Huntington's disease: hope for a cure following 'ground-breaking' trial

Huntington's breakthrough may stop disease

'Urea is natural chemical produced by the body that is normally cleared away in our urine, but this study suggests a build-up of urea in the brain could be involved in the development of Huntington's disease. BBC quoted Sarah Tabrizi, lead researcher, as saying, "I've been seeing patients in clinic for almost 20 years, I've seen many of my patients over that time die..."

Most cases are genetic, but 10 per cent of patients do not have a family history.

In 2013, Lyon made the documentary The Inheritance about the toll this disease has had on three generations of her family.

"It worries me that I am going to change, and people around me are going to be put through what I went through with my father". "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?"

Symptoms like depression and uncontrollable movements typically begin between the ages of 30 and 50. Patients have a 50 per cent chance of passing the disease on to each of their children. Huntingtin protein levels are markers of this disease.

Patients were from the UK, Germany and Canada and roughly a quarter were given a placebo treatment.

But she said the transferability to Alzheimer's treatment is not so "clear-cut".

"I really hoped that something would come up and it has", she said of the drug. "I am very, very excited", he told the Guardian.


An experimental injectable drug from Ionis Pharmaceuticals is one of the first to show potential in treating the root causes of Huntington's disease, a devastating and fatal rare genetic disorder that causes nerve cells in the brain to break down.

The experimental drug, IONIS-HTTRx, was injected into patients' spinal fluid.

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".

In Australia, more than 1,800 people have the disease and approximately 9,000 are at risk, according to Huntington's New South Wales.

But Professor Mallucci, who is the associate director of UK Dementia Research Institute at the University of Cambridge, cautioned it was still a big leap to expect gene-silencing to work in other neurodegenerative diseases. This stage of clinical trial is meant to test the safety of a drug or treatment in humans, so that its effects can be better studied in larger groups.

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.

The results of the present trial are being prepared to be presented at scientific meetings and published in scientific journals. Muted investor response aside, the results (called "groundbreaking" by lead researcher Sarah Tabrizi of the University College London) prompted Swiss pharmaceutical giant Roche to exercise its licensing option for Ionis-HTTRx. But the drug manufacturer Roche is seeking to bring the drug to market and has paid $45 million for development rights. More studies are planned next year.

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