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Published on: October 10, 2013
by Sky News:
Scientists are hailing a landmark British study which has paved the way for a pill that can cure brain diseases such as Alzheimer’s and Parkinson’s by stopping the death of neurons.
The Medical Research Council (MRC) team focused on the root cause of many degenerative brain diseases – abnormally shaped proteins that stick together in clumps and fibres.
When enough misshapen protein builds up in the brain it can trigger a reaction that results in the death of nerve cells. Other approaches have sought to stop or limit the accumulation of the abnormal protein, whose structure is folded the wrong way.
But this research, published in the journal Science Translational Medicine, targeted the harmful way brain cells react to misfolded proteins. Using a drug injected into the stomachs of mice through a mouth tube, 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.
During the study, a neurodegenerative disease caused by abnormal prion proteins was induced in the mice.
Prion diseases, which include Creutzfeldt-Jakob Disease (CJD), are rare in humans but share the same underlying cause – misfolded proteins – as more common conditions such as Alzheimer’s.
Lead scientist Professor Giovanna Mallucci, from the MRC Toxicology Unit, said: “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 in the mice.”
The scientists stress human trials are a long way off and point out that the mice suffered serious side effects, including significant weight loss and raised blood sugar.
But they also believe the research demonstrates in principle the possibility of developing an oral treatment – a pill or swallowed liquid – that can protect the brain from neurodegenerative disease.
The research is at a very early stage and it could be a decade or more before the medicine is actually developed.
But experts said the findings were highly significant.
Professor Roger Morris, from King’s College London, said: “This is the first convincing report that a small drug, of the type most conveniently turned into medicines, stops the progressive death of neurons in the brain as found, for instance, in Alzheimer’s disease.
“True, this study has been done in mice, not man; and it is prion disease, not Alzheimer’s, that has been cured.
“However, there is considerable evidence that the way neurons die in both diseases is similar; and lessons learned in mice from prion disease have proved accurate guides to attenuate the progress of Alzheimer’s disease in patients.”
He added: “This finding, I suspect, will be judged by history as a turning point in the search for medicines to control and prevent Alzheimer’s disease.”
Professor David Allsop, from the University of Lancaster, said: “Inhibiting this pathway has produced some very dramatic and highly encouraging results in mice infected with prion disease.
“The main caveats of the research, however, are that prion disease is very rare in humans, and it is not yet clear if the same approach will be viable for much more common neurodegenerative conditions like Alzheimer’s disease and Parkinson’s disease.
“More research is needed to determine if this approach is valid for any condition other than prion disease, and also to find ways of getting around these problematic side-effects.”
Picture: Getty Images
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