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Gene therapy could give hope to Parkinson's patients

WebPosted Thu Oct 26 15:14:48 2000

http://cbc.ca/cgi-bin/view?/news/2000/10/26/monkey001026

WASHINGTON - A gene transferred to the brains of monkeys was able to
control symptoms of Parkinson's disease in the animals. Experts say
the results offer "promise" for human patients.

Parkinson's disease is a progressive brain disorder affecting cells
that make dopamine. The cause of the disease is not known, but this
new gene therapy technique prompted the brains of monkeys to produce
more of the brain chemical, thereby diminishing symptoms of the
disease.

The technique uses a virus linked to a gene that triggers production
of dopamine.

In the experiment, a group of Rhesus monkeys were given a drug,
MPTP, that artificially induces Parkinson's disease symptoms. Ten of
the monkeys developed pronounced symptoms.

Five in the group were then given the gene therapy injection into
their brains. Five others received a placebo injection. One monkey
in each group died of unknown causes after being injected.

Of the four remaining monkeys in the gene therapy group, three were
totally relieved of the Parkinson's symptoms. The four control
monkeys continued to show signs of the disease and became "severely
impaired," said Jeffrey H. Kordower, first author on the study.

He said the beneficial effect lasted for at least eight months. The
monkeys were then killed for a detailed analysis of their brains.

The study will appear in Friday's issue of Science.

Cautious optimism

Dr. Ted M. Dawson, head of the Parkinson's Disease Research Center
at Johns Hopkins University Medical Center says while the experiment
shows dramatic effects, he is only cautiously optimistic.

He says there are still questions about safety that would have to be
answered before the gene therapy could be tried in humans.

Dr. Mark H. Tuszynski, head of a brain disorder research centre at
the University of California, San Diego, says there is uncertainty
about whether the brain cells affected by the chemically induced
Parkinson's in monkeys are the same as those affected by the actual
disease in humans.

Kordower said that another study in monkeys is under way to test the
safety of the gene therapy. When that is completed in about six
months, he said an application for human clinical trials would be
prepared for presentation to the U.S. Food and Drug Administration.

But even if all of the studies go smoothly, said Kordower, it could
take three to five years before the gene therapy is ready for
testing in humans.

Links:

Study will appear inScience magazine Friday, October 27, 2000

http://www.sciencemag.org/

The Parkinson Foundation of Canada

http://www.parkinson.ca/

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