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Pharmacologic approaches to the treatment of Huntington's disease

Charles S Venuto1, Andrew McGarry, Qing Ma

  • 1Center for Human Experimental Therapeutics, University of Rochester School of Medicine and Dentistry, Rochester, New York, USA.

Movement Disorders : Official Journal of the Movement Disorder Society
|October 15, 2011
PubMed

Insights

Huntington's disease (HD) treatments are limited, with current options focusing on symptom management for motor and mood disorders. Research is ongoing for novel therapies to address cognitive decline and disease progression in this inherited neurodegenerative condition.

Area of Science:

  • Neuroscience
  • Genetics
  • Pharmacology

Background:

  • Huntington's disease (HD) is an inherited, progressive neurodegenerative disorder.
  • Key features include chorea, cognitive impairment, and behavioral disturbances.
  • Current treatment options for HD are limited, with a paucity of approved therapeutic interventions.

Purpose of the Study:

  • To review common pharmacologic treatments for Huntington's disease (HD).
  • To discuss data on suboptimal agents tested for HD.
  • To survey treatments currently under investigation for HD.

Main Methods:

  • Review of existing literature on pharmacologic treatments for HD.
  • Analysis of clinical trial data and case reports for various agents.
  • Survey of ongoing research and investigational therapies.

Main Results:

  • Symptomatic treatment is primarily limited to antidopaminergic agents for motor dysfunction and antidepressants for mood disorders.
  • Treatment for cognitive dysfunction in HD remains largely unaddressed.
  • Over 80 agents across various classes have been investigated, with varying degrees of success or limitations.

Conclusions:

  • Effective disease-modifying treatments for Huntington's disease are still needed.
  • Current pharmacologic strategies offer symptomatic relief but do not halt disease progression.
  • Ongoing research targets diverse mechanisms to improve outcomes for HD patients.

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