An exploratory analysis of bezisterim treatment associated with decreased biological age acceleration, and improved clinical measure and biomarker changes in mild-to-moderate probable Alzheimer's disease

IntroductionAging is the primary risk factor for sporadic Alzheimer's disease. Chronic low-grade inflammation associated with aging drives cognitive impairment through multiple mechanisms involving oxidative stress, insulin resistance, and dysregulation of metabolic, immunologic, and hematologi...

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Main Authors: Christopher L. Reading, Jiayan Yan, Marcia A. Testa, Donald C. Simonson, Hira Javaid, Lisa Schmunk, Daniel E. Martin-Herranz, Robert Brooke, Juozas Gordevicius, Jeffrey Zhang, Harvey Yuan, Clarence Ahlem, Lixia Wang, Penelope Markham, Nily Osman, Stephen O'Quinn, Joseph Palumbo
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Language:English
Published: Frontiers Media S.A. 2025-05-01
Series:Frontiers in Neuroscience
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Online Access:https://www.frontiersin.org/articles/10.3389/fnins.2025.1516746/full
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author Christopher L. Reading
Jiayan Yan
Marcia A. Testa
Donald C. Simonson
Hira Javaid
Lisa Schmunk
Daniel E. Martin-Herranz
Robert Brooke
Juozas Gordevicius
Jeffrey Zhang
Harvey Yuan
Clarence Ahlem
Lixia Wang
Penelope Markham
Nily Osman
Stephen O'Quinn
Joseph Palumbo
author_facet Christopher L. Reading
Jiayan Yan
Marcia A. Testa
Donald C. Simonson
Hira Javaid
Lisa Schmunk
Daniel E. Martin-Herranz
Robert Brooke
Juozas Gordevicius
Jeffrey Zhang
Harvey Yuan
Clarence Ahlem
Lixia Wang
Penelope Markham
Nily Osman
Stephen O'Quinn
Joseph Palumbo
author_sort Christopher L. Reading
collection DOAJ
description IntroductionAging is the primary risk factor for sporadic Alzheimer's disease. Chronic low-grade inflammation associated with aging drives cognitive impairment through multiple mechanisms involving oxidative stress, insulin resistance, and dysregulation of metabolic, immunologic, and hematologic systems.MethodsIn a 7-month, randomized, double-blind, placebo-controlled trial (NCT04669028), we investigated the safety and activity of bezisterim, a first-in-class, oral, blood–brain barrier–permeable, anti-inflammatory agent on cognitive, molecular, biochemical, physiological, and biological aging parameters in a subset of 50 mild-to-moderate probable Alzheimer's disease participants. These participants had source-document-verified clinical measures and samples, and they completed the protocol. This study focuses on epigenetic, metabolic, biomarker, and cognitive measures in the exploratory biomarker population that completed the protocol.ResultsBezisterim was associated with non-significant directional improvements in multiple measures of cognitive and functional performance compared to placebo, with correlations to biological age (determined by DNA methylation “clocks”) and to metabolism, inflammation, and dementia biomarkers. In addition, clinical measures correlated with the extent of DNA methylation of certain cytosine-phosphate-guanine (CpG) sites in genes associated with metabolic inflammation and neurodegeneration.DiscussionThe results suggest the possible use of bezisterim to target the multifactorial processes underlying dementia.Clinical trial registrationhttps://clinicaltrials.gov/study/NCT04669028, Identifier: NCT04669028.
