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MS takes toll as patients wait for scarce new drug

This was the year medical science finally unleashed a drug that slows the course of multiple sclerosis, and Lisa Schenning would take it tomorrow if she could. Perhaps, it could stave off the attacks that progressively rob sensation from her hands, weaken her right side and cloud her vision.

But instead of the drug, she has lottery number 21,126. That means she may have to wait until early 1995 to get Betaseron, even though the drug was approved by the Food and Drug

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Administration last summer.

In a turn of events that has surprised, frustrated and angered many people with MS, the company marketing Betaseron found itself unprepared to satisfy the flood of patients wishing to take it. So the company devised what it calls a "random access" system -- a lottery by any other name -- to determine who gets the drug now and who must wait.

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In August, Berlex Laboratories gave patients a three-week window to register for the drug, then let its computer put them in random order. Some 67,000 patients signed up. Everyone is guaranteed the drug. The only question is when.

"You feel angry, disappointed, hopeful, confused," said Ms. Schenning, 25, who recently took leave from her job with a Baltimore accounting firm when an MS attack left her so weak she couldn't rise from her chair.

She has recovered substantially and now walks with a slight limp but uncanny vigor. Still, she cannot escape the fact that each attack dims her vision a little more.

Someday, she may go blind.

"I've had two attacks," Ms. Schenning said. "Both were at the end of the year, around Christmastime. What if I don't get the drug until the end of next year? Maybe I won't have my vision anymore when the drug comes."

Now there's hope

Like many patients who weren't lucky enough to draw low numbers in the Betaseron lottery, Ms. Schenning tempers her anger with understanding. She realizes that after decades, researchers have at last given MS sufferers a drug on which to hang their hopes.

"Finally, there's something out there. There was nothing before."

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Multiple sclerosis, which afflicts an estimated 300,000 people in the United States, occurs when the immune system unaccountably attacks the myelin sheath that protects nerve fibers of the brain and spinal cord. This causes a confusing "cross-talk" between fibers, slowing and sometimes interrupting signals that control such functions as movement, balance, feeling and sight.

The disease may cause dizziness, impaired vision, numbness, tingling, incontinence and paralysis. The symptoms vary from patient to patient and tend to worsen with age -- but at a rate that is utterly unpredictable.

For many people, the disease flares in attacks that can occur months or years apart -- a pattern known as "relapsing-remitting." Typically, the symptoms subside between attacks, but not totally. Each attack leaves the patient weaker, never knowing when the next flare-up will occur and what its effects will be.

Betaseron is a genetically engineered drug that seems to suppress the autoimmune attacks. It's no cure -- no one has figured out how to eliminate the attacks -- but a clinical trial completed this year demonstrated that the drug reduced their frequency by about 30 percent.

Buying more time

The three-year trial raised justifiable hopes that MS sufferers can buy more time between attacks and possibly slow the long-term course of their illness.

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So far, the drug is not for everybody. It has been tried only on people in the early stages of relapsing-remitting disease, a subgroup that accounts for about one-third of all sufferers. Accordingly, the FDA has approved distribution to these patients.

Betaseron costs about $10,000 a year and is covered by most insurers.

Jeffrey Latts, Berlex's vice president for clinical research and development, said the company wasn't surprised by the volume of patients eager to take Betaseron. The real surprise came more than a year earlier, when preliminary test results indicated that the drug might work.

Over the past decade, test after test had produced nothing but failure.

Although Betaseron was more than a year way from market approval, Dr. Latts said, the company realized the one plant making the drug couldn't possibly satisfy the demand after it was approved.

Expanding to meet demand

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Seeing this, the biotechnology firm that makes Betaseron for Berlex started expanding its production capacity early in 1992. The company, Chiron Corp., expects that by 1995 it will be able to meet demand for the drug.

"I've talked to a lot of physicians and a lot of patients," Dr. Latts said. "All would prefer that we had more Betaseron. But the majority realize we are doing the best we can do given the limitations of supply."

At the heart of the problem is the wondrous technology by which Betaseron is produced. Rather than mixing chemicals in a laboratory, Chiron coaxes the drug from nature.

'Smart bacteria'

This is done by infusing bacteria with the human gene for beta interferon, a human protein that seems to inhibit the immune system attacks that are the hallmark of MS. The result is "smart bacteria," biological factories that produce the natural product dependably but slowly.

After government approval, Berlex first awarded 3,500 treatment slots to hospitals that are considered major centers of MS treatment. The University of Maryland Medical Center received 100, which it allocated through an internal lottery. Many of those patients started therapy this month.

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The rest of the patients seeking Betaseron at UM and elsewhere -- more than 60,000, all told -- will get the drug in random order.

Berlex projects that people holding numbers between one and 12,000 will receive the drug within the next few months, those with numbers between 12,000 and 20,000 will get it later in 1994, and those above 20,000, sometime in 1995.

About 100 people registered for the national lottery through the University of Maryland. Thirty-nine were awarded numbers below meaning they should soon receive their first doses of Betaseron. The rest will have to wait longer.

Trying to be fair

"I think they tried to do this to ensure fairness," said Dr. Kenneth Johnson, a University of Maryland neurologist who played a key role in the beta interferon trials. "I can't imagine they're not doing it as fast as they can."

Cathy Costello, a UM nurse who recently began teaching patients how to mix and inject the drug, said most patients who received high numbers tried to take the news philosophically.

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"They wish they could get it now," she said. "They hope this disease doesn't change in the meantime. They already feel pretty unlucky they have MS. But they're not totally unlucky. They will be getting the drug."

Waiting anxiously

Laurie Huse, 43, of Montpelier, Vt., has had numerous attacks since she was diagnosed in 1974. But the last one, on Mother's Day 1992, was by far the worst. It paralyzed the left side of her body, draining her of so much strength she struggled to breath.

Today, she walks with a cane, but is able to drive and maintain her full-time job. Still, she can't help but wonder how she will fare next time. With number 49,944, she may have to wait until 1995.

"The fact is, I am angry," said Ms. Huse, communications director for the National Education Association in Montpelier. "The drug is not a brand new thing. They've been experimenting with it and, obviously, the company was in this to sell it. They had to have had an idea of how many people would be anxious to have this drug.

"One more attack, and I'm afraid I won't function," said Ms. Huse, who is married and has four generations of family living in Vermont. "I'm afraid for my husband and family that if I'm attacked again, I won't recover. The fear is natural."

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Offer to buy low number

Steve Mailman, 34, of Dallas created a flurry of interest -- and controversy -- on a computer electronic bulletin board for MS patients when he offered to buy someone's low number.

He was assigned 48,657.

"By buying a lower number, I really wouldn't displace anyone else," said Mr. Mailman, co-founder of a national chain of sign stores. When he approached Berlex with his idea, he was rebuffed.

"We designed the system to be as equitable as possible," Dr. Latts said. "We clearly did not want anything resembling a secondary market, or a black market, or anything of the sort."

Ms. Huse, who uses the bulletin board, said she abhors the notion of selling numbers but understands the motivation. "I see it as a symptom of everybody's anxiety," she said.

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'Should I take it?'

A Columbia woman with a number around 29,000 said she would take the drug immediately if she could. But she acknowledged feeling some relief that she will have to wait a year or so. That will give her time to gauge the side-effects through the experiences of others.

Safety studies have not identified serious side effects, although some people have experienced fever, loss of appetite and painful joints.

"The euphoria since the drug came out was overwhelming," said the woman, who asked not to be identified. "Now, the deadline is getting closer, and there's a lot of anxiety. People are saying, 'If my number comes up, should I take it or not?' "

'Life is tough'

Diagnosed in 1981, the 45-year-old woman endured an attack six years ago that paralyzed her. Now, she walks with an unsteady gait that lends what she calls "a drunken appearance."

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"I don't have a problem with the lottery," she said. "They can only make so much of the drug because of the technology.

"It's hard when you have these symptoms and you're getting worse and there's something out there that you can't have. But life is tough."


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