A panel of medical experts listened to compelling and often emotional testimony Tuesday from multiple sclerosis patients who say the drug Tysabri succeeded in bettering their lives after other medications had failed. The testimony was presented on the first day of a two-day meeting before a Food and Drug Administration advisory panel convened to decide whether Tysabri is safe enough to again be sold. One of the people who appeared before the committee was a woman from Oregon who says she tried three treatments without seeing improvements before Tysabri. However, after one infusion of Tysabri, she says her muscle spasms stopped. Now the woman, who says she has no financial interest in either of the drug's marketers, Biogen Idec ( BIIB) and Elan ( ELN), wants to decide for herself if she should continue taking it, an option that she currently doesn't have. Those comments were echoed by other of the drug's supporters, many of whom said they simply want to have the choice of using Tysabri. In what some patients at the hearing called an overreaction, Tysabri was pulled from the market in February 2005 after being linked to a potentially fatal brain illness. Much more will be known about the drug's fate after the advisory committee meeting wraps up Wednesday. The group will make recommendations as to whether the FDA should clear the drug and under what circumstances. Many analysts expect that Tysabri will ultimately return to the market, but that certain restrictions might be placed on its use. Tysabri was withdrawn after being connected to a disease called progressive multifocal leukoencephalopathy. Two PML cases appeared in MS patients who took Tysabri along with another Biogen MS treatment, Avonex. A patient who received Tysabri during a Crohn's disease trial was later found to have PML. In two of the cases, the patients died.
Multiple sclerosis is a chronic, debilitating disease that affects the brain and spinal cord. MS is thought to be an autoimmune disease, and while it isn't considered deadly, it can severely compromise a patient's quality of life, possibly leading to numbness, difficulty thinking and coordination and speech problems. People with MS can become severely disabled, but most don't, according to the National Multiple Sclerosis Society. Effective MS drugs besides Tysabri do exist, but some patients whose disease has come back after those treatments say they'd rather face the risk of PML than be forced to struggle with their condition. Biogen laid out plans for following up with patients on Tysabri if the drug gains the FDA's reapproval, saying everyone who takes it would be tracked to determine the rate at which adverse incidents occur. Additionally, Biogen said it would query physicians after six months and track patient deaths through a national index and a collection of death certificates. The companies expect to follow 5,000 patients, including 3,000 in the U.S., to monitor for side effects or serious infections. They say they'll gather information on patients who have problems while using Tysabri in combination with another drug that suppresses the activity of the body's immune system. Not all of the testimony before the panel was positive, of course. The family of Anita Smith, a Tysabri patient who died of PML, also spoke at the FDA hearing. Smith was initially treated with Avonex for MS, but after hearing of Biogen Idec's trial on Avonex plus Tysabri, she enrolled. After her death, her MS diagnosis was questioned, and in turn, so were Biogen's enrollment procedures. Following the PML cases, Biogen and Elan undertook a safety evaluation of more than 3,000 Tysabri patients in collaboration with leading experts in PML and MS, turning up no new confirmed cases of the disease beyond the three that had been reported. They submitted a new application to regulators for Tysabri, and the FDA said it would expedite its review of the drug. The meeting of the FDA panel will be key to Tysabri's future, because the group is made up of medical experts who will decide if the pros outweigh the cons. The committee will then make a recommendation to the full FDA, which isn't obligated to follow the suggestions of its panels, but it usually does.