A new surgical gene therapy for Parkinson's disease appears to be producing improvements for the first human patients.
The treatment, performed under local anaesthetic, involves boring a hole through the skull to deliver the genes in several drops containing a safe virus. Seven patients have so far been treated in
the trial at New York Presbyterian Hospital.
The trial that began last year involves 12 patients with severe Parkinson's disease, for whom current therapies have stopped working.
Auckland University scientist Professor Matthew During, who led development of the gene therapy, said yesterday: "There are anecdotal reports of improvement. There's real promise.
"But we'll have to wait until we finish the study before making any official statement on efficacy.
"Even though it's not a blinded trial, we want to analyse it as a group. There's a lot of hype about gene therapy."
Professor During, who divides his time between Auckland and New York, was in Queenstown yesterday to talk to the Medical Sciences Congress about gene therapy in Parkinson's disease.
It affects about 7000 New Zealanders and is characterised by trembling, rigid posture, slow movements and a shuffling gait. It is caused by the loss of the nerve cells that make dopamine, a chemical messenger.
The trial therapy delivers a gene called GAD in a bid to calm certain brain cells whose overactivity produce the disease's symptoms.
In tests on rats, the treatment produced a 75 to 80 per cent reduction in Parkinson's symptoms. There was also evidence it might stop or delay the disease's progression.
