Once in a lifetime  pg. 2

Beginning in 1998, Bernard’s team of investigators fielded calls from critical care physicians in 160 centers around the globe to determine, in split-second decisions, if a patient could be included in the trial. To qualify, all of the patients had to be dying of sepsis and not from a primary disease such as cancer or heart disease.

Within two years, it became apparent that Xigris was having a significant impact on survival: among the sickest patients the mortality rate for those who took the drug was 13 percent lower than the rate among those on placebo. An independent board of advisors stopped the PROWESS trial in 2000, because it was no longer ethical to give the placebo.

“It was a huge day,” says William Macias, M.D., Ph.D., medical director of the Xigris Product Team at Lilly. “First there was a brief moment of disbelief. We knew it was a long shot for the overall trial to be successful, but to stop early was even bigger. This was a once-in-a-lifetime experience.”

The PROWESS team had met what had been considered an unattainable benchmark—for every eight patients treated with Xigris, one life was saved. “There are very few drugs for which such a sweeping statement can be made,” says Bernard.

“The company realized the risk of creating and testing biological products,” Macias says. “You have to invest very heavily before you ever know if you have a drug that will be worth the investment.”

Vanderbilt’s involvement was crucial, adds Mark Williams, M.D., medical director for Xigris. “One of the main reasons that PROWESS was successful was because Vanderbilt served as the coordinating center… a triage center under very difficult circumstances,” he says.

Williams estimates that more than 7,500 lives have been saved since 2001, when the FDA approved Xigris. With increasing use, as many as 40,000 lives could be saved each year, he says. Because it can cause bleeding, however, not all severe sepsis patients are candidates for the drug.

“The mortality rate (from severe sepsis) is still too high,” says Bernard. “But to make inroads into something this horrific is wonderful. It says we need to keep plugging away at scientific intervention and drug development.”

Which is exactly what is happening. Williams is currently running a large clinical trial in 110 sites around the world, testing the efficacy of Xigris in critically ill children. He also is examining the drug’s effectiveness in certain cancer patients, with the cautious expectation that treating severe sepsis in this patient cohort may lead to a drop in the overall cancer mortality rate.

The success of Xigris, says Williams, demonstrates how a partnership between university scientists, industry and the FDA can bring a life-saving product to fruition. “Each party has different expertise and strengths,” he says, “and when they work together for the common good, we all see the benefit.”

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