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Parkinson's Gene Therapy Seems to Work
Patient: 'If You Didn't Know I Had Parkinson's Disease, You Couldn't Tell'
(continued)
A Pacemaker for the Brain
Brain degeneration in Parkinson's disease overexcites a part of the brain
called the subthalamic nucleus (STN). This leads to the abnormal movements,
tremor, rigidity, and gait problems that make life miserable for Parkinson's
patients.
The therapy created by During and Michael G Kaplitt, MD, PhD, attaches the
gene for a chemical messenger called GAD to a harmless virus. After infusion
into the STN via a thin needle, this genetically engineered virus gets into
brain cells and makes them send out GAD signals. It's a signal that tells
the brain to calm down.
It doesn't work right away. It takes time for GAD levels to build up.
"Nothing happened for the first week. And then nothing happened for the
second week. And then a month, and two months, and then at three months I
thought I was a little better, nothing much," Klein says. "It was like
watching grass grow. But about six months later, I started feeling a lot
better."
During says that Klein's movement problems got about 40% better after
treatment. Not all patients have done that well. But those who got the
highest dose tended to get the most improvement.
One year after treatment, nine of the 12 patients showed an average
improvement of 37% on a measure of Parkinson's disease severity. Five
patients had between 40% and 65% improvement.
"We are encouraged the results seem to be maintained over time," During
says. "When we look at the other side of the brain, the side we did not
treat, we always see worsening of disease. This is a progressive disease. If
we hadn't done the treatment, we would expect these patients to be doing
significantly worse."

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