Biomarker Development for Huntington's Disease

Huntington's disease (HD) is a fatal inherited neurodegenerative disorder, treatment to slow the progression of which has not yet been found. Human clinical trials to test a number of therapeutic strategies are underway or imminent, facilitated in part by the recent development of biomarkers that might be used as surrogate endpoints in such trials. However, although much progress in developing HD biomarkers has been made, ongoing work seeks to improve the sensitivity and reliability of current measures, and to demonstrate that they correspond to clear meaningful benefit to patients. Of particular importance is the identification of state biomarkers that can be used in pre-manifest HD gene carriers to test therapies hoped to delay symptom onset in these individuals. Functional, neuroimaging and biochemical biomarkers continue to be investigated for use in the development of disease-modifying treatments of HD.