Breakthrough in Parkinson's Disease on Horizon

Dr Kaplitt and his colleagues [at the Weill Cornell Medical Centre] targeted this problem by injecting a gene called glutamic acid decarboxylase into the subthalamic region of the brain.

"Our hope was that with a single operation to this single site, we could boost Gaba production and thereby normalise the function of the entire circuit," Dr Kaplitt said.

The scientists genetically modified a virus that had been rendered harmless to carry the human gene into the brain cells. They injected only one side of the brain andused this to compare the effects of the therapy with the untreated hemisphere.

Standard methods of monitoring Parkinson's patients showed they fared better as a result of the treatment, with a 25-30 per cent improvement when they were not talking their usual medication, and a 40-65 per cent improvement when they were talking their drugs.

"We have discovered a compelling new therapeutic approach for Parkinson's disease, which we expect will allow our scientists -- as well as those at pharmaceutical and biotech companies -- to pursue innovative new drugs that will treat and perhaps even cure this disorder," says Aleksey Kazantsev, PhD, director of MGH-MIND Drug Discovery Laboratory, who led the Science study. "Since the same sort of aggregation of misfolded proteins has been reported in Huntington's and Alzheimer's diseases - as well as Lewy body dementia, which also involves alpha-synuclein deposits - we plan to test this approach in those conditions as well."