WASHINGTON -- A Pennsylvania company says it can no longer afford to provide sufferers of Lou Gehrig's disease with an experimental drug that many believe prolongs life, prompting fear and anger as patients scramble to find alternatives.
Cephalon Inc., of West Chester, Pa., began notifying 159 patients last week that their last shipment of the experimental drug Myotrophin will arrive in December.
It is the latest development in an emotional three-year controversy over Myotrophin -- a drug patients clamor for even though its maker has never proved to the Food and Drug Administration that the medicine is effective.
"They have taken away life," was the reaction from Kyle Hahn of Trenton, Ohio, who painstakingly spelled out that sentence, a letter at a time, by nodding his head.
Lou Gehrig's disease has paralyzed much of Hahn's body, including his vocal cords. He believes Myotrophin helped him because he had almost lost the ability to swallow before using it, but now eats with little trouble and has regained 16 pounds, said Hahn's friend, Terry Frank, who translated his communication.
Patients are furious because Myotrophin gave them hope against a hopeless disease, said Abbey Meyers, president of the National Organization for Rare Disorders, which ran Cephalon's special Myotrophin access program. She read a letter from one Myotrophin user that said by stopping the program, "you're murdering people."
But testimonials do not prove a drug works. The problem with Myotrophin: One scientific study suggested Myotrophin might offer about three months of better health -- yet a second study found absolutely no benefit.
Myotrophin is a genetically engineered growth factor that proponents hoped would protect nerve cells from destruction by Lou Gehrig's disease, formally known as amyotrophic lateral sclerosis or ALS. That nerve destruction causes progressive, ultimately fatal, paralysis. There is only one somewhat helpful treatment, called Rilutek.
When the conflicting Myotrophin results emerged in 1997, the FDA and its independent scientific advisers urged Cephalon to perform a tie-breaking study, but the company refused, saying it had already spent enough money on the drug.
In a classic clash of emotion versus science, feeble patients, many in wheelchairs, picketed the FDA, demanding the drug anyway. The government allowed Cephalon to provide the drug free to desperate patients until the scientific battle is settled, but so many patients wanted the small supply that Cephalon held a lottery.
Last week, Cephalon wrote the 159 Myotrophin users that it cannot afford to continue the program because it has no sign the drug will be approved any time soon.
A Japanese company is finishing its own study of Myotrophin, and results are expected next year. If they are positive, Cephalon could ask the FDA to re-evaluate Myotrophin.
The FDA was surprised by Cephalon's decision to stop its drug-access program, said deputy drug chief Dr. Rachel Behrman. But she said Cephalon has told the agency that patients' final December shipment should contain a four-month supply, giving them time to search out other treatments.
The ALS Association offers a list of experimental drugs at http://www.alsa.org on the Internet. But it has been a disappointing year for ALS drug development, with three compounds failing clinical trials. Only a few studies are enrolling new patients.