Therapeutic approaches to Huntington disease: from the bench to the clinic

Nat Rev Drug Discov. 2018 Oct;17(10):729-750. doi: 10.1038/nrd.2018.133. Epub 2018 Sep 21.

Abstract

The 25 years since the identification of the gene responsible for Huntington disease (HD) have stood witness to profound discoveries about the nature of the disease and its pathogenesis. Despite this progress, however, the development of disease-modifying therapies has thus far been slow. Preclinical validation of the therapeutic potential of disrupted pathways in HD has led to the advancement of pharmacological agents, both novel and repurposed, for clinical evaluation. The most promising therapeutic approaches include huntingtin (HTT) lowering and modification as well as modulation of neuroinflammation and synaptic transmission. With clinical trials for many of these approaches imminent or currently ongoing, the coming years are promising not only for HD but also for more prevalent neurodegenerative disorders, such as Alzheimer and Parkinson disease, in which many of these pathways have been similarly implicated.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Clinical Trials as Topic
  • Drug Evaluation, Preclinical
  • Humans
  • Huntingtin Protein / metabolism
  • Huntington Disease / drug therapy*
  • Huntington Disease / metabolism
  • Pharmaceutical Preparations / administration & dosage*
  • Signal Transduction / drug effects

Substances

  • Huntingtin Protein
  • Pharmaceutical Preparations