical Sciences; University of Copenhagen = Københavns Universitet (UCPH)-University of Copenhagen = Københavns Universitet (UCPH); University of Leeds; Lund University; Monash Institute of Pharmaceutical Sciences Parkville (MIPS); Faculty of Pharmacy and Pharmaceutical Sciences - Monash University Parkville; Monash university-Monash university; Institut Cochin (IC UM3 (UMR 8104 / U1016)); Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université Paris Cité (UPCité)
Source: ISSN: 2375-2548 ; Science Advances ; https://hal.science/hal-05398100 ; Science Advances , 2025, 11 (34), ⟨10.1126/sciadv.adx2434⟩.
Publisher Information: CCSD; American Association for the Advancement of Science (AAAS)
Publication Year: 2025
Subject Terms: [SDV.BBM.BC]Life Sciences [q-bio]/Biochemistry; Molecular Biology/Biochemistry [q-bio.BM]; [SDV.BBM.BM]Life Sciences [q-bio]/Biochemistry; Molecular Biology/Molecular biology; [SDV.MHEP.EM]Life Sciences [q-bio]/Human health and pathology/Endocrinology and metabolism
Description: International audience ; Inhibition of adenosine 5′-monophosphate (AMP)–activated protein kinase (AMPK) is under increasing investigation for its therapeutic potential in many diseases. Existing AMPK inhibitors are however limited, with poor selectivity and substantial off-target effects. Here, we provide mechanistic insights and describe the cellular selectivity of the recently identified AMPK inhibitor BAY-3827. A 2.5-Å cocrystal structure of the AMPK kinase domain with BAY-3827 revealed distinct features including a disulfide bridge between the αD helix Cys 106 and the activation loop residue Cys 174 . This bridge appears to stabilize the activation loop such that Asn 162 repositions the Asp-Phe-Gly (DFG) motif Phe 158 toward the C-terminal lobe, displacing His 137 and disrupting the regulatory spine, promoting an inactive kinase state. In hepatocytes, BAY-3827 blocked AMPK activator (MK-8722)–mediated phosphorylation of ACC1 and corresponding inhibition of lipogenesis. Transcriptome analysis revealed that BAY-3827 down-regulated ~30% of MK-8722–stimulated AMPK-dependent genes. We establish the molecular and cellular basis of BAY-3827’s selectivity and utility for delineating AMPK functions while highlighting its limitations.
Document Type: article in journal/newspaper
Language: English
DOI: 10.1126/sciadv.adx2434
Availability: https://hal.science/hal-05398100; https://hal.science/hal-05398100v1/document; https://hal.science/hal-05398100v1/file/sciadv.adx2434.pdf; https://doi.org/10.1126/sciadv.adx2434
Rights: http://creativecommons.org/licenses/by/ ; info:eu-repo/semantics/OpenAccess
Accession Number: edsbas.7CED0831
Database: BASE