The 55-year-old television producer was up and about, and in
wise-cracking good spirits on Wednesday, at a press conference
at New York-Presbyterian Hospital, where surgeons performed the
risky five-hour brain operation on Monday.
"It feels good to get all this attention. I've never been on
this end of the camera before," said Klein, who helped produce
the syndicated television program "Everyday with Joan Lunden"
in the early 1990s.
Klein said he felt no better or any worse than before the operation,
during which Dr. Michael Kaplitt delivered gene therapy droplets
to the target area of his brain through an opening in his skull.
The droplets contained countless copies of a normally occurring
gene, called GAD, that doctors hope will begin producing its designated
protein. The protein, in turn, is meant to produce a molecule
called GABA, whose role in the brain is to calm overexcited nerve
cells.
The genes are unable by themselves to enter brain cells. So
each copy was stuffed into a seemingly harmless virus called the
adeno-associated virus, which can penetrate human cells and drop
off its gene cargo.
Parkinson's, which affects 1.5 million Americans, is a progressive
disorder in which damage to nerve cells in a deep part of the
brain eventually causes muscle shaking or rigidity, poor coordination
and difficulty in walking.
The nerve damage disrupts production of GABA as well as a brain
messenger chemical called dopamine that sends nerve signals to
muscles.
"GABA normally acts as a brake to control firing of neurons,
but the firing becomes extremely rapid" among people with Parkinson's,
said Dr. Matthew During, a medical professor at the University
of Auckland who helped conduct earlier animal trials of the gene
therapy technique.
The hope is the transplanted genes will spur production of enough
new GABA molecules to replace those lost to the disease.
Klein is the first of 12 patients with advanced Parkinson's
disease the hospital aims to treat with the technique, which was
approved by U.S. regulators. All must have had the disease for
at least five years and no longer benefit from available drugs
and surgical treatments.
New York-Presbyterian plans to begin treating the second patient
in about a month, after assessing whether Klein is safely weathering
the procedure.
In the meantime, doctors said they hope he will not encounter
serious side effects from the therapy, including fever and potentially
dangerous brain inflammation.
Klein, his full head of silver hair parted on either side of
an eight-inch surgical scar, exuded nothing but optimism.
"I hope the gene therapy gives me a chance of getting better
because right now I have trouble walking and can't play ball with
my son," he said in an interview.
He said he was no longer helped by Parkinson's treatments, including
a widely used pill called levodopa that is converted into dopamine
when it enters the brain.
Klein said he plans to return home today to Port Washington,
New York, and enjoy some good home cooking prepared by his wife,
Claire.
"A cheeseburger would be fine; nothing too healthy," said Klein,
whose 14-year-old twin daughter and son will help throw him a
return-home party over the weekend.
Scores of gene therapy trials have been launched without success
since 1990, for a wide array of diseases, including cystic fibrosis,
AIDS (news
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sites) and cancer.
Two French boys with severe immune deficiency were cured with
gene therapy, but they later developed leukemia -- casting a cloud
over the potential of such treatments.