NEW ZEALAND: First Human Test Of New Parkinson's Treatment

[Murray Charters <mcharters@xxxxxxxxxxxxxx>]

The New Zealand Herald, New Zealand

First human test of new Parkinson's treatment

14.08.2003
By MARTIN JOHNSTON

An American with severe Parkinson's disease will on Monday be the first person 
to have brain surgery with a promising
gene therapy developed by a New Zealand-United States team.

The brain of the 55-year-old television producer from Long Island will receive 
several drops of the liquid therapy at
New York Hospital.

It contains a gene to calm certain brain cells whose overactivity produce the 
disease's symptoms.

In animal trials it also appeared to halt the destruction of the nerve cells 
that make dopamine - the process that
causes Parkinson's. Dopamine is a message-carrying chemical important for 
movement.

The research leader, Professor Matthew During, who divides his time between 
Auckland University and New York's Columbia
University, said he was optimistic the treatment would help reduce the man's 
symptoms.

"It takes about a month for the gene to turn on. That's when we would expect to 
see some benefit."

Parkinson's, a progressive disease, affects about 7000 New Zealanders. It is 
characterised by trembling, rigid posture,
slow movements and a shuffling, unbalanced gait. It is caused by the loss of 
dopamine-making cells.

The New York trial will involve 12 people with severe Parkinson's for whom 
current therapies have stopped working.

It follows promising tests on rats, in which the treatment produced a 75 to 80 
per cent reduction of symptoms. There
was also evidence it might stop or delay the disease's progression.

Professor During's plan to give the treatment to some New Zealanders has 
received a setback, but he still hopes part of
a yet-to-be-approved second trial can be run in the country. It could start in 
2005.

The liquid therapy was being made at his team's Auckland laboratory until the 
Health Ministry ordered a halt after the
US Food and Drug Administration approved its trial use in humans. Now only the 
bulk material was made at Auckland and
it had to be purified in the US.

He said the ministry threatened prosecution if his laboratory made the therapy 
for human use without a drug
manufacturing licence, even though it was only a trial. The FDA required such a 
licence only for commercial production.


"The laws here are somewhat draconian. People are more risk-averse," said 
Professor During, known for pushing
boundaries.

In 1996 he was criticised over breakthrough, experimental Auckland Hospital 
operations that treated two American
infants with Canavan disease, a rare and fatal brain disorder, using synthetic 
genes he and his US team had developed.
The work was later approved by an Auckland ethics committee.

Ministry senior medical adviser Dr Stewart Jessamine said New Zealand adopted 
international guidelines on medicine
manufacturing last year. They stipulated that clinical-trial medicines must be 
made in licensed facilities.

On Monday, neuro-surgeons will deliver the therapy to the Parkinson's patient's 
sub-thalamic nucleus, a walnut-sized
part of the brain extremely overactive in people with the disease.

The man, who will be under local anaesthetic, will have a small hole bored in 
his head. A hollow needle will be guided
to the exact spot by magnetic resonance imaging and by monitoring the 
electrical impulses that vary around the brain.
The electrical probe will be withdrawn from the needle and replaced by a 
catheter to convey the tiny amount of liquid.

It will carry several billion particles of a safe virus each containing a gene 
called GAD. The gene makes molecules of
GABA - a substance released by nerve cells to calm overactivity in the brain - 
which restores greater control of body
movement.

Professor During said the treatment promised a significant advance, but was not 
a cure.

It mirrored another, more-invasive therapy, which calmed overactive cells 
through a battery-powered electrode
surgically implanted in the brain. But at US$25,000 ($42,450) the gene surgery 
cost less than half as much.

SOURCE: The New Zealand Herald, New Zealand




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