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Death Tied to Experimental MS Drug Stirs Debate

As Tysabri's makers seek to resume selling it, critics say a woman who died should not have taken it in the first place.

February 18, 2006|Denise Gellene | Times Staff Writer

A year ago, Anita Louise Smith died from effects of Tysabri -- a drug that was supposed to help her cope with multiple sclerosis.

Within days, drug makers yanked Tysabri off the market, devastating many people with multiple sclerosis who had hoped that the intravenous treatment would bring them relief.

Now, as makers of the drug prepare to seek approval to put Tysabri back on the market, some prominent neurologists contend that Smith shouldn't have been taking it to begin with -- because, they say, she didn't have MS.

Smith's case dramatizes a new debate over how multiple sclerosis drugs are tested: In recent years, drug companies have been trying out medicines on people with mild symptoms or none at all at the time of treatment, several experts say, including some who, like Smith, might not have the disease. These trials broadened the market for MS drugs but, critics say, put patients who don't need powerful new medicines at risk.

Smith, 46, was taking the drug as part of a clinical trial that regulators had required when they granted Tysabri fast-track approval. Most of the 1,171 patients in her trial suffered no disabilities, according to Food and Drug Administration documents.

"People with no active disease -- in other words, people who are doing fine -- shouldn't be given an experimental drug with unknown risks," said Stanford University neurology professor Lawrence Steinman, a co-inventor of Tysabri who has previously spoken out about the drug's dangers.

This week, Steinman and another Stanford neurologist, Annette Langer-Gould, urged the FDA to tighten criteria for selecting patients in MS drug trials.

"We are concerned that not only were patients put at risk by Tysabri, but we feel that the risk was absolutely unnecessary to assume," they said in an e-mail to the agency.

Biogen Idec Inc. and Elan Corp. hope to win government permission as soon as next month to resume sales of Tysabri. They say that it is an effective treatment for multiple sclerosis and that the risks can be managed by carefully selecting and monitoring patients.

Biogen Idec spokeswoman Amy Brockelman said privacy requirements prevented the company from commenting on Smith's case. But all aspects of the Tysabri clinical trials were approved by the FDA and by oversight boards at the medical centers where the studies were conducted, she said.

FDA spokeswoman Susan Cruzan said the agency would not comment on matters regarding Tysabri before an advisory panel meets March 7 to discuss the drug's possible return to the market. This week the FDA allowed the companies to resume clinical trials in MS patients who had been receiving the drug, a step that would provide added information about the risks of Tysabri.

Experts see the drug as a potential boon in the battle against multiple sclerosis, a disease in which errant white blood cells destroy the coating that protects cells in the brain and spinal cord. About 400,000 Americans have the disease.

For most patients, the disease is marked by flare-ups and remissions. Many experience only minor symptoms, such as numbness or tingling. But though the disease is seldom fatal, about half of MS patients become disabled -- such as comedian Richard Pryor, who was unable to speak, swallow or get out of bed unassisted in the years before his death late last year.

Though not a cure, Tysabri was designed to prevent or delay progression of the disease by blocking the white blood cells that damage the brain.

A once-monthly medicine, administered through an intravenous infusion in a doctor's office, Tysabri received fast-track approval in late 2004 -- on condition that clinical trials continue -- because the FDA deemed it a significant advance over existing treatments. Researchers in one study noted that Tysabri nearly doubled patients' chance of infection, although the overall risk was tiny.

Despite an annual price tag of $23,500, initial sales of Tysabri were strong. When the drug was withdrawn, 5,000 patients were taking it and 15,000 more were awaiting insurance verification and their first dose. The companies needed just 20,000 patients to break even on the drug.

Biogen Idec, based in Cambridge, Mass., and Elan, based in Dublin, Ireland, saw Tysabri as a potential blockbuster, and Wall Street expected sales to one day reach $3 billion. Now analysts are forecasting annual sales of as much as $1 billion should the drug return to the U.S. market and receive approval in Europe.

Questionable Diagnosis

Smith, who lived in Colorado Springs, Colo., was diagnosed with multiple sclerosis in 2000 after a bout of weakness in her right leg, followed by blurred vision -- both common symptoms of MS.

Although the symptoms disappeared, she started taking injections of Avonex, another Biogen Idec drug for MS. Insurance covered some of the cost, but Smith still had to pay $1,000 a month, her husband, Walter, said.

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