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Landmark British study could lead to Alzheimer's pill.

A LANDMARK British study has raised the prospect of a pill that can treat brain diseases such as Alzheimer's by halting the death of neurons.

The research, performed on sick mice, is at an early stage and it could be a decade or more before any medicine suitable for patients is developed.

But experts say the findings are highly significant, and one predicted that they would be judged by future generations as historic. The Medical Research Council (MRC) team focused on the root cause of many degenerative brain diseases, including Alzheimer's and Parkinson's - abnormallyshaped proteins that stick together in clumps.

When enough builds up in the brain it can trigger a reaction that kills nerve cells.

Other approaches have sought to stop or limit the accumulation of the abnormal protein, whose structure is folded the wrong way. Instead, the MRC researchers targeted the harmful way brain cells react to misfolded proteins.

Using a drug injected into mice, they flipped a cellular switch from 'off' to 'on' to prevent neurons dying.

Five weeks after treatment one group of mice remained free of symptoms such as memory loss, impaired reflexes and limb dragging. They also lived longer than untreated animals with the same brain disease.

The scientists stress that human trials are a long way off and point out that the mice suffered serious side effects, including significant weight loss.

But they also believe the research demonstrates in principle the possibility of developing a pill or liquid that can protect the brain from neurodegenerative disease.

The research, reported in the journal Science Translational Medicine, duplicated previous results achieved by the same team by means of genetic engineering.

Lead scientist Professor Giovanna Mallucci, from the MRC Toxicology Unit at the University of Leicester, said: "Our previous study predicted that this pathway could be a target for treatment to protect brain cells in neurodegenerative disease. So we administered a compound that blocks it to mice with prion disease. We were extremely excited when we saw the treatment stop the disease in its tracks and protect brain cells, restoring some normal behaviours and preventing memory loss. "The fact that we have established that this pathway can be manipulated to protect against brain cell loss means that developing drug treatments targeting this pathway for neurodegenerative diseases is now a possibility."

The experimental drug, known as GSK 2606414, is made by pharmaceutical giants Glaxo-SmithKline.

Expert Professor David Allsop, from the University of Lancaster, said: "Inhibiting this pathway has produced some encouraging results."

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Publication:Western Mail (Cardiff, Wales)
Date:Oct 10, 2013
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