Fight of his life
Man helping to lead way in Parkinson's debate
October 5, 2006
By TOM POLANSEK SUN-TIMES NEWS GROUP
Before receiving an experimental drug called GDNF, Steve Kaufman could not
hold a nail because his fingers were so immobilized by Parkinson's disease.
Just months after his treatments began in 2003, however, he was able to
renovate the kitchen in his Algonquin home. Later, in an even more ambitious
project, he added a deck on to the rear of the house.
"I was feeling so wonderful," Steve, 52, said. "It was like getting my life
back."
But the euphoria didn't last long. Less than a year after he started on
GDNF, officially known as Gilal-cell-lined derived neurotrophic factor, the
drug's manufacturer, Amgen Inc., canceled the clinical trial in which Steve
was enrolled.
» Steve Kaufman of Algonquin and his wife Maggie sit on the deck Steve built
while participating in a drug trial to treat his Parkinson's disease. The
deck was built in the summer of 2004 after the treatments began. Maggie said
Steve's body allowed him to do things while he was being treated with the
trial drugs.
sun-times news group
That meant he no longer had access to the medicine that had taken away pain
in his legs, stopped much of his shaking and allowed him to resume normal
activities. Steve and his wife, Maggie, did not take the news lying down.
Instead, for the past two years, they have networked with other GDNF
patients from around the world and lobbied Amgen hard to make the drug
available again.
The determined couple have contacted the company's top executives, met with
officials from the Food and Drug Administration, and shared Steve's story
with Michael J. Fox, the actor who became an advocate for Parkinson's
patients after being diagnosed with the disease.
For all Parkinson's patients
Although GDNF drastically improved Steve's quality of life, the Kaufmans say
they no longer are fighting just for themselves. They now fight for every
person stricken with Parkinson's, a degenerative disorder of the central
nervous system that affects the control of muscles.
"The drug's effective," Maggie said. "We know it's effective. Everyone that
sees Steve knows it's effective."
Indeed, it has become the Kaufmans' mission to connect those suffering from
Parkinson's with a drug that may improve their lives.
Steve and Maggie, who describe themselves as religious, feel they were put
on Earth to put GDNF into the hands of those who need it. Other Parkinson's
patients regularly call their home and send them e-mails to discuss the
battle for GDNF or hear about Steve's story.
For many doctors and Parkinson's patients, Steve has become an inspiration.
In February, the couple attended the World Parkinson's Congress in
Washington, D.C. To illustrate the effectiveness of GDNF, the Kaufmans
showed pictures and a video of Steve building the deck.
Steve became a minor celebrity at the conference. "People wanted to meet
me," he recalled.
One of those people was Robin Elliott, executive director of the Parkinson's
Disease Foundation, a private, New York-based organization dedicated to
advancing Parkinson's research and assisting victims of the diseases.
Elliott recalled being impressed by Steve's ability to function. "My
reaction was that this was one healthy, happy man," Elliott said.
Quick results
In the clinical trial, GDNF was administered to patients as a liquid through
pumps that were implanted in their abdomens. Catheters were then fished
under the patients' skin to take the medicine to their brains.
Steve's surgery took place in 2003, and lasted six hours. He was one of
three trial patients in the Chicago area. Others were in New York, Kentucky
and Canada.
The benefits of the surgery and medication became apparent relatively
quickly. Six weeks after the first injection, Steve was shaving, and he
noticed his facial muscles had relaxed.
Formerly frozen, his face became animated once again. The pain he felt in
his legs also melted away slowly. By February 2004, Steve said he "really
started to feel good."
Then came the news that Amgen halted the study. Although Steve was thriving,
the company reported that tests showed the drug was ineffective and could be
dangerous.
"We were devastated," Maggie said. "A week before they took the drug away, I
forgot he was sick."
Without the drug, the pain started to seep back into Steve's leg, although
he still functions better than he did before the GDNF.
Now what?
The Kaufmans asked Amgen to provide the drug to trial participants on the
basis of "compassionate use." Amgen, thus far, has refused.
Amgen CEO Kevin Sharer said in a statement that company researchers spent
several months reviewing the company's decision to halt GDNF clinical trials
and concluded that it was for the best.
Many patients involved in the GDNF trial have had their delivery systems
removed since the trial ended.
Steve, however, says he will keep his device in while he fights to get the
drug-and his life-back. "We will not stop fighting until there is nothing to
fight for," he said.

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