FROM: Hospital Business Week
September 26, 2004
SECTION: EDITOR'S CHOICE; Pg. 8
HEADLINE: UNIVERSITY OF AUCKLAND;
First Parkinson gene therapy patient passes 1 year
The first-ever patient to have undergone gene therapy for Parkinson
appears
to have come through phase I without a hitch, suggesting that the therapy
is
safe and effective, according to new research.
"We have yet to analyze efficacy data, with the seventh of the
planned 12
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. "Our therapy is extremely
safe
and we hope there will be some symptomatic improvement."
The new findings were reported in Chemistry & Industry.
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 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 regimen, 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 patients, causing jerky movements.
Introducing this gene leads to the production of natural chemicals that
inhibit
the overactive brain cells .
Phase II should start at the beginning of 2005.
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