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A Parent Scrambles to Keep Hope Alive

The sudden unavailability of an epilepsy drug that had helped a daughter cope with the disorder leads her mother into a frustrating bureaucratic maze.


Pat Harris cried for three days in April when she got the letter from UCLA Children's Hospital.

A drug that had transformed her daughter's life would no longer be available in the U.S. as of September.

Harris, a San Marino mother of two, had four months to find a solution.

"There was just no way my daughter was going off that drug," she said.

Not after what Harris had lived through for five years in the 1980s, watching her daughter suffer 40 to 50 seizures a day.

Amy Harris, now 18, has Lennox-Gastaut syndrome, a rare and distressing form of childhood epilepsy. As a little girl, she experienced endless "drop" attacks that sent her crashing to the ground without warning like a marionette with its strings cut. She dislocated her shoulders so often that her mother became adept at shoving the bone back into place.

The disorder usually leads to gradual mental deterioration and retardation. In addition, 80% who are afflicted continue to have seizures throughout their lives.

Amy has an IQ of 40 and functions at the level of a 2-year-old. When she was 5, her mother enrolled her in an experimental study for a drug called nitrazepam, which had previously been used overseas for treatment of sleep disorders.

The girl's seizures stopped immediately. She has been seizure-free ever since.

During the past few months, Amy's doctor at UCLA agreed to take the steps necessary to acquire nitrazepam from an overseas pharmacy. But that did not forestall the fears of the Harris family that Amy will no longer be able to count on a stable supply.

Galvanized by that fear, Harris has single-handedly tracked down and mobilized dozens of nitrazepam-dependent families throughout the country, creating an emotional campaign to guarantee the drug's availability.

"She was the one who focused everyone in a direction," said Peggy Marsh, a Cincinnati woman whose son, Brian, has taken nitrazepam for the last 17 years.

The makers of nitrazepam, Swiss-based pharmaceutical giant Hoffman-La Roche, decided in the early 1980s to quit seeking Food and Drug Administration approval to sell the compound in this country.

A U.S. spokeswoman for the company refused in a recent interview to explain the decision beyond saying that the FDA thought that the information the company had submitted was not adequate.

Instead, Hoffman-La Roche decided to send free shipments of nitrazepam from its overseas labs to the small community of needy patients that was soon to include Amy Harris.

As many as 500 UCLA outpatients were taking the drug at one point, but that number has since been pared to 25 as new epilepsy drugs have come on the market. Amy's physician, Dr. Donald Shields, said Amy's seizures were controllable only by nitrazepam.

How many patients still need the drug is a matter of dispute. The drug company says it believes there are only a handful in the nation. Harris says she has been able to document more than 120 through contact with a small fraction of nitrazepam-prescribing doctors in the U.S. She estimates that there are about 500 nitrazepam-dependent patients in all.

Earlier this year, Hoffman-La Roche, which does $12 billion in annual sales, made another business decision: It would no longer assume responsibility for importing the drug from overseas and distributing it to U.S. patients.

Harris' three days of tears began. She wrote to protest.

"Periodically we reassess our product line and how we are spending our resources," a Hoffman-La Roche spokeswoman wrote back. "In the case of nitrazepam, a significant amount of staff time was being spent in coordinating the importation of the drug from overseas."

The calculus was simple, a Hoffman-La Roche executive wrote Harris in another letter:

"The resources that become available to us following closure of this program will allow us to better focus on developing new medicines for patients with major illnesses."

The company told Harris a straightforward solution existed: Doctors could import the drugs themselves. They would simply have to file for a special Drug Enforcement Administration license to import nitrazepam as a Class IV controlled substance.

"It's fairly simple paperwork," said company spokeswoman Kelli McLaughlin.

Doctors and families shake their head in bewilderment at that claim.

Jumping Through Hoops

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