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Here's one of the guys that would make you a believer in GDNF.

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.
» Click to enlarge image
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.



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.


No
w 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.

-The Elgin Courier News


Paula