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A new gene therapy for Parkinson's disease does no harm and
may help, paving the way for a clinical trial that could
revolutionize treatment, a small new study shows.
There's evidence that the treatment works, but it will take
larger clinical trials to prove the treatment really helps
patients. "Some patients did dramatically better. After one
year, 10 of the 12 patients were better than they were at [the
beginning]," says Michael Kaplitt, MD, PhD.
Gene Therapy for Parkinson's The gene therapy calls for
injection of a genetically engineered virus deep into the brain
-- into a region called the subthalamic nucleus or STN. During
Parkinson's disease, the STN becomes wildly overactive. The
virus gets into brain cells and causes them to make a chemical
signal that calms the STN.
Parkinson's disease affects both sides of the brain. But for
safety's sake, regulators insisted
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that Kaplitt and colleagues
test the gene therapy on only one side.
The result: Improvements were seen on the side of the body
controlled by the treated part of the brain. Improvement began
three months after treatment and lasted for a year in most
patients.
Nobody threw away their Parkinson's medications. But the
patients who did best had fewer Parkinson's symptoms, even in
the "off" state between doses of medication.
This proves nothing, Kaplitt is quick to say, because untreated
Parkinson's patients fluctuate wildly. A larger study -- in
which both sides of patients' brains will be treated -- is
likely to start later this year.
"Gene therapy is coming from the fringe to the mainstream. It is
good for patients and their families to know this is emerging now." For Kaplitt, it's the exciting culmination of years of
research.
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"The world is changing for patients with neurodegenerative
diseases," Kaplitt says. "If they have early or even moderate
disease, over the course of their illness this and other things
coming down the line hold promise -- not just for future
generations, but for them in their disease."
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To read the complete article on this research
project and to learn more abut Parkinson’s please visit
WebMD.com
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