Shortage of Nerve Drug Leaves Thousands Blind

A decision to stop producing an experimental drug has caused several thousand patients to suffer blindness and facial spasms, Harvard medical professors said this week.

The discovers of the drug--called oculinum--which is used to paralyzes eye muscles, have decided to end production because they said insurance costs are too high. Alan B. Scott, the drug's sole producer, was ordered by the Smith-Kettlewell Research Foundation, which funds his work, to stop distrubiting the drug because it was unable to obtain liability insurance.

"To have the drug unavailable is a tragedy," said Dr. Gary E. Borodic of the Harvard-affiliated Massachusetts Eye and Ear Infirmary.

"These patients are so desperate they will go to virtually any lengths to get the problem cured," said Dr. Daniel J. Townsend, an opthamologist at the infirmary who joined Borodic and three of their 130 patients at a news conference Tuesday.

The cause of the sickness is a rare neurological disorder, blepharospasm, that causes involuntary closure of the eyelids and spasms in the face muscles.

"I can't use my eyes at all," said one patient, Mary M. Sousa, 76, of Sommerville. "By evening I can do nothing but go to bed with a scarf over my head to cover my eyes. It's miserable."

The cause of the disorder is unclear, but the drug created from the highly potent botulism toxin is the only medication that can give relief to most of the estimated 50,000 patients afflicted in the U.S., Townsend said.

Based on a study of 95 patients with neurologic blepharospasm, Borodic and Townsend said that the drug appears to be a safe and effective means of treating the unusual disease.

Scott, the associate director of the Smith-Kettlewell Eye Research Foundation, told the doctors he could not find liability insurance while awaiting final approval from the federal Food and Drug Administration (FDA) for full licensure.

If the FDA approves the drug, it should be possible to gain liability insurance, doctors said.

"The application is sitting on somebody's desk while a great many people are suffering intolerably," said Dr. Thomas Crowe, 73, a Northampton radiologist who retired 10 years ago when his involuntary blinking rendered him unable to work. He is one of 2,000 patients who have written appeals to the FDA and Congress to speed approval of the drug.

"We really sympathize with the situation," said John Cipriano, Department Director for the Office of Biologics Research and Review of the FDA. "But obviously, we can't waive all the rules either," he said. He said the drug was currently being evaluated by the licensing committee and the advisory committee.

The drug, Oculinum, is made from botulinum A toxin, a deadly sub-stance that can poison food, a link that Scott said makes it even harder for him to find insurance.

The drug paralyzes the nerves that contract involuntarily. Patients say they feel the effects minutes after injections around their eyes. The side effects include tiny bruises where the injections are made, drooping eyelids and tearing.