May have potential to halt or even reverse disease
A new gene therapy tested at Rush University Medical Center reduced
Parkinson's disease symptoms by a remarkable 40 percent, researchers
announced Tuesday.
Researchers cautioned that the study at Rush and another center
involved only 12 patients, and was potentially biased by the placebo
effect.

However, if the preliminary results hold up, gene therapy could
become the first treatment with the potential to slow, halt or even
reverse the course of the progressive disease. Existing treatments
relieve symptoms, but do not prevent the disease from getting worse.

Results were announced at a meeting in Chicago of the American
Neurological Association. The Michael J. Fox Foundation for
Parkinson's Research is encouraged enough to donate $1.9 million for
a follow-up study.

Cells produce dopamine

Between 500,000 and 1.5 million Americans have Parkinson's disease,
which is characterized by rigid and slow movements and tremors. The
disease results from the death of brain cells that produce a
critical brain chemical called dopamine. Symptoms worsen as more
cells die.

In the new therapy, a brain surgeon drills two nickel-size holes in
the top of the head. The surgeon inserts a needle deep into the
brain and injects a harmless virus containing the desired gene.
(Following surgery, the skull holes are plugged with plastic caps.)

The virus delivers the gene to brain cells. The gene then instructs
the cells to produce a protein that protects and regenerates cells
that make dopamine, said Rush neurologist Dr. Leo Verhagen.

Risk of hemorrhage, stroke

The developer, Ceregene Inc., is testing similar gene therapies for
Alzheimer's disease and Lou Gehrig's disease. The surgery involves a
slight risk of hemorrhage and stroke, but that didn't happen to any
patient in the study.

The operation and two-day hospital stay costs $40,000 to $50,000,
said Rush brain surgeon Dr. Roy Bakay. This doesn't count the gene
injection, which also will be costly, Bakay said.

Four patients at Rush, and eight at the University of California,
San Francisco, had the surgery.

Parkinson patient Robert Lee of Orland Park said that before his
surgery last February, he couldn't walk more than a block or two
without excruciating nerve pain in his leg. Now, he can walk three
miles. He also has been able to cut back on his Parkinson meds.

"This gives you some promise and some hope," said Lee, 59. "The
situation I was in was pretty hopeless."

Some patients to just get holes in head

The placebo effect frequently complicates medical research: A
patient who has faith in an experimental treatment will improve,
even if the treatment is worthless. In drug studies, therefore,
researchers typically give an experimental drug to some volunteers
and an inactive placebo pill to others.

The drug is deemed effective only if it works better than the
placebo.

Researchers will use a similar approach in a planned study on a new
gene therapy surgery for Parkinson's disease.

Researchers are enrolling 51 patients at Rush University Medical
Center and seven other centers. Thirty-four patients will receive
the gene therapy. The other 17 patients will have holes drilled in
their skulls, but won't receive the genes.

To prevent bias, neither the patients nor the researchers who
evaluate them will know who received the fake surgery and who got
the real thing.

If the procedure proves effective, patients who received the fake
surgery will later be offered the real surgery for free.

Jim Ritter
October 11, 2006`(Chicago Sun -Times) -