Chinese School - New treatment promising for Parkinson's

WORLD / Health

New treatment promising for Parkinson's

(AP)
Updated: 2007-06-22 10:34

NEW YORK - An experimental treatment for Parkinson's disease seemed to
improve symptoms - dramatically so, for one 59-year-old man - without
causing side effects in an early study of a dozen patients.

The gene therapy treatment involved slipping billions of copies of a gene
into the brain to calm overactive brain circuitry.

The small study focused on testing the safety of the procedure rather
than its effectiveness, and experts cautioned it's too soon to draw
conclusions about how well it works. But they called the results
promising and said the approach merits further studies.

"We still have quite a bit more testing to do," said Dr. Michael Kaplitt
of Weill Cornell Medical College in New York, an author of the study.
Still, "the initial results are extremely encouraging."

Kaplitt and collaborators report their results in this week's issue of
the British medical journal, The Lancet.

They're not alone in trying gene therapy for Parkinson's. In April,
another team told a medical meeting that its experiments, which delivered
a different kind of gene to a different part of the brain, also appeared
safe and gave a preliminary hint of benefit.

More than half a million Americans have Parkinson's. They endure symptoms
that include tremors, rigidity in their limbs, slowness of movement and
impaired balance and coordination. Eventually they can become severely
disabled.

Nathan Klein, a 59-year-old freelance television producer in Port
Washington, N.Y., said the disease left him "pretty messed up." It
weakened his voice, impaired his walking and made his hand tremble so
badly he couldn't hold a glass of wine without spilling it all.

Klein was the first patient to be treated with Kaplitt's gene therapy
procedure in 2003, and he said his symptoms gradually subsided afterward.
Nowadays, he said, apart from freezing now and then when he wants to
walk, the symptoms are basically gone.

"I'm elated," said Klein, who continues to take his regular pills for the
disease. "It's unbelievable."

Kaplitt, who has a financial interest in Neurologix Inc., which paid for
the research, noted that the 12 patients in the study still have
Parkinson's symptoms. The amount of medication they were already taking
for their symptoms didn't change significantly in the year after the
surgery.

Current medicines can control symptoms, but can't stop the disease from
getting worse over time, and they can produce troublesome side effects
like uncontrollable movement.

Some patients gain relief from a surgical treatment called deep brain
stimulation, in which electrodes are placed in the brain and connected to
a programmable stimulator.

Kaplitt's procedure was aimed at achieving the same goal as that surgery,
calming overactive circuitry in the brain. It gets overactive because it
loses the normal supply of a chemical called GABA. The gene therapy was
designed to make the brain produce more GABA.

For the gene therapy surgery, a tube about the width of a hair was
threaded through a hole about the size of a quarter at the top of the
skull. The tube delivered a dose of a virus engineered to ferry copies of
a gene into cells of a brain region called the subthalamic nucleus. The
gene copies enable the cells to pump out more GABA.

The Lancet paper reports that over a year, patients showed no side
effects from the procedure. What's more, they showed improvements in an
overall assessment of symptoms like tremors, stiffness and walking
problems.

The improvements were evident at a checkup three months after the
procedure and persisted to the end of the study, one year after the
surgery, researchers reported. By that time, the overall amount of
improvement from before surgery was about 24 percent when measured at
times that patients were off their normal medication, and 27 percent at
times when they were on medication.

Most of the effect appeared on just one side of the body. Because of
concerns about safety with the untested procedure, the researchers
treated only the brain circuitry controlling one side of the body.

Dr. Karl Kieburtz of the University of Rochester Medical Center, who
didn't participate in Kaplitt's work, said the lack of any apparent side
effects is itself significant.

But he urged caution in interpreting the evidence of benefits in
symptoms. Other experimental therapies that looked good at such a
preliminary stage have failed to pan out in more rigorous studies, he
said, so more research is needed.

Future studies could include a head-to-head test against deep brain
stimulation to see which relieves symptoms better, said neurosurgeon Dr.
Guy M. McKhann of the Columbia University Medical Center in New York.

Dr. J. Timothy Greenamyre of the University of Pittsburgh, who was also
familiar with the results, said the new study and prior research in
animals leave him "very optimistic" about Kaplitt's approach.

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