UK scientists hail ‘ historic’ turning point on Alzheimer’s
A BREAKTHROUGH by British scientists has been hailed as a turning point in the fight against Alzheimer’s disease. The landmark study also raises the prospect of new drugs for other brain conditions, including Parkinson’s and motor neurone disease. It may even be possible to create one pill that treats several diseases.
The work is still at an early stage but is described as the first real evidence that it is possible to use a drug to stop diseased brain cells from dying.
Professor Roger Morris, of King’s College London, said: ‘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.’
In Alzheimer’s disease, which affects 500,000 Britons, brains are clogged up by a sticky, ‘misfolded’ protein. Most scientists are trying to find drugs that break up the toxic protein and clear it from the brain, but the latest study, from scientists funded by the Medical Research Council, tackled the problem in a different way.
Rather than trying to declog the brain, they looked at how mis-folded proteins damage it. In much the same way as a deckchair needs to be opened and closed in a certain way, the proteins in our cells have to be folded in a certain way if they are to work properly.
Research on mice showed that mis- shapen proteins do not directly kill brain cells. Instead, cells die because in a misguided attempt to protect themselves, they stop making new proteins – including some vital for their survival.
The researchers showed it was possible to give mice a drug that switches protein production back on, and stops brain disease in its tracks.
Treated mice stayed symptomfree, while untreated animals developed memory and movement problems and eventually died, the journal Science Trans- lational Medicine reports. Lead scientist Giovanna Mallucci, of the University of Leicester, said: ‘It was very striking. The treated mice were completely protected. More importantly, their brains were completely protected.’
The tests were on the misfolded brain protein called a prion which causes scrapie, a fatal, degenerative disease that affects the nervous systems of sheep and goats. But it is thought a similar process is
‘Wide-reaching
benefits’
behind several other brain diseases, including Alzheimer’s, motor neurone and Parkinson’s diseases.
However, the drug had severe side-effects and any new treatments for people are at least a decade away.
Prof Morris 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 in man; and it is a prion disease, not Alzheimer’s, that has been cured.
‘But there is considerable evidence that the way neurons die in both diseases is similar.’
Professor Hugh Perry, chairman of the MRC’s neuroscience and mental health board, said: ‘Despite the toxicity of the compound used, this study indicates that, in mice at least, we have proof of principle of a therapeutic pathway t hat can be targeted.
‘This might eventually aid the development of drugs to treat people suffering from dementias and other devastating neurodegenerative diseases.’
Dr Eric Karran, of Alzheimer’s Research UK, said: ‘Targeting a mechanism relevant to a number of degenerative diseases could yield a single drug with widereaching benefits.
‘However, what is true in animals does not always hold true in people and the ultimate test for this compound will be to see whether it is safe and effective in people with these diseases.’