Pridopidine: Overview of Pharmacology and Rationale for its Use in Huntington's Disease

Journal of Huntington's Disease
Susanna WatersNicholas Waters

Abstract

Despite advances in understanding the pathophysiology of Huntington's disease (HD), there are currently no effective pharmacological agents available to treat core symptoms or to stop or prevent the progression of this hereditary neurodegenerative disorder. Pridopidine, a novel small molecule compound, has demonstrated potential for both symptomatic treatment and disease modifying effects in HD. While pridopidine failed to achieve its primary efficacy outcomes (Modified motor score) in two trials (MermaiHD and HART) there were consistent effects on secondary outcomes (TMS). In the most recent study (PrideHD) pridiopidine did not differ from placebo on TMS, possibly due to a large enduring placebo effect.This review describes the process, based on in vivo systems response profiling, by which pridopidine was discovered and discusses its pharmacological profile, aiming to provide a model for the system-level effects, and a rationale for the use of pridopidine in patients affected by HD. Considering the effects on brain neurochemistry, gene expression and behaviour in vivo, pridopidine displays a unique effect profile. A hallmark feature in the behavioural pharmacology of pridopidine is its state-dependent inhibition or activation ...Continue Reading

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Citations

May 6, 2020·Expert Opinion on Emerging Drugs·Sohaila AlShimemeriNaomi P Visanji
Feb 23, 2020·Reviews in the Neurosciences·Magdalena JabłońskaPrzemysław Gałązka
Sep 26, 2019·Frontiers in Neuroscience·Daniel A RyskampIlya Bezprozvanny
Jan 17, 2020·Brain Sciences·Ashok KumarJong-Joo Kim
Apr 20, 2019·Frontiers in Molecular Biosciences·Talya ShachamGerardo Z Lederkremer
Jul 29, 2021·Expert Opinion on Emerging Drugs·Robert Wiggins, Andrew Feigin

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Methods Mentioned

BETA
transgenic
HART

Software Mentioned

Open

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