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'Ground-breaking' new discovery in search for Huntington's cure

Article By: Michaela Chirgwin

A ‘ground-breaking’ new drug trialled by University College London (UCL) is thought to be one of the biggest finds in the treatment of neurodegenerative disease in a generation.

Credit: University College London

In the trials, an experimental drug was injected into the spine of Huntington’s disease patients to prevent the progression of the condition.

Researchers found the drug managed to reduce the amount of the protein which causes the disease.

Although the discovery is not being called a cure (a larger test and more data analysis is required) the ‘breakthrough’ could have a massive impact on those who have been diagnosed with the debilitating condition.

The successful trial results are also likely to have big implications for the future treatment of other neurodegenerative diseases such as Parkinson’s and Alzheimer’s.

Professor Tabrizi, director of the UCL Huntington’s Disease Centre said: “The results of this trial are of ground-breaking importance for Huntington’s disease patients and families.

“For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated. The key now is to move quickly to a larger trial to test whether the drug slows disease progression.”

The trial involved 46 patients, male and female, with early Huntington’s disease at nine study centres in the UK, Germany and Canada. The procedure itself was carried out at the Leonard Wolfson Experimental Neurology Centre at the National Hospital for Neurology and Neurosurgery in London.

Each patient received four doses of the new drug or placebo, given by injection into the spinal fluid to enable it to reach the brain. As the phase trial progressed, the dose of the drug was increased several times.

The injections in the trial were aimed at a faulty DNA called the ‘huntingtin gene’. According to the UCL research team, the injection “produced significant, dose-dependent lowering of the level of mutant huntingtin; the first time the protein known to cause Huntington’s has been lowered in the nervous system of patients.”

According to the Huntington's Disease Association (HDA), Huntington’s is a congenital degenerative condition caused in a person’s DNA. It affects the body’s nervous system – the network of nerve tissues in the brain and spinal cord that co-ordinate the body’s activities.

There are currently 6,000-10,000 individuals in the UK who are thought to have the symptoms of Huntington’s; it usually develops between the ages of 30 and 50, but can start at any age.

The HDA website states that “Huntington’s is not something you can catch; it is inherited. Every child conceived naturally to a parent who carries the Huntington’s gene has a 50 per cent chance of inheriting it.”


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