Earlier this year, after taking an experimental drug in a clinical trial, Kaufman built new kitchen cabinets and an outdoor deck. He was so steady he could walk across a narrow piece of lumber like an Olympic gymnast on the balance beam.
The drug, however, is no longer available to Kaufman or other Parkinsons patients in clinical trials. In June, its developer, Amgen, announced that the drug, which is called glial cell line-derived neurotrophic factor, or GDNF, had not proved better than a placebo.
Two months later, the company said that safety issues had been discovered and it abruptly ordered all patients taken off the drug.
Amgens move has provoked an outcry from patients who say the company is robbing them of their only hope. Its almost the same thing as a diabetic losing their insulin, said Kaufman, who is 50 and has had Parkinsons for 10 years.
The story of Amgens drug shows the clash between the faith of patients and the cold logic of science and business. At a time when public debate is focused on whether unsafe drugs like Vioxx are remaining on the market too long, this story shows patients who are more than willing to accept risks to get a drug.
Their willingness also raises an ethical question: If a company stops developing a drug for safety or efficacy reasons, is it obligated to continue supplying it to patients from its clinical trials
A complicating factor in the clinical trials is the nature of Parkinsons, a disease that chokes off the supply of dopamine, a signaling chemical, in the brain. Some studies have suggested that mere anticipation of treatment can induce a patients brain to produce more dopamine, which alleviates the symptoms. The patients not only feel better, they are better at least for a time. In fact, some of patients in the GDNF trial who improved the most had received a placebo.
Patients and their families have written letters to Amgen, imploring the company to continue providing the drug. Some of the most poignant have come from England, where the drug has been tested the longest, since 2001.
To quote a headline that my daughter used in a story she wrote for a national womens magazine GDNF has given me my dad back, wrote Stephen Waite, who had used the drug for more than three years. In a telephone interview, Waite, 60, began crying when talking about the loss of the drug.
I would sign a disclaimer, anything, in order to continue, he said.
Another family told Amgen how the drug allowed their mother to shop, dance and go on vacation for the first time in three years. GDNF deserves more time, and so does our mother, they wrote.
But while Amgen executives say they empathize with the four dozen patients who have participated in the trials, they also say they cannot keep giving them a drug that does not work and might be dangerous. How can we ethically justify administering this drug said Roger Perlmutter, executive vice president for research and development.
In August, the companys studies found that high doses of the drug damaged some monkey brains.
It also found that a few patients had developed antibodies against the drug, posing a potential danger.
Kevin Sharer, the chief executive, called the failed trial a tragedy and the single most disappointing one in his 12 years at the company.
Some of the doctors involved in the clinical trial have citicized Amgen, saying the trial failed because it was poorly designed, not because GDNF does not work. I dont think theres ever been a trial like this where so many investigators were so indignant about the way things were handled, said Michael Hutchinson, an associate professor of neurology at New York University who was involved in the trial. Critics also say the safety concerns are not serious. Some even suspect Amgen is killing the drug because it believes it will not be a big seller.
But Sharer denied that business considerations were behind the companys decision, noting that Amgen had spent hundreds of millions of dollars over 10 years on the drugs development.
ANDREW POLLACK / NY TIMES