Two Parkinson's disease patients Tuesday sued Amgen Inc. to force the biotechnology giant to provide an experimental and potentially dangerous drug that the patients and their families view as their only hope.
Medical ethicists said the case was unusual because it pit desperately ill patients and their physicians against a drug company. In that sense, it differs from the early struggles of AIDS patients for access to unapproved medications.
"In those cases, drug companies and doctors agreed the drugs weren't ready," said Jonathan Moreno, a medical ethicist at the University of Virginia.
The suit, filed in federal court in New York, demands that Amgen offer the Parkinson's medicine to patients who had once received it in a clinical trial that ended last year.
The patients said they went to court after months of begging Amgen for the drug, which they considered a godsend. Robert Suthers, 70, said the drug quelled his tremors and restored his energy; now he lacks the strength and coordination to bathe himself.
"It was tremendous," he said, recalling his treatment with GDNF. "I could walk two miles like it was nothing."
The patients' pleas stand in poignant contrast to the accusations leveled at manufacturers in the recent debate over drug safety. The withdrawals of painkillers Vioxx and Bextra came amid assertions that companies failed to protect patients from rare but harmful side effects.
The Parkinson's patients want to assume the risks of taking the Amgen drug, but the company won't allow them to do so.
"The shoe is on the wrong foot," said Arthur Caplan, a medical ethicist at the University of Pennsylvania.
Amgen declined to comment on the lawsuit.
The company did not consult with physicians involved in the clinical trial before making its decision, according to the lawsuit. Six doctors who administered the drug to patients in the trial said in court documents that they disagreed with Amgen's decision and considered GDNF safe and effective.
Amgen broke its contract with patients when it pulled the drug, the suit charges. Patients allowed physicians to "drill holes in their brains and insert catheters" in return for the right to receive "GDNF indefinitely."
The case could redefine the obligations of drug companies to participants in clinical trials, Moreno said. If experts believed that an experimental drug had a benefit, a company might have an obligation to continue testing it.
"It seems to me that a company can't wave its hands and do whatever it wants," Moreno said. "The wrinkle here is whether a company can be forced to investigate something it doesn't want to investigate."
Patients in the Amgen clinical trial received GDNF directly into their brains through tubes that led from pumps implanted in their abdomens. Half of a total of 34 patients received GDNF for six months while the rest received a placebo of saline solution.
At the end of the study in June, Amgen said it found no differences between the groups in performing such tasks as walking, talking and writing, even if some individual patients believed they did improve.
However, some physicians who treated patients in the trial disagreed with Amgen's findings. The doctors said the statistical differences between the groups appeared small because patients received too low a dose of GDNF.
Dr. Don Gash, a scientist at the University of Kentucky, said he had 10 patients on the drug and all "showed significant improvement in motor functions and quality of life."
Without the drug, Gash said, his patients "are deteriorating. We are seeing shaking, muscle cramps, hallucinations and dementia."
After the trial failed, Amgen allowed all patients to receive the drug while it tried to figure out what went wrong.
The company said the dose or the duration of treatment -- six months -- might have been inadequate.
Amgen went back to its labs and retested GDNF in monkeys. The company became alarmed when four of 70 monkeys developed brain lesions, according to the lawsuit. Concluding that the risk of brain damage was too high, Amgen stopped supplying the drug to the Parkinson's patients.
However, Michael Hutchinson, a neurologist at New York University who treated patients in the Amgen trial, said the monkeys might have been harmed not by GDNF but by the sudden withdrawal of the drug.
The suit was filed by two patients, Suthers and Niwana Martin, 60, of Harpers Ferry, W.Va. Four other patients submitted affidavits in support of the suit.
"I don't think Amgen was being sensitive. I don't think they looked at what they asked us to do for them," Martin said. "I went through brain surgery and I had holes drilled in my head and catheters and pumps in my abdomen all to have the benefit of the drug."
Martin and Suthers said saline solution was being pumped through their catheters, so they were ready should Amgen be forced to provide the drug.