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Breakthrough in Parkinson's gene therapy
By Roger Highfield, Science Editor
Last Updated: 10:01pm GMT 19/11/2007
Evidence that a breakthrough has been achieved in gene therapy for serious brain diseases has come with the release of the hard evidence that it works in Parkinson's disease.
Patients were given injections of billions of copies of genetically altered viruses into parts of the brain
The world's first gene therapy for a brain disease brought about significant improvements in the mobility of Parkinson's sufferers. American doctors said it could also herald a landmark in the treatment of other neurological disorders, such as Alzheimer's or epilepsy but there was a lingering doubt that the reports by a dozen patients of improvements of up to 65 per cent in mobility could be anecdotal or due to the placebo effect.
Today, Prof David Eidelberg of the of The Feinstein Institute for Medical Research in Manhasset, New York, and colleagues report in the Proceedings of the National Academy of Sciences evidence that the brain chemistry of the patients has been altered by gene therapy, ending concerns that the evidence that it worked depended too much on what the patients said and not enough on objective measures. "It is the first solid evidence of benefit from gene therapy. It is objective," Prof Eidelberg told The Daily Telegraph.
Parkinson's affects about 120,000 people in Britain, with 10,000 new cases diagnosed every year. It robs sufferers of the ability to walk and even eat, causes long motionless periods known as "freezing" as well as head and limb tremors. As the disease progresses, higher doses of drugs are required, leading to side-effects that include involuntary movements.
Sufferers include the former world boxing champion Muhammad Ali and the actor Michael J Fox.
advertisementThe research team, from the New York-Presbyterian Hospital/Weill Cornell Medical Centre and the New Jersey-based company Neurologix, used the gene therapy to fix some of the damage caused by the disease.
Parkinson's occurs when the brain cells - neurons - that release the messenger chemical known as dopamine die. Protein deposits also form in the brain, and levels of another messenger chemical called GABA - which calms overexcited brain cells - drop.