Randomized, double-blind, placebo-controlled pilot study of metformin as an adjunctive therapy in Parkinson’s disease

BackgroundParkinson’s disease (PD) is caused by the progressive loss of dopaminergic neurons in the substantia nigra. Neuroinflammation is considered a key factor contributing to the pathophysiology of PD. Current gold-standard therapies for PD provide only symptomatic relief without slowing disease...

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Main Authors: Hayam Ali AlRasheed, Mostafa M. Bahaa, Thanaa A. Elmasry, Eman I. Elberri, Fedaa A. Kotkata, Ramy M. El Sabaa, Yasmine M. Elmorsi, Mostafa M. Kamel, Walaa A. Negm, Amir O. Hamouda, Khlood Mohammad Aldossary, Muhammed M. Salahuddin, Mohamed Yasser, Mamdouh Eldesouqui, Manal A. Hamouda, Nashwa Eltantawy, Mirna E. Elawady, Mahmoud S. Abdallah
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-05-01
Series:Frontiers in Pharmacology
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Online Access:https://www.frontiersin.org/articles/10.3389/fphar.2025.1497261/full
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author Hayam Ali AlRasheed
Mostafa M. Bahaa
Thanaa A. Elmasry
Thanaa A. Elmasry
Eman I. Elberri
Fedaa A. Kotkata
Ramy M. El Sabaa
Yasmine M. Elmorsi
Mostafa M. Kamel
Walaa A. Negm
Amir O. Hamouda
Khlood Mohammad Aldossary
Muhammed M. Salahuddin
Mohamed Yasser
Mohamed Yasser
Mohamed Yasser
Mamdouh Eldesouqui
Manal A. Hamouda
Nashwa Eltantawy
Mirna E. Elawady
Mahmoud S. Abdallah
Mahmoud S. Abdallah
author_facet Hayam Ali AlRasheed
Mostafa M. Bahaa
Thanaa A. Elmasry
Thanaa A. Elmasry
Eman I. Elberri
Fedaa A. Kotkata
Ramy M. El Sabaa
Yasmine M. Elmorsi
Mostafa M. Kamel
Walaa A. Negm
Amir O. Hamouda
Khlood Mohammad Aldossary
Muhammed M. Salahuddin
Mohamed Yasser
Mohamed Yasser
Mohamed Yasser
Mamdouh Eldesouqui
Manal A. Hamouda
Nashwa Eltantawy
Mirna E. Elawady
Mahmoud S. Abdallah
Mahmoud S. Abdallah
author_sort Hayam Ali AlRasheed
collection DOAJ
description BackgroundParkinson’s disease (PD) is caused by the progressive loss of dopaminergic neurons in the substantia nigra. Neuroinflammation is considered a key factor contributing to the pathophysiology of PD. Current gold-standard therapies for PD provide only symptomatic relief without slowing disease progression, highlighting the need to develop new disease-modifying treatments. Metformin has been demonstrated to exert a neuroprotective role in several neurodegenerative disorders including PD.AimThis study aimed to clarify the role of metformin as adjuvant therapy in patients with PD.MethodsSixty patients with PD were divided into 2 groups (n = 30). Patients in group 1 received levodopa/carbidopa (250/25 mg) three times daily for 3 months plus placebo (Control group), while those in group 2 received levodopa/carbidopa (250/25 mg) three times daily and 500 mg metformin two times daily (Metformin group). Patients were assessed via Unified Parkinson’s Disease Rating Scale (UPDRS). The serum concentrations of toll like receptor 4 (TLR-4), α-synuclein, brain derived neurotropic factor (BDNF), and high mobility group box 1 (HMGB-1) were measured before and after treatment.Primary outcomeThe improvement in UPDRS from baseline to 3 months.Secondary outcomeChange in the level of biological markers.ResultsThe control group did not show significant difference in UPDRS when compared to their baseline value by Wilcoxon test (P > 0.05), meanwhile the metformin group showed significant difference when compared to before treatment by Wilcoxon test (P < 0.05). There were no significant differences between the two groups in UPDRS after treatment (P > 0.05) by Man Whitney test. However, the metformin group showed a significant decrease in TLR-4, HMGB-1, and α-synuclein along with a statistically significant increase in BDNF (P < 0.05) when compared to its baseline and control group. The control group did not show any significant changes in all markers when compared to their baseline.ConclusionWhile no significant differences in UPDRS scores were observed between the metformin and control groups, trends in biomarker changes suggest a potential impact of adjunctive metformin use on the underlying pathophysiology of PD. Further studies are needed to assess its effects on motor symptoms over a longer duration.Clinical Trial Registrationidentifier NCT05781711.
