Research into Huntington’s Disease is being driven forward by a £3.3m investment, in a project hailed as giving the first ‘realistic chance’ of finding treatments to slow down its progression.
The five-year study will help advance knowledge of disease mechanisms in HD and identify robust outcome measures for future disease-modifying clinical trials.
This ambitious project will advance current understanding of mechanisms underlying HD and exploit this knowledge for therapeutic development.
The work, funded by a Wellcome Trust Collaborative Award, will not only give hope to the HD community but will provide valuable insights into other neurodegenerative diseases.
“For the first time we have a realistic chance of finding treatments which actually slow down the disease process,” says Professor Sarah Tabrizi, principal investigator of the Wellcome Centre for Human Neuroimaging’s Huntington’s Disease team and director of UCL’s Huntington’s Disease Centre.
“Ideally we aim to administer the most successful therapies to gene carriers years before widespread damage has occurred, with the hope that we can prevent or at least significantly delay disease onset.”
This new project will probe disease mechanisms at the molecular, cellular and systems level and will investigate instability within the HD gene which is known to drive disease progression.
Mechanisms underlying the gene’s effect on molecular processes will be elucidated within cells and in mouse models of HD.
Ongoing collaborations with industrial partners, Roche and Ionis Pharmaceuticals, will allow assessment of the potential therapeutic benefit of drugs directly targeting these pathways.
The study is being led by researchers from UCL, the University of Cambridge and University of Glasgow.
While ongoing trials currently seek to modify the disease in symptomatic patients, the HD Centre’s HD Young Adult Study has shown that the earliest signs of neuronal damage occur up to 24 years before the expected onset of clinical symptoms; this is likely to be the optimum time to intervene with treatments to slow or halt disease progression.
It will use advanced imaging techniques including 7T and magnetoencephalography to examine brain structure and function in far-from-onset gene carriers.
Professor Michael Hanna, director of UCL Queen Square Institute of Neurology, adds: “I am delighted that Sarah and her colleagues have been awarded this important grant from the Wellcome Trust which fits perfectly with the translational neuroscience strategy of the ION and will fund fundamental mechanistic research into finding and testing prevention therapies for Huntington’s disease.”
