Source: The Lancet/The Lancet Neurology Press Release
TRACK-HD investigators, including UCL Institute of Neurology researchers have identified a set of objective, validated measures that can be used to assess new treatments for Huntington’s disease (HD). The study, published last week in The Lancet Neurology, should increase the likelihood of success of future trials of new drugs to delay the onset and reduce the severity of HD.
The TRACK-HD study was established in 2007 to identify measures (biomarkers) that could be used to more accurately measure and predict the progression of HD, by comparing a series of techniques to assess motor function, behaviour, cognition, and advanced brain imaging. The study enrolled 366 individuals from Canada, France, the Netherlands, and the UK—120 presymptomatic carriers of the HD gene mutation, 123 patients with early symptomatic HD, and 123 healthy controls.
TRACK-HD investigators had previously shown that presymptomatic HD patients monitored over 12 months had a progressive reduction of white matter and whole-brain volume, and cognitive and motor function decline. This current study, reports on the 24 month follow-up of patients with the goal of establishing the ability of these measures to track disease progression when used annually and to calculate their effect sizes, to assess potential sensitivity and efficiency.
The researchers found that measures derive from brain imaging techniques were the most effective and detecting disease progression over 24 months. Measures of brain atrophy increased at a significantly higher, measurable rate, in both individuals without noticeable symptoms and those with early HD, and provide the strongest outcome measure for both stages of the disease.
Lead author, UCL Institute of Neurology, Professor Sarah Tabrizi comments: “HD research is at a critical point, with new drugs in the later stages of development, and we propose a battery of assessments for use in clinical trials in people with early HD. Hypothetical treatment effects defined by slower longitudinal changes in these measures should be detectable over a realistic timescale with practical sample sizes. These new tools provide a key contribution towards our ultimate aim of establishing effective treatments for this devastating condition.”
She concludes:“Future studies to investigate the validity of the markers as indicators of clinically meaningful and potentially reversible progression will advance the development of treatments for this devastating neurodegenerative disease.”
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