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spelling doaj-art-ccf4b849028b4878908b103a9cae1eb32025-08-20T03:49:04ZengFrontiers Media S.A.Frontiers in Pharmacology1663-98122025-05-011610.3389/fphar.2025.14972611497261Randomized, double-blind, placebo-controlled pilot study of metformin as an adjunctive therapy in Parkinson’s diseaseHayam Ali AlRasheed0Mostafa M. Bahaa1Thanaa A. Elmasry2Thanaa A. Elmasry3Eman I. Elberri4Fedaa A. Kotkata5Ramy M. El Sabaa6Yasmine M. Elmorsi7Mostafa M. Kamel8Walaa A. Negm9Amir O. Hamouda10Khlood Mohammad Aldossary11Muhammed M. Salahuddin12Mohamed Yasser13Mohamed Yasser14Mohamed Yasser15Mamdouh Eldesouqui16Manal A. Hamouda17Nashwa Eltantawy18Mirna E. Elawady19Mahmoud S. Abdallah20Mahmoud S. Abdallah21Department of Pharmacy Practice, College of Pharmacy, Princess Nourah bint Abdulrahman University, Riyadh, Saudi ArabiaPharmacy Practice Department, Faculty of Pharmacy, Horus University, New Damietta, EgyptPharmacology and Toxicology Department, Faculty of Pharmacy, Tanta University, Tanta, Al-Gharbia, EgyptPharmacy Practice Department, Faculty of Pharmacy, Sinai University, Arish Branch, Arish, EgyptDepartment of Clinical Pharmacy, Faculty of Pharmacy, Tanta University, Tanta, Al-Gharbia, EgyptDepartment of Clinical Pharmacy, Faculty of Pharmacy, Tanta University, Tanta, Al-Gharbia, EgyptDepartment of Clinical Pharmacy, Faculty of Pharmacy, Menoufia University, Shebin El-Kom, Menoufia, EgyptDepartment of Clinical Pharmacy, Faculty of Pharmacy, Tanta University, Tanta, Al-Gharbia, EgyptPsychiatry Department, Faculty of Medicine, Tanta University, EgyptPharmacognosy Department, Faculty of Pharmacy, Tanta University, Tanta, Al-Gharbia, EgyptDepartment of Biochemistry and Pharmacology, Faculty of Pharmacy, Horus University, New Damietta, EgyptDepartment of Pharmacy Practice, College of Pharmacy, Princess Nourah bint Abdulrahman University, Riyadh, Saudi ArabiaDepartment of Biochemistry and Pharmacology, Faculty of Pharmacy, Horus University, New Damietta, Egypt0Department of Pharmaceutics, Faculty of Pharmacy, Port Said University, Port Said, Egypt1Department of Pharmaceutics, Faculty of Pharmacy, East Port Said National University, Port Said, Egypt2Department of Pharmaceutics and Industrial Pharmacy, Faculty of Pharmacy, Horus University, New Damietta, Egypt0Department of Pharmaceutics, Faculty of Pharmacy, Port Said University, Port Said, EgyptDepartment of Clinical Pharmacy, Faculty of Pharmacy, Menoufia University, Shebin El-Kom, Menoufia, Egypt4Department of Pharmacy Practice, Faculty of Pharmacy and Drug Technology, Egyptian Chinese University, Cairo, Egypt5Department of Pharmacy Practice, Faculty of Pharmacy, Sinai University, Kantara, Ismailia, Egypt6Department of Clinical Pharmacy, Faculty of Pharmacy, University of Sadat City (USC), Sadat City, Menoufia, Egypt7Department of PharmD, Faculty of Pharmacy, Jadara University, Irbid, JordanBackgroundParkinson’s disease (PD) is caused by the progressive loss of dopaminergic neurons in the substantia nigra. Neuroinflammation is considered a key factor contributing to the pathophysiology of PD. Current gold-standard therapies for PD provide only symptomatic relief without slowing disease progression, highlighting the need to develop new disease-modifying treatments. Metformin has been demonstrated to exert a neuroprotective role in several neurodegenerative disorders including PD.AimThis study aimed to clarify the role of metformin as adjuvant therapy in patients with