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spelling doaj-art-dd0bc5bca5f84c6e8c557615b87c0a8b2025-08-20T02:27:50ZengFrontiers Media S.A.Frontiers in Neuroscience1662-453X2025-05-011910.3389/fnins.2025.15167461516746An exploratory analysis of bezisterim treatment associated with decreased biological age acceleration, and improved clinical measure and biomarker changes in mild-to-moderate probable Alzheimer's diseaseChristopher L. Reading0Jiayan Yan1Marcia A. Testa2Donald C. Simonson3Hira Javaid4Lisa Schmunk5Daniel E. Martin-Herranz6Robert Brooke7Juozas Gordevicius8Jeffrey Zhang9Harvey Yuan10Clarence Ahlem11Lixia Wang12Penelope Markham13Nily Osman14Stephen O'Quinn15Joseph Palumbo16BioVie Inc., Carson City, NV, United StatesBioVie Inc., Carson City, NV, United StatesDepartment of Biostatistics, Harvard T. H. Chan School of Public Health, Boston, MA, United StatesDivision of Endocrinology, Diabetes, and Hypertension, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, United StatesHurdle.bio/Chronomics Ltd., London, United KingdomHurdle.bio/Chronomics Ltd., London, United KingdomHurdle.bio/Chronomics Ltd., London, United KingdomEpigenetic Clock Development Foundation, Torrance, CA, United StatesEpigenetic Clock Development Foundation, Torrance, CA, United StatesPrinceton Pharmatech, Princeton, NJ, United StatesPrinceton Pharmatech, Princeton, NJ, United StatesBioVie Inc., Carson City, NV, United StatesBioVie Inc., Carson City, NV, United StatesBioVie Inc., Carson City, NV, United StatesBioVie Inc., Carson City, NV, United StatesBioVie Inc., Carson City, NV, United StatesBioVie Inc., Carson City, NV, United StatesIntroductionAging is the primary risk factor for sporadic Alzheimer's disease. Chronic low-grade inflammation associated with aging drives cognitive impairment through multiple mechanisms involving oxidative stress, insulin resistance, and dysregulation of metabolic, immunologic, and hematologic systems.MethodsIn a 7-month, randomized, double-blind, placebo-controlled trial (NCT04669028), we investigated the safety and activity of bezisterim, a first-in-class, oral, blood–brain barrier–permeable, anti-inflammatory agent on cognitive, molecular, biochemical, physiological, and biological aging parameters in a subset of 50 mild-to-moderate probable Alzheimer's disease participants. These participants had source-document-verified clinical measures and samples, and they completed the protocol. This study focuses on epigenetic, metabolic, biomarker, and cognitive measures in the exploratory biomarker population that completed the protocol.ResultsBezisterim was associated with non-significant directional improvements in multiple measures of cognitive and functional performance compared to placebo, with correlations to biological age (determined by DNA methylation “clocks”) and to metabolism, inflammation, and dementia biomarkers. In addition, clinical measures correlated with the extent of DNA methylation of certain cytosine-phosphate-guanine (CpG) sites in genes associated with metabolic inflammation and neurodegeneration.DiscussionThe results suggest the possible use of bezisterim to target the multifactorial processes underlying dementia.Clinical trial registrationhttps://clinicaltrials.gov/study/NCT04669028, Identifier: NCT04669028.https://www.frontiersin.org/articles/10.3389/fnins.2025.1516746/fullAlzheimer'sneuroinflammationinsulinagingDNA methylationbezisterim
spellingShingle Christopher L. Reading
Jiayan Yan
Marcia A. Testa
Donald C. Simonson
Hira Javaid
Lisa Schmunk
Daniel E. Martin-Herranz
Robert Brooke
Juozas Gordevicius
Jeffrey Zhang
Harvey Yuan
Clarence Ahlem
Lixia Wang
Penelope Markham
Nily Osman
Stephen O'Quinn
Joseph Palumbo
An exploratory analysis of bezisterim treatment associated with decreased biological age acceleration, and improved clinical measure and biomarker changes in mild-to-moderate probable Alzheimer's disease
Frontiers in Neuroscience
Alzheimer's
neuroinflammation
insulin
aging
DNA methylation
bezisterim
title An exploratory analysis of bezisterim treatment associated with decreased biological age acceleration, and improved clinical measure and biomarker changes in mild-to-moderate probable Alzheimer's disease
title_full An exploratory analysis of bezisterim treatment associated with decreased biological age acceleration, and improved clinical measure and biomarker changes in mild-to-moderate probable Alzheimer's disease
title_fullStr An exploratory analysis of bezisterim treatment associated with decreased biological age acceleration, and improved clinical measure and biomarker changes in mild-to-moderate probable Alzheimer's disease
title_full_unstemmed An exploratory analysis of bezisterim treatment associated with decreased biological age acceleration, and improved clinical measure and biomarker changes in mild-to-moderate probable Alzheimer's disease
title_short An exploratory analysis of bezisterim treatment associated with decreased biological age acceleration, and improved clinical measure and biomarker changes in mild-to-moderate probable Alzheimer's disease
title_sort exploratory analysis of bezisterim treatment associated with decreased biological age acceleration and improved clinical measure and biomarker changes in mild to moderate probable alzheimer s disease
topic Alzheimer's
neuroinflammation
insulin
aging
DNA methylation
bezisterim
url https://www.frontiersin.org/articles/10.3389/fnins.2025.1516746/full
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