Drug breakthrough gives patients hope of halting Alzheimer’s

SCIenTISTS are hoping for a potential cure for Alzheimer’s after discovering a drug that may correct another deadly brain disease.

The experimental treatment has been hailed as the biggest breakthrough in neurodegenerative disease for half a century. Injected into spinal fluid, the drug appears to silence the faulty gene that causes huntington’s – a crippling illness that devastates families.

The human trials, which were carried out by university College London, left patients with reduced levels of a protein that kills off brain cells.

The researchers are already trialling the same drug in Alzheimer’s sufferers to see whether it can also target the tangles of a specific protein that causes memory loss.

‘This is, potentially, the biggest breakthrough in neurodegenerative disease in the past 50 years,’ said John hardy, one of the world’s leading dementia experts.

‘We have treatments to treat some of the symptoms of dementia, Parkinson’s disease and huntington’s, but we have never before been able to prevent the diseases killing cells in the brain.’

Professor hardy, who was not involved in the research, added: ‘This is fantastic for the thousands of people with huntington’s, but the real prize is whether similar approaches can work for other diseases.

‘If the approach will work for one brain disease, why shouldn’t it work for the others which are similar? That is what the next step will determine.’

Many people do not realise they have huntington’s disease until symptoms of personality changes and jerky movements appear between the age of 35 and 55. The disease, which kills brain cells affecting mobility, behaviour and memory, can kill sufferers within ten to 25 years.

Those who watch their relatives die face the knowledge that they have a 50/50 chance of developing the disease themselves.

It has taken 15 years to find a huntington’s drug ready to be tested on patients, who each carry a faulty gene making a protein in the brain become ‘misfolded’ and ‘sticky’.

The protein forms clumps,

‘Groundbreaking importance’

similar to those seen in people with Alzheimer’s, which kill brain cells. But the new drug appears to tackle the protein by killing the ‘messenger’ – the blueprint for the killer protein that comes from the faulty gene and is contained in a short strand of genetic code.

experts stress that a potential cure is many years away. however the lower levels of the protein, called ‘huntingtin’, seen in the spinal fluid of the study participants have excited experts.

Within 15 years it is hoped people who carry the huntington’s gene could be prevented from ever developing the disease. Alzheimer’s and Parkinson’s disease, like huntington’s, are caused by the production of a mutant protein, or the overproduction of a normal one.

A drug that could reduce the amount of these damaging proteins could slow or stop these illnesses also.

Professor Sarah Tabrizi, head of uCL’s huntington’s disease centre, said: ‘The results of this trial are of groundbreaking 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 drug was tested on 46 patients. Fears that injecting it into the spine, which is a long way away from the brain, might not help tackle disease have been dispelled by the drug trial.

researchers had also been concerned that a change to spinal fluid could trigger deadly meningitis in the brain.

however, while people within the study saw their protein levels fall, it is too soon to tell how this affected their symptoms of huntington’s. Full details of the trial are due to be presented at scientific meetings and published in a journal next year.