PD.MethodsSixty patients with PD were divided into 2 groups (n = 30). Patients in group 1 received levodopa/carbidopa (250/25 mg) three times daily for 3 months plus placebo (Control group), while those in group 2 received levodopa/carbidopa (250/25 mg) three times daily and 500 mg metformin two times daily (Metformin group). Patients were assessed via Unified Parkinson’s Disease Rating Scale (UPDRS). The serum concentrations of toll like receptor 4 (TLR-4), α-synuclein, brain derived neurotropic factor (BDNF), and high mobility group box 1 (HMGB-1) were measured before and after treatment.Primary outcomeThe improvement in UPDRS from baseline to 3 months.Secondary outcomeChange in the level of biological markers.ResultsThe control group did not show significant difference in UPDRS when compared to their baseline value by Wilcoxon test (P > 0.05), meanwhile the metformin group showed significant difference when compared to before treatment by Wilcoxon test (P < 0.05). There were no significant differences between the two groups in UPDRS after treatment (P > 0.05) by Man Whitney test. However, the metformin group showed a significant decrease in TLR-4, HMGB-1, and α-synuclein along with a statistically significant increase in BDNF (P < 0.05) when compared to its baseline and control group. The control group did not show any significant changes in all markers when compared to their baseline.ConclusionWhile no significant differences in UPDRS scores were observed between the metformin and control groups, trends in biomarker changes suggest a potential impact of adjunctive metformin use on the underlying pathophysiology of PD. Further studies are needed to assess its effects on motor symptoms over a longer duration.Clinical Trial Registrationidentifier NCT05781711.https://www.frontiersin.org/articles/10.3389/fphar.2025.1497261/fullParkinson diseasemetforminneuro-inflammationα-synucleinTLR-4
spellingShingle Hayam Ali AlRasheed
Mostafa M. Bahaa
Thanaa A. Elmasry
Thanaa A. Elmasry
Eman I. Elberri
Fedaa A. Kotkata
Ramy M. El Sabaa
Yasmine M. Elmorsi
Mostafa M. Kamel
Walaa A. Negm
Amir O. Hamouda
Khlood Mohammad Aldossary
Muhammed M. Salahuddin
Mohamed Yasser
Mohamed Yasser
Mohamed Yasser
Mamdouh Eldesouqui
Manal A. Hamouda
Nashwa Eltantawy
Mirna E. Elawady
Mahmoud S. Abdallah
Mahmoud S. Abdallah
Randomized, double-blind, placebo-controlled pilot study of metformin as an adjunctive therapy in Parkinson’s disease
Frontiers in Pharmacology
Parkinson disease
metformin
neuro-inflammation
α-synuclein
TLR-4
title Randomized, double-blind, placebo-controlled pilot study of metformin as an adjunctive therapy in Parkinson’s disease
title_full Randomized, double-blind, placebo-controlled pilot study of metformin as an adjunctive therapy in Parkinson’s disease
title_fullStr Randomized, double-blind, placebo-controlled pilot study of metformin as an adjunctive therapy in Parkinson’s disease
title_full_unstemmed Randomized, double-blind, placebo-controlled pilot study of metformin as an adjunctive therapy in Parkinson’s disease
title_short Randomized, double-blind, placebo-controlled pilot study of metformin as an adjunctive therapy in Parkinson’s disease
title_sort randomized double blind placebo controlled pilot study of metformin as an adjunctive therapy in parkinson s disease
topic Parkinson disease
metformin
neuro-inflammation
α-synuclein
TLR-4
url https://www.frontiersin.org/articles/10.3389/fphar.2025.1497261/full
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