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Huntington’s Disease: New Frontiers in Therapeutics

  • Movement Disorders (T. Simuni, Section Editor)
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Abstract

Purpose of Review

This article describes and discusses new potential disease-modifying therapies for Huntington’s disease that are currently in human clinical trials as well as promising new therapies in preclinical development.

Recent Findings

Multiple potential disease-modifying therapeutics for HD are in active development, including direct DNA/gene therapies, RNA modulation, and therapies targeted at aberrant downstream pathways.

Summary

The etiology of Huntington’s disease (HD) is well-known as an abnormally expanded trinucleotide repeat within the huntingtin gene. However, the pathogenesis downstream of the mutant huntingtin gene is complex, involving multiple toxic pathways, including abnormal protein fragmentation and neuroinflammation. The current treatment of HD focuses largely on symptomatic management. This article discusses new, potential disease-modifying therapies that are currently in human clinical trials and preclinical development.

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Authors and Affiliations

  1. Department of Neurology, The Marlene and Paolo Fresco Institute for Parkinson’s and Movement Disorders, NYU Langone Health, 222 East 41st Street – 13th Floor, New York, USA

    Ling Pan & Andrew Feigin

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  1. Ling Pan
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  2. Andrew Feigin
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Correspondence to Ling Pan.

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Ling Pan declares that she has no conflict of interest. Andrew Feigin has consulted for NeuExcell Therapeutics, Voyager Therapeutics and Stealth BioTherapeutics. He has received grant support from Vaccinex, and Prilenia Therapeutics.

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This article is part of the Topical Collection on Movement Disorders

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Pan, L., Feigin, A. Huntington’s Disease: New Frontiers in Therapeutics. Curr Neurol Neurosci Rep 21, 10 (2021). https://doi.org/10.1007/s11910-021-01093-3

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