MS patients urge FDA to reapprove controversial drug

WASHINGTON — Multiple-sclerosis patients pleaded with the Food and Drug Administration Tuesday to again allow sales of a drug they described as a "miracle" for the disabling disease, while the family of a woman who died taking it warned of devastating side effects.

The drug, Tysabri, was withdrawn from the market in February 2005 after three patients unexpectedly developed a rare brain infection usually seen in AIDS patients and others with weak immune systems. Two of them died. Now the manufacturers are asking the FDA to allow Tysabri to be prescribed again, with a series of limitations on its use.

Such a reversal would be highly unusual. And because there are compelling arguments on both sides, the case is seen as particularly difficult for the FDA advisory panel that will recommend whether to return the drug to market.

The panel of experts is scheduled to vote today, the second day of a public hearing that has included testimony from FDA staff members, pharmaceutical company officials, patients and advocates on both sides.

Tysabri has been shown to be twice as effective as currently available drugs in preventing recurring bouts of MS, which affects an estimated 300,000 Americans. It also appears to delay the overall worsening of the disease.

But Tysabri, manufactured by Massachusetts-based Biogen Idec and Elan of Ireland, also has the potential to affect the immune system. An estimated 1 in 1,000 patients may contract a brain infection known as progressive multifocal leukoencephalopathy (PML). The infection is not easily diagnosed in its early stages and there is no effective cure for it.

The husband and the daughter of Anita Smith, who died while taking the drug, told the panel that the 46-year-old woman should have never been offered Tysabri because her form of MS was mild. But her family said she was persuaded to enroll in a clinical trial of the drug, at least in part, by the promise of free medications.

"We were never told Tysabri could result in Anita's death," a statement from her family said.

But Barbara Crooks, 48, told the committee that "life is all about trade-offs" and that she is willing to take a calculated risk in order to enjoy the benefit of Tysabri again. Crooks, who uses a cane in each hand or a wheelchair to get around, said she was given three doses before it was withdrawn from sale and considered it the most effective MS medication she has ever taken.

Tysabri, the first new MS drug in 10 years, was approved for sale in 2004 after a series of clinical trials. It is thought to work by preventing certain immune cells from attacking the nervous system. But FDA staff doctors said it may also block infection-fighting cells from reaching places in the body where they are needed.

If Tysabri is reapproved for sale, the manufacturers have pledged to institute a system of requirements and checks to try to prevent problems.