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FROM: International Herald Tribune online (Originally published in New York Times 11/26/04) Patients plead to keep drug for Parkinson's By Andrew Pollack The New York Times Saturday, November 27, 2004 With his condition deteriorating from Parkinson's disease last year, Steve Kaufman gave up making improvements to his home in Algonquin, Illinois. "I couldn't even hold a nail stable," he recalled. 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 Parkinson's 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. Amgen's move has provoked an outcry from patients who say the company is robbing them of their only hope. "It's almost the same thing as a diabetic losing their insulin," said Kaufman, who is 50 and has had Parkinson's for 10 years. The story of Amgen's 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 At a time when public debate is focused on whether drugs like Vioxx, withdrawn in September for safety reasons, are remaining on the market too long, it shows patients who are more than willing to accept risks to get a drug.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 Parkinson's, 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 patient's 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 the 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 women's 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. 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 company's 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 Amgen chief executive, called the failed trial a "tragedy" and "the single most disappointing" one in his 12 years at the company. Some doctors involved in the clinical trial have criticized Amgen, saying the trial failed because it was poorly designed, not because GDNF does not work. "I don't think there's 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 company's decision, noting that Amgen had spent hundreds of millions of dollars over 10 years on the drug's development. Some doctors and patients want Amgen to license the drug to another company or a university willing to continue trials. But Sharer said Amgen could not do so because it would still be liable if safety problems arose. The doctors and patients also want Amgen to provide the drug as a compassionate gesture to the patients from the clinical trials under the U.S. Food and Drug Administration's rules. Amgen says the drug does not qualify for this because it is not under development and has safety problems. Some of the doctors plan to plead their case to the agency. http://www.iht.com/articles/2004/11/26/business/amgen.html SEE ALSO: Grassroots Connection: GDNF Research http://www.grassrootsconnection.com/grcissue_GDNF_research.htm ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn