Huntington's disease: 'Ground-breaking' new drug gives hope for patients

IONIS-HTTrx Proves Effectiveness in Treating Cause of HD

Huntington's disease: 'Ground-breaking' new drug gives hope for patients

According to Professor Tabrizi, "The results of this trial are of ground-breaking importance for Huntington's disease patients and families". The code is copied by a messenger molecule and dispatched to the cell's protein making machinery.

Huntington's patient Michelle Dardengo, of Vancouver, B.C., watched six members of her family die of the disorder.

Scientists at the University College London (UCL) on Monday announced that for the first time a drug has been used to successfully lower the level of a protein known to cause Huntington's disease in the nervous system of patients.

An worldwide research team led by Professor Russell Snell and Dr Renee Handley from the University of Auckland's School of Biological Sciences, Centre for Brain Research, have identified elevated levels of the chemical urea in the brain of a transgenic sheep model which matches those of human brains affected by Huntington's.

They were each given four spinal injections of either the drug or a placebo. Therefore, the main aim of the UCL study was determining whether the experimental drug - given to the majority of the 46 men and women from the UK, Germany and Canada with early stages Huntington's who took part - is safe.

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This work was supported by Brain Research New Zealand, Cure Huntington's disease initiative, the Neurological Foundation and the Freemasons of New Zealand.

The animal research suggests it would.

"We wouldn't have expected to see any change in rates of progression and certainly don't see any signs of stopping the disease". Some motor function even recovered in those experiments.

Mr Allen added: "I'm the luckiest person in the world to be sitting here on the verge of having that [a treatment]".

"Hopefully that will be made available to everybody, to my brothers and sisters and fundamentally my children". Those with the faulty gene may not have the disease for years, but eventually they will develop symptoms.

"That sounds like hyperbole - in a year I might be embarrassed by saying that - but that's how I feel at the moment".

Levels of the toxic HTT protein are reduced, thereby minimising damage to the brain.

The protein synuclein is implicated in Parkinson's while amyloid and tau seem to have a role in dementias.

The therapy was developed by Ionis Pharmaceuticals, which said the drug had "substantially exceeded" expectations, and the licence has now been sold to Roche. That trial is now being planned.

"The question is whether this is enough to make a difference to patients and their clinical course, and for that we will have to wait for bigger trials", said Roger Barker of the University of Cambridge, who was also involved in the research.

"Alzheimer's and Huntington's are at opposite ends of the dementia spectrum - so if this holds true for these types, then I believe it is highly likely it will hold true for all the major age-related dementias", Cooper said, in the paper published in the Proceedings of the National Academy of Sciences.

She told the BBC: "The case for these is not as clear-cut as for Huntington's disease, they are more complex and less well understood".

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