Scientists to develop life-transforming drugs for Huntington’s disease sufferers

Scientists at the University of Sussex have started work on a multi-million pound project to develop drugs to treat the debilitating loss of cognitive function associated with Huntington’s disease.

The uncontrolled movements that characterise Huntington’s disease generally appear in middle age – however, a decline in cognitive function can occur in patients 10-15 years prior to the movement disorders and can have life-changing consequences, such as a loss of employment as well as a loss of the ability to drive.

Currently there are no drugs on the market which treat these symptoms – but now scientists from the University’s Sussex Drug Discovery Centre (SDDC) have been awarded £3 million from the Wellcome Trust to develop drugs which could help to transform the lives of Huntington’s disease sufferers around the world.

The SDDC team, which is a part of the University’s School of Life Sciences, will work to identify and develop novel compounds which target a specific brain protein known as the alpha5 GABAA receptor – which is already known to control cognitive function. The pharmaceutical company AstraZeneca are involved in the project and have a strong interest in not only the disease but also the alpha5 GABAA drug mechanism.

Scientists from the University of Dundee, who are also key collaborators on this project, have previously shown that an experimental compound, called α5IA, can reverse some of the cognitive deficits observed in a mouse model of this degenerative brain disorder.

Professor John Atack, from the University of Sussex, said: “The earlier symptoms of Huntington’s disease, which cause problems with multitasking, organising and planning, are life-changing for sufferers and often lead to them being unable to carry out everyday tasks such as working and driving.

“By developing a drug which treats these debilitating symptoms we can significantly improve the life quality, not only of the patients themselves, but also of their families and carers.”

Cath Stanley, Chief Executive of the Huntington’s Disease Association, said:Cognitive impairment in Huntington’s disease is in fact the most disabling symptom of the illness and brings with it devastating consequences for both the individual affected and their family.

“Currently there is very little research carried out that involves the development of a drug to improve this part of the condition, so this research has the potential to be literally life changing for individuals.”


Source: University of Sussex

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