TREAT-HD: targeting neurodegeneration in Huntington's disease

Huntington’s disease (HD) is a devastating inherited neurodegenerative disease. A faulty gene causes the build-up of a toxic protein – mutant-huntingtin – which damages brain cells, leading to problems with movement, thinking and behaviour. Currently, no treatments slow down the underlying disease process.

We want to improve understanding of the disease process in HD and its response to treatment. We will study a cohort of young adults who carry the faulty gene decades before symptoms begin, to find the earliest changes in the brain or behaviour. We will also link with the first human trial of a drug which effectively ‘silences’ the gene-producing mutant huntingtin using short DNA strands, known as antisense oligonucleotides (ASOs). We want to develop a new generation of treatments based on this ASO approach which target a specific part of the mutant huntingtin that we know is harmful. We will also investigate how early therapies should be administered.

HD shares many similarities with other neurodegenerative conditions such as Alzheimer’s and Parkinson’s disease. Consequently this work may also accelerate the development of treatments not just for HD but also other more common diseases, thereby reducing the public health burden.