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Breakthrough drug could prevent Huntingtons disease

Wednesday 13th December 2017
UK-based scientists have developed a new drug that could help to prevent the onset of Huntingtons disease.
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Scientists are hailing a new drug that has shown promise in potentially helping to prevent the onset of the degenerative condition Huntington's disease, which they are hailing as a major breakthrough.

Experts at University College London believe that they have created a drug that is able to stop the development of the genetic condition, which could provide hope to thousands of people throughout the UK.

Huntington's disease occurs in people who have a DNA error in their huntingtin gene. The role of this gene in healthy individuals is to create the protein huntingtin, which is essential for brain development.

However, in those with the genetic error, the protein is corrupted and attacks the brain cells instead, leading to significant cognitive decline. In the past, Huntington's disease has been likened to a cross between Parkinson's disease, Alzheimer's and motor neurone disease, with symptoms of all three often presenting themselves in patients.

The newly developed drug is designed to stop this error from attacking the brain, therefore preventing the onset of the degenerative disease. A trial involving 46 patients produced positive results, with no adverse side effects.

Currently, around 8,500 people in the UK are living with Huntington's disease, with an additional 25,000 expected to develop it as they get older. It most commonly affects people in their 30s or 40s, with the average life expectancy following the first appearance of symptoms between ten and 20 years.

But by treating those known to be at risk of the condition with the new drug, the disease's onset could potentially be stopped, providing thousands of people with a lifeline.

Speaking to BBC News, Professor John Hardy, winner of the Breakthrough Prize for his research into Alzheimer's disease, stated: "I really think this is potentially the biggest breakthrough in neurodegenerative disease in the past 50 years."

Meanwhile, Professor Giovanna Mallucci, associate director of the UK Dementia Research Institute at the University of Cambridge, added: "The principle that a gene, any gene affecting disease progression and susceptibility, can be safely modified in this way in humans is very exciting and builds momentum and confidence in pursuing these avenues for potential treatments."

Written by James Puckle

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