Damage of Huntington's disease could be slowed down with new drug

Emmett Rice
December 13, 2017

Because it is known that neurons are lost in the brain prior to any symptoms of Huntington's, the long term research focus of this group has been to determine the first biochemical change caused by the gene that leads to this cell death.

Scientists have for the first time corrected a protein defect that causes Huntington's disease, by injecting a drug into the spine, offering new hope for patients with the devastating genetic disease.

The first human trial of a drug for Huntington's disease has shown it to be safe, well-tolerated and successful in lowering the level of huntingtin protein in the nervous system.

It's hoped this form of drug could be adapted to target proteins in other now incurable brain disorders, such as Alzheimer's disease.

Now treatments only exist for the symptoms of Huntington's, which usually begin to appear between the ages of 30 and 50, leading to their gradual decline over the next 10 to 25 years. Huntington's is caused by an abnormality in a section of DNA called the huntingtin gene, which contains the instructions for making the protein huntingtin.

"What we don't know is the possibility of long-term consequences of knocking down or removing both the normal Huntington protein and the protein that carries the mutation".

The progressive neurological disorder, which now affects an estimated 10,000 people in the United Kingdom, is incurable - but an experimental drug, which has been shown to lower levels of the harmful protein responsible, could be a "game-changer".

The trial involved 46 patients with early Huntington's disease at nine study centres in the UK, Germany and Canada.

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"I am a neuroscientist".

A breakthrough procedure that could halt the progress of Huntington's disease is being heralded as potentially the biggest development in neurodegenerative medicine in half a century. "They may just need a pulse every three to four months", she said.

For example, a comparable engineered strand of DNA could be made to focus on the detachment that produces distorted amyloid or tau proteins in Alzheimer's. "One day we want to prevent the disease".

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On the off chance that the future trial is fruitful, Tabrizi trusts the medication could eventually be utilized as a part of individuals with the Huntington's quality before they turn out to be sick, perhaps ceasing side effects regularly happening. "You can target any protein". "I would prefer not to exaggerate this excessively, yet in the event that it works for one, for what reason wouldn't it be able to work for a considerable measure of them?"

Prof Giovanna Mallucci, associate director of UK Dementia Research Institute at the University of Cambridge, described the work as a "tremendous step forward" for individuals with Huntington's disease and their families. There is also hope that the methodology can be applied to other neurodegenerative diseases, such as Alzhiemer's and Parkinson's.

"The case for these is not as clear-cut as for Huntington's disease, they are more complex and less well understood". Most people with Huntington's inherited the gene from a parent, but about one in five patients have no family history of the disease.

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