As MS Pills Debut, Doctors Prescribe a Dose of Caution

Multiple-sclerosis patients have waited years for the first generation of treatments in pill form. Now that they're becoming available, however, doctors are warning: not so fast.

The pills are easier to take than shots that have long been used to treat the disease. Yet they could have serious side effects, and for patients who are stable on their current regimen, doctors say shifting to pills may not be worth it.

Jill Kider had been anticipating the pills since she began injecting herself daily two years ago with a drug called Copaxone.

Copaxone stopped Ms. Kider's attacks of her multiple sclerosis. But when she travels, it isn't always easy to carry the syringes through airport security or refrigerate them at hotels. Still, a talk with her doctor, Peter Calabresi, persuaded not to take a chance on a new drug.

"I don't have any side effects, and it seems to be working very well for me. Why switch it?" said the 52-year-old from Rockville, Md.

The newest pill, Aubagio, was approved in September. Another, dubbed BG-12 until it is named, could be approved early next year. They join Gilenya, the first pill, which was cleared for use in late 2010.

Dr. Calabresi, who helped lead the clinical testing for Gilenya and consults for other drug makers involved in multiple sclerosis, likes to spend two hours discussing the various options with patients.

For "all of those patients who had been waiting years for a pill, the conversation isn't quite as rosy as we'd like it to be," said Dr. Calabresi, director of the Johns Hopkins Multiple Sclerosis Center in Baltimore.

Multiple sclerosis is a neurological disease that strikes painfully and unpredictably in 250,000 or more people in the U.S., about two-thirds of them women. In the U.S. last year, doctors filled 2.2 million prescriptions for multiple-sclerosis drugs, according to IMS Health, which tracks drug sales. World-wide, 2.1 million people have the disease, according to the National Multiple Sclerosis Society.

Symptoms, often starting with vision problems, muscle weakness and dizziness, commonly surface between 20 and 40 years of age. Most patients will face stretches of relief, interrupted by periodic attacks of the symptoms. Multiple-sclerosis drugs are measured, in part, by their effectiveness reducing the recurrence of those attacks.

Multiple sclerosis takes different forms. The most common involves periodic attacks of the disease—or relapses—while in other forms the disease progressively worsens. The new pills treat only the disease's relapsing forms.

The pills and injectable drugs typically cost $45,000 a year or more, and they are usually covered by insurance.

When Ms. Kider first sought treatment, the most popular options, such as Copaxone, Avonex and Rebif, were shots. Yet the injections can cause welts, itching or scarring. The most effective treatment is a drug taken intravenously, Tysabri, but it had been linked to a rare brain infection.

Tysabri reduced the rate of attacks by 68% over the course of two years compared with placebo, says Biogen Idec Inc.,its maker. But a medical test identifies about half of multiple sclerosis patients as at a higher risk of developing the brain infection if they take the drug, according to Lawrence Steinman, a Stanford University neurologist.

Generally, Dr. Steinman says he will prescribe Gilenya to new patients who haven't tried one of the older therapies, have aggressive cases of multiple sclerosis and want to take a pill. Gilenya reduced relapses by 54% at two years treatment, according to Novartis AG, its maker.

Yet Dr. Steinman says Gilenya won't work for some patients. He wouldn't give it to heart patients, because it could dangerously slow their heart beats, which could lead to death. Another concern with Gilenya, which works by suppressing the immune system, is that it increases the risk of herpes virus infections that can lead to shingles.

Aubagio cut relapses by 36% during an average of 18 months treatment, according to Sanofi SA'sGenzyme unit, which sells the drug. Doctors say that rate is comparable to the effectiveness of the older injections, and that clinical testing didn't indicate any serious side effects. But lab testing with animals suggested a risk of fetal death—and the drug's label warns against giving the drug to women of "childbearing potential."

Choosing which drug is most appropriate for patients is "all about safety and efficacy, not oral versus needle," said Dr. Steinman, who has consulted for Novartis and many other companies selling multiple sclerosis drugs.

It is difficult to find multiple-sclerosis treatments that are both effective and safe, experts say, because the understanding of the disease continues to evolve.

Researchers say multiple sclerosis wreaks havoc with a patient's immune system, causing it to damage healthy nerves and the myelin sheaths that wrap around nerve fibers, disrupting signals from the brain. Researchers still don't understand why the immune system goes haywire.

They believe it is a combination of genetic and environmental factors, including exposure to a virus.

The progressive forms can cause fatigue and depression and make it difficult for sufferers to concentrate and make decisions. Eventually, the disease can become debilitating, putting many patients in wheelchairs.

Timothy Coetzee, chief research officer at the National Multiple Sclerosis Society, says treatment "is in a remarkable place than it was years ago, when we had no disease modifying" drugs. He expects the new pills will help patients stay on their regimens, an important component to avoiding attacks.

For now, Mark Keegan, who heads the multiple-sclerosis section at the Mayo Clinic in Rochester, Minn., has supported use of the older shots. That is "mainly because of an overall safety profile that is good," said Dr. Keegan. His counsel may change, he said, as doctors gain more experience with the new drugs in pill form.

By: Jonathan Rockoff, Wall Street Journal
Reviewed / Posted by: Scott W Yates, MD, MBA, MS, FACP

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