Glucagon-like peptide-1 receptor agonists in neurodegenerative diseases: Promises and challenges

Glucagon-like peptide-1 (GLP-1) receptor agonists (GRA) belong to a class of compounds that reduce blood glucose and energy intake by simulating actions of endogenous incretin hormone GLP-1 after it is released by the gut following food consumption. They are used to treat type 2 diabetes mellitus (T...

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Main Authors: Zhi Dong Zhou, Lingxiao Yi, Karolina Popławska-Domaszewicz, Kallol Ray Chaudhuri, Joseph Jankovic, Eng King Tan
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Pharmacological Research
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Online Access:http://www.sciencedirect.com/science/article/pii/S1043661825001951
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author Zhi Dong Zhou
Lingxiao Yi
Karolina Popławska-Domaszewicz
Kallol Ray Chaudhuri
Joseph Jankovic
Eng King Tan
author_facet Zhi Dong Zhou
Lingxiao Yi
Karolina Popławska-Domaszewicz
Kallol Ray Chaudhuri
Joseph Jankovic
Eng King Tan
author_sort Zhi Dong Zhou
collection DOAJ
description Glucagon-like peptide-1 (GLP-1) receptor agonists (GRA) belong to a class of compounds that reduce blood glucose and energy intake by simulating actions of endogenous incretin hormone GLP-1 after it is released by the gut following food consumption. They are used to treat type 2 diabetes mellitus (T2DM) and obesity and have systemic effects on various organs, including the brain, liver, pancreas, heart, and the gut. Patients with T2DM have a higher risk of developing neurodegenerative diseases (NDs), including Alzheimer’s disease (AD), Parkinson's disease (PD), amyotrophic lateral sclerosis (ALS) and Huntington’s disease (HD), accompanied by more severe motor deficits and faster disease progression, suggesting dysregulation of insulin signaling in these diseases. Experimental studies have shown that GRA have protective effects to modulate neuroinflammation, oxidative stress, mitochondrial and autophagic functions, and protein misfolding. Hence the compounds have generated enormous interest as novel therapeutic agents against NDs. To date, clinical trials have shown that three GRA, exenatide, liraglutide and lixisenatide can improve motor deficits as an add-on therapy in PD patients and liraglutide can improve cognitive function in AD patients. The neuroprotective effects of these and other GRA, such as PT320 (a sustained-released exenatide) and semaglutide, are still under investigation. The dual GLP-1/gastric inhibitory polypeptide (GIP) receptor agonists have been demonstrated to have beneficial effects in AD and PD mice models. Overall, GRA are highly promising novel drugs, but future clinical studies should identify which subsets of patients should be targeted as potential candidates for their symptomatic and/or neuroprotective benefits, investigate whether combinations with other classes of drugs can further augment their efficacy, and evaluate their long-term disease-modifying and adverse effects.
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spelling doaj-art-bcd52fb812eb4d629c85a8fc11fca72b2025-08-20T02:32:20ZengElsevierPharmacological Research1096-11862025-06-0121610777010.1016/j.phrs.2025.107770Glucagon-like peptide-1 receptor agonists in neurodegenerative diseases: Promises and challengesZhi Dong Zhou0Lingxiao Yi1Karolina Popławska-Domaszewicz2Kallol Ray Chaudhuri3Joseph Jankovic4Eng King Tan5National Neuroscience Institute of Singapore, 11 Jalan Tan Tock Seng, 308433, Singapore; Signature Research Program in Neuroscience and Behavioral Disorders, Duke-NUS Medical School, 8 College Road, 169857, SingaporeNational Neuroscience Institute of Singapore, 11 Jalan Tan Tock Seng, 308433, SingaporeDepartment of Neurology, Poznan University of Medical Sciences, Poznan 60-355, Poland; Parkinson’s Foundation Centre of Excellence, King’s College Hospital, Denmark Hill, London SE5 9RS, UKDepartment of Basic and Clinical Neuroscience, The Maurice Wohl Clinical Neuroscience Institute, King's College London, Cutcombe Road, London SE5 9RT, UKParkinson’s Disease Center and Movement Disorders Clinic, Department of Neurology, Baylor College of Medicine, Houston, TX, USANational Neuroscience Institute of Singapore, 11 Jalan Tan Tock Seng, 308433, Singapore; Signature Research Program in Neuroscience and Behavioral Disorders, Duke-NUS Medical School, 8 College Road, 169857, Singapore; Correspondence to: Department of Neurology, National Neuroscience Institute of Singapore (Singapore General Hospital campus), Outram Road, 169608, Singapore.Glucagon-like peptide-1 (GLP-1) receptor agonists (GRA) belong to a class of compounds that reduce blood glucose and energy intake by simulating actions of endogenous incretin hormone GLP-1 after it is released by the gut following food consumption. They are used to treat type 2 diabetes mellitus (T2DM) and obesity and have systemic effects on various organs, including the brain, liver, pancreas, heart, and the gut. Patients with T2DM have a higher risk of developing neurodegenerative diseases (NDs), including Alzheimer’s disease (AD), Parkinson's disease (PD), amyotrophic lateral sclerosis (ALS) and Huntington’s disease (HD), accompanied by more severe motor deficits and faster disease progression, suggesting dysregulation of insulin signaling in these diseases. Experimental studies have shown that GRA have protective effects to modulate neuroinflammation, oxidative stress, mitochondrial and autophagic functions, and protein misfolding. Hence the compounds have generated enormous interest as novel therapeutic agents against NDs. To date, clinical trials have shown that three GRA, exenatide, liraglutide and lixisenatide can improve motor deficits as an add-on therapy in PD patients and liraglutide can improve cognitive function in AD patients. The neuroprotective effects of these and other GRA, such as PT320 (a sustained-released exenatide) and semaglutide, are still under investigation. The dual GLP-1/gastric inhibitory polypeptide (GIP) receptor agonists have been demonstrated to have beneficial effects in AD and PD mice models. Overall, GRA are highly promising novel drugs, but future clinical studies should identify which subsets of patients should be targeted as potential candidates for their symptomatic and/or neuroprotective benefits, investigate whether combinations with other classes of drugs can further augment their efficacy, and evaluate their long-term disease-modifying and adverse effects.http://www.sciencedirect.com/science/article/pii/S1043661825001951Glucagon-like peptide-1GLP-1 receptor agonistsNeurodegenerative diseasesAlzheimer’s diseaseParkinsons' diseaseType 2 diabetes mellitus
spellingShingle Zhi Dong Zhou
Lingxiao Yi
Karolina Popławska-Domaszewicz
Kallol Ray Chaudhuri
Joseph Jankovic
Eng King Tan
Glucagon-like peptide-1 receptor agonists in neurodegenerative diseases: Promises and challenges
Pharmacological Research
Glucagon-like peptide-1
GLP-1 receptor agonists
Neurodegenerative diseases
Alzheimer’s disease
Parkinsons' disease
Type 2 diabetes mellitus
title Glucagon-like peptide-1 receptor agonists in neurodegenerative diseases: Promises and challenges
title_full Glucagon-like peptide-1 receptor agonists in neurodegenerative diseases: Promises and challenges
title_fullStr Glucagon-like peptide-1 receptor agonists in neurodegenerative diseases: Promises and challenges
title_full_unstemmed Glucagon-like peptide-1 receptor agonists in neurodegenerative diseases: Promises and challenges
title_short Glucagon-like peptide-1 receptor agonists in neurodegenerative diseases: Promises and challenges
title_sort glucagon like peptide 1 receptor agonists in neurodegenerative diseases promises and challenges
topic Glucagon-like peptide-1
GLP-1 receptor agonists
Neurodegenerative diseases
Alzheimer’s disease
Parkinsons' disease
Type 2 diabetes mellitus
url http://www.sciencedirect.com/science/article/pii/S1043661825001951
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