An international team led by researchers at the UCL Institute of Neurology has identified a set of tests that could help identify whether - and how - Huntington’s disease (HD) is progressing in groups of people who are not yet showing symptoms.
The latest findings from the TRACK-HD study are published in The Lancet Neurology and could be used to assess whether potential new treatments are slowing the disease up to 10 years before the development of noticeable symptoms.
“Currently, the effectiveness of a new drug is decided by its ability to treat symptoms," says lead author Professor Sarah Tabrizi. "These new tests could be used in future preventative drug trials in individuals who are gene positive for HD but are not yet showing overt motor symptoms.
"These people have the most to gain by
initiating treatment early to delay the start of these overt symptoms and give
them a high quality of life for a longer period of time."
Writing in a linked Comment in The Lancet Neurology, Professor Francis Walker from Wake Forest
Medical School in the USA says that the TRACK-HD investigators have set the
standard for observational studies in other neurodegenerative diseases. He says: “Virtual roadmaps of disease in the
minds of practitioners are good for care in the framework of the traditional
patient encounter, but it takes substantial effort, teamwork, and genius to
turn them into rigorous, quantifiable timelines that can be used to test
efficacy in future therapeutic trials.”
Sarah J Tabrizi et al. Predictors of phenotypic progression and disease onset in premanifest and early-stage Huntington's disease in the TRACK-HD study: analysis of 36-month observational data. The Lancet Neurology. Available online 9 May 2013. DOI: 10.1016/S1474-4422(13)70088-7
Francis O Walker. Huntington's disease: the road to progress
The Lancet Neurology. Available online 9 May 2013
DOI: 10.1016/S1474-4422(13)70105-4