From Chicago Sun Times
Promising MS drug should not be withheld
February 18, 2006
BY ROBERT M. GOLDBERG
Bartira Tiburtius wakes up at night frightened. In 2001 the language teacher who lives in Chicago was diagnosed with multiple sclerosis, a disease where myelin, the protective insulation of the nerves necessary for healthy brain function, and then the nerves themselves, are slowly destroyed. That year, she had two horrible relapses where, as she recalls, "I lost my sight, I lost my ability to think clearly. I couldn't smell or taste food. I was waking up and not remembering what had happened. I was afraid to tell my doctor.''
She was already on a drug called Avonex when her doctor enrolled her in a trial for a new medicine, Tysabri. "After a few months, my balance returned, all the fatigue and horrible double vision went away."
Bartira wasn't alone. Dr. John Foley, chief of Neurology at Intermountain Health System in Salt Lake City, conducted studies with Tysabri and said he was amazed when he saw patients who had been wheelchair-bound by MS actually get up and take steps on their own after being on the drug for a short time. Another patient actually walked up two flights of stairs -- something he hadn't been able to do in two years, according to Foley. He recalls thinking: "This is anecdotal. But then we looked at the results after a year of experience. There were not only fewer relapses, but we also found a major reduction in enlarging or new lesions. I can't tell you how excited we were about this.'' Indeed, on average, Tysabri cut in half the risk of losing the ability to see, walk and remember.
Based on such robust results, the FDA approved Tysabri in November 2004 instead of waiting longer and asking for more people to be enrolled in more clinical trials. But last February, Tysabri was voluntarily withdrawn from the market after someone taking it in combination with Avonex, another MS drug, died from progressive multifocal leukoecephalopathy, a rare neurological disease. (PML is a problem only for people with compromised immune systems and is caused by a virus that is present in a dormant state in most people.)
A safety review was conducted by an independent committee of PML experts. Bartira was one of those evaluated as part of the review. She understood the need for the scrutiny. But she also remembers thinking at the time that other MS drugs carry dangerous side effects too.
Moreover, as Kevin Kam of Marketocracy notes: "[The study found] there is a 0.1 percent risk of contracting PML if you use Tysabri but so far, no cases of PML have turned up in patients who took Tysabri alone. To put this risk in perspective, a recent study showed that the mortality risk for aspirin is 0.2 percent." Meanwhile, MS causes about 53,000 relapses yearly and 5 percent of relapses cripple or kill their victims.
Patients considering Tysabri would be tested for PML through brain MRIs and molecular diagnostics. Data about how this and other MS drugs will be used to predict genetic and individual responses to a combination of MS medicines. That means patients will have continuously better information about benefits and risks over time.
It's hard to see why people with MS, armed with such information and new scientific tools, shouldn't be able to take Tysabri. Even the FDA acknowledges that a one-size-fits-all approach to drug approval is outdated in an era of individualized post-market tracking and personalized medicine. Its new approach to drug approval, called the Critical Path, relies on the same kind of personalized approach to drug approval and post marketing monitoring that Bartira and other MS patients have just gone through.
Bartira and Foley are hopeful the FDA lets MS patients have Tysabri back when the agency votes on its return to the market in March. Incredibly, some want to keep Tysabri from Bartira and other MS patients forever. Merrill Goozner, a senior fellow at Public Citizen, a group that lobbies against speedy approval of new drugs, said, ''Was the medical need for this drug [Tysabri] so serious, was its promise so promising, that it outweighed the need to take it through the normal clinical trial process?''
I asked Bartira how she would respond. Her answer was feisty, filled with hope, not fear or anger: "If someone told me Tysabri would take 10 years off my life, but make me feel like the way I am, I would do it. The decision is mine. Not my doctor's or the politicians. It is mine." Let's hope the FDA feels the same way.
Robert M. Goldberg is director of the Manhattan Institute's Center for Medical Progress in New York City, and recently testified before the U.S. Senate on health care policy issues.
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