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Holding off a notorious disabler
Maura Lerner, Star Tribune
Alicia Blank hasn't been able to walk in years and needs a motorized scooter to get around.
But at age 45, she considers herself one of the lucky ones.
She might have been in a nursing home by now, if not for a new generation of drugs that is changing the face of her disease -- multiple sclerosis.
Blank, of Fridley, has lived almost half of her life with MS, the notorious crippler of young adults. At the start, she learned that there was nothing doctors could do to stop it.
But in the past decade, a half-dozen drugs have been approved to slow or halt the progression of MS, a nerve disease that affects 400,000 Americans. One of those drugs, says Blank, "gave the quality of my life back." And the newest one, a genetically engineered drug called Tysabri, is said to be twice as effective as anything on the market.
"These drugs have changed the landscape of MS," said Blank's physician, Dr. Randall Schapiro, director of the Schapiro Center for Multiple Sclerosis in Golden Valley.
They are not a cure, he's quick to add, because they don't restore any lost muscle or nerve function. Nor do they work for everyone.
Yet they suggest that the battle is turning, and that it may be possible to stop MS in patients before it does lasting damage. "I think it's happening in front of my eyes," said Schapiro, a neurologist.
The drug Tysabri won approval from the Food and Drug Administration (FDA) last month. It's designed to stop the disease from destroying the protective tissue around the nerves, which is how MS wreaks havoc. Studies showed that Tysabri reduced the number of relapses -- the episodes when symptoms flare up and worsen -- by up to 66 percent, nearly twice the rate of other MS drugs.
Now, doctors have six drugs to choose from, ranging in cost from $1,000 to $2,000 a month.
It's a long way from the 1970s, when the medicine chest was bare and the state of MS treatment was famously described as "diagnose and adios." Some patients literally felt abandoned. At the time, the best that doctors could do was treat the symptoms, give steroids when they flared up and try to help patients cope.
Blank's story
Alicia Blank was a vibrant young newlywed in 1980 when she first had the symptoms no one understood: numbness, tingling, loss of balance. One doctor thought she had an inner-ear problem. An avid runner and biker, she was almost never sick. Then one of her legs stopped working. Sometimes she got better; sometimes not. After four puzzling years, a neurologist told her, "I think you have MS."
At 25, she found herself trapped in a downward spiral. She was in and out of the hospital as flare-ups stole her ability to control her bodily functions. Her disease was getting progressively worse.
MS is known for being maddeningly unpredictable. "No two people with MS are alike," said Schapiro, who is on the national board of directors of the Multiple Sclerosis Society and author of a book, "Managing the Symptoms of Multiple Sclerosis."
The symptoms can be all over the map -- from double vision to memory problems to muscle weakness. Experts aren't even sure it's one illness; it has at least four distinct forms. Something causes the white blood cells that normally fight infection to attack the tissue (called myelin) that surrounds the nerves. That in turn disrupts the signals that control movement.
Some people are disabled; while many have milder symptoms -- such as muscle weakness or numbness -- that are barely noticeable to others. About two-thirds of patients are still walking 20 years after their diagnosis, according to Schapiro. But the threat of getting worse, he said, is always "hanging over you."
Historically, MS has been a prime target for quackery, Schapiro said. In the absence of anything else, people turned to magnets, bee stings, cobra venom, "a lot of just crazy therapies," he said. "Wishful thinking."
But in 1993, the FDA set off a frenzy when it approved a drug called Betaseron. It's a genetically engineered version of a natural substance called interferon, which regulates the immune system. Scientists found that it could reduce the frequency and severity of MS attacks.
The reaction was overwhelming.
People were scrambling to get it, "sort of like the flu vaccine," recalled Schapiro. A lottery was held to decide who would get the first 20,000 doses.
Alicia Blank threw her name into the lottery, and won. She started giving herself a shot of Betaseron every other day and hoped for the best.
A leap of faith
It wasn't an easy drug to take, Schapiro said. It was costly, painful, had side effects and didn't make anyone feel better. Many were tempted to give up.
But over time, Schapiro began to see a difference in his practice. He had fewer patients in the hospital. Fewer patients losing the ability to walk. Fewer flare-ups and fewer setbacks.
Blank hasn't been hospitalized in 11 years, since she started taking the shots. Essentially, she says, "It's stopped the progression of the disease." She lives at home with her husband, Tim; drives a specially fitted van, does volunteer work and travels the world. She doesn't like to think about where she might be without the drug. But if she'd had it sooner, she says confidently, "I'd be walking and riding my bike. ... I wouldn't even have this disability."
Four more drugs were added to the arsenal between 1996 and 2002. Yet many patients, including Martha Cincoski of Eagan, are still searching for the right one, or perhaps the right combination.
Cincoski, 37, has tried almost everything since MS sidelined her career as a budding surgeon in 1995. Yet her disease is progressing, and she started using a cane last summer.
Taking this kind of drug, she said, requires a leap of faith. All of them are designed, one way or another, to prevent further damage to the nerves, but they can't repair damage already done. And it's hard to measure the drug's effect in any one person.
"The problem is ... you don't know where you'd be without it," Cincoski said. "I just think that if I hadn't been on drugs, would I even be walking now?" Now, she says, she's eager to try the newest drug, Tysabri. "The studies are showing me that this is working, so let's try it," she said.
Doctors say they're impressed with Tysabri's results in early studies. But with new drugs, "there's always a big surge of excitement, and one always has to be careful," said Dr. Gary Birnbaum, an MS specialist and a colleague of Schapiro. He, for one, wants more research on who's likely to benefit, as well as possible side effects, which include headache, depression, infections and joint pain.
So far, the MS drugs as a group have not been linked to long-term problems, according to Schapiro. But last month, when one of his patients wondered aloud about the risk of taking these drugs indefinitely, he acknowledged that it's still an open question.
"My answer is, there's terrible long-term effects of multiple sclerosis if it gets out of control," he said.
"That is what you're weighing it against."
Maura Lerner is at mlerner@....