RACHEL MARTIN, HOST:
NPR has learned that hospitals in Michigan and New York this week started testing an experimental treatment that could tackle one of the leading causes of death in the U.S. More than 700 Americans die every day from sepsis. This is when the body rages out of control in a desperate effort to fight infection. Last year, one respected doctor said he had developed an effective treatment. His claim is now being put to the test in large, carefully designed studies. NPR's Richard Harris reports.
RICHARD HARRIS, BYLINE: The researcher is Dr. Paul Marik, and his claims of an effective treatment for deadly sepsis were so audacious, Marik says, some doctors called it snake oil.
PAUL MARIK: There obviously was enormous resistance at the beginning, but it seems that, you know, with time, you know, people started thinking about it and saying, you know what? Maybe this is not so outrageous as we first thought.
HARRIS: The treatment involves a cocktail of intravenous vitamin C, thiamine and steroids. He has a scientific explanation for how it could work and continues to use it at his hospital in Norfolk, Va., where he teaches at the Eastern Virginia Medical School.
MARIK: At last count, over 700 patients have received the cocktail. And, you know, the response is reproducible.
HARRIS: A year later, he's still getting his remarkable results. Doctors elsewhere have also been trying it on their patients, but most are waiting for real, hard science to decide whether Marik's experience is just a fluke. That evidence could come from a gold standard of medical science, a study where some patients get the treatment, others get a placebo and neither the patients nor doctors know who got what.
RICHARD ROTHMAN: We've all been pretty much working 24/7 on this for the past, I guess, like, three to five months.
HARRIS: Dr. Richard Rothman at Johns Hopkins is helping head up one of those careful studies involving at least two dozen hospitals. One of his collaborators at Emory University, Craig Coopersmith, has eagerly been pushing to get the study off the ground since he first read of Marik's success.
CRAIG COOPERSMITH: And so this is something which if proved to be true would absolutely be a game-changer, almost a miracle cure, honestly.
HARRIS: Dr. Coopersmith was in the wait-and-see camp after the first results were published, but he decided to get some hands-on experience with the protocol before the formal launch of the study. He has had patients die, but he also tells the story of one man in his hospital.
COOPERSMITH: So sick that we actually had to flip him upside down to get enough oxygen into his body. His kidneys had failed. His liver wasn't working. His bone marrow wasn't working. And statistically, his chance of dying was nearly 100 percent.
HARRIS: Coopersmith gave the man the Marik cocktail, and his condition quickly reversed. Within a week, he was well enough to leave the intensive care unit.
COOPERSMITH: So it would be something that we would call a miracle cure. What we don't know is, was he going to get better independent of the vitamin C, steroids and thiamine, or did that make him better?
HARRIS: The answer to that question requires a rigorous study. Theirs is funded