First Parkinson’s gene therapy patient well after 1 year suggesting that the therapy is safe and effective

Sep 2 2004

The first ever patient to have undergone gene therapy for Parkinson’s appears to have come through phase I without a hitch, suggesting that the therapy is safe and effective, reports Marina Murphy in Chemistry & Industry.

‘We have yet to analyse efficacy data, with the seventh of the planned twelve subjects only just having undergone gene transfer. . . But there have been no adverse effects related to the gene therapy so far,’ said lead researcher Matthew During, of the University of Auckland. He continues, ‘Our therapy is extremely safe and we hope there will be some symptomatic improvement.’

It is just over a year since the first patient, Nathan Klein, had a virus carrying a gene injected in a part of his brain. He claims to have experienced an improvement of 40–60% in overall symptoms when he is on his medication, and a 10–20% improvement when he is not. Prior to the surgery, he habitually suffered from a tremor on his right side.

Roger Barker, a Parkinson’s expert at the University of Cambridge, who was critical of the decision to go ahead with the trial, said the fact that there has been no adverse effects is good news, but because the patient is on low doses of his medication, it is not possible to determine whether the gene therapy is any better than a more aggressive drug regime, or subthalamic nucleus (STN) deep brain stimulation.

The treatment uses a harmless virus to introduce a gene into a part of the brain that is overactive in Parkinson’s patients, causing jerky movements. Introducing this gene leads to the production natural chemicals that inhibit the overactive brain cells. During’s group is the only one conducting human gene therapy trials in Parkinson’s and experts think that gene therapy is 20 years ahead of stem-cell therapy for Parkinson’s disease.

Phase II should start at the beginning of 2005.