Neurotherapeutics

, Volume 5, Issue 2, pp 226–236

Neuroprotection for Huntington’s disease: Ready, set, slow

Neuroprotective Approach

DOI: 10.1016/j.nurt.2008.01.003

Cite this article as:
Hersch, S.M. & Rosas, H.D. Neurotherapeutics (2008) 5: 226. doi:10.1016/j.nurt.2008.01.003

Summary

The ultimate goal for Huntington’s disease (HD) therapeutics is to develop disease-modifying neuroprotective therapies that can delay or prevent illness in those who are at genetic risk and can slow progression in those who are affected clinically. Neuroprotection is the preservation of neuronal structure, function, and viability, and neuroprotective therapy is thus targeted at the underlying pathology of HD, rather than at its specific symptoms. Preclinical target discovery research in HD is identifying numerous distinct targets, along with options for modulating them, with some proceeding into large-scale efficacy studies in early symptomatic HD subjects. The first pilot studies of neuroprotective compounds in premanifest HD are also soon to begin. This review discusses the opportunities for neuroprotection in HD, clinical methodology in premanifest and manifest HD, the clinical assessment of neuroprotection, molecular targets and therapeutic leads, and the current state of clinical development.

Key Words

Huntington’s diseaseneuroprotectiondisease modificationneurogeneticsgenetic testingclinical trialspresymptomaticpremanifest
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Copyright information

© The American Society for Experimental NeuroTherapeutics, Inc. 2008

Authors and Affiliations

  1. 1.MassGeneral Institute for Neurodegenerative DiseaseMassachusetts General Hospital, Harvard Medical SchoolCharlestown
  2. 2.Center for Neuroimaging of Aging and Neurodegenerative DiseasesMassachusetts General Hospital, Harvard Medical SchoolCharlestown