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Pharmacologic approaches to the treatment of Huntington's disease

Authors

  • Charles S. Venuto PharmD,

    1. Center for Human Experimental Therapeutics, University of Rochester School of Medicine and Dentistry, Rochester, New York, USA
    2. Department of Pharmacy Practice, School of Pharmacy and Pharmaceutical Sciences, University at Buffalo, Buffalo, New York, USA
    3. Translational Pharmacology Research Core, New York State Center of Excellence in Bioinformatics and Life Sciences, Buffalo, New York, USA
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  • Andrew McGarry MD,

    1. Department of Neurology, University of Rochester School of Medicine and Dentistry, Rochester, New York, USA
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    • Full financial disclosures and author roles may be found in the online version of this article.

  • Qing Ma PhD,

    1. Center for Human Experimental Therapeutics, University of Rochester School of Medicine and Dentistry, Rochester, New York, USA
    2. Department of Pharmacy Practice, School of Pharmacy and Pharmaceutical Sciences, University at Buffalo, Buffalo, New York, USA
    3. Translational Pharmacology Research Core, New York State Center of Excellence in Bioinformatics and Life Sciences, Buffalo, New York, USA
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    • Full financial disclosures and author roles may be found in the online version of this article.

  • Karl Kieburtz MD, MPH

    Corresponding author
    1. Center for Human Experimental Therapeutics, University of Rochester School of Medicine and Dentistry, Rochester, New York, USA
    2. Department of Neurology, University of Rochester School of Medicine and Dentistry, Rochester, New York, USA
    • University of Rochester Medical Center, CHET/CTCC, 265 Crittenden Blvd., Rochester, NY 14642-0694
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    • Full financial disclosures and author roles may be found in the online version of this article.


  • Relevant conflicts of interest/financial disclosures: Authors Venuto, McGarry, and Kieburtz receive research support from a contract between the University of Rochester and NeuroSearch. Author Kieburtz also receives research support from Medivation, Inc.

Abstract

Huntington's disease (HD) is an inherited, progressive neurodegenerative disorder characterized by chorea, cognitive impairment, and behavioral disturbances. Despite advances in diagnosis and improved understanding of HD, treatment remains difficult due to challenging symptoms and a paucity of approved therapeutic interventions. Nonpharmacologic and pharmacologic strategies have been evaluated; regarding the latter, over 80 agents of various classes have been investigated in clinical trials or examined in case reports. Symptomatic treatment, however, is generally confined to antidopaminergic agents for motor dysfunction and antidepressants for mood disorders, while treatment for cognitive dysfunction remains vacant. Several different mechanisms to modify symptoms and disease progression have been targeted in clinical trials. This article reviews some of the more common pharmacologic treatments used for HD, discusses data regarding suboptimal agents that have been tested, and surveys treatments under investigation. © 2011 Movement Disorder Society

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