By Denise Gellene

Los Angeles Times

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

Within days, drugmakers yanked Tysabri off the market, devastating many people with multiple sclerosis who hoped 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 are arguing that Smith should not have been taking it to begin with - because she did not 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, several experts say - including some who, like Smith, might not have the disease. Critics say these trials broadened the market for MS drugs but put at risk some patients who do not need powerful new medicines.

Smith 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 Lawrence Steinman, a Stanford University neurology professor and a co-inventor of Tysabri who previously has spoken out about the drug's dangers.

Last 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 wrote in an e-mail to the agency.

Biogen Idec Inc. and Elan Pharmaceuticals Inc. 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 carefully selecting and monitoring patients can manage the risks.

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 surrounding Tysabri before an advisory panel meets March 7 to discuss the drug's possible return to the market.

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 people in the United States 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 while the disease is seldom fatal, about half of MS patients become disabled - such as comedian Richard Pryor, who could not speak, swallow, or get out of bed unassisted in the years before he died late last year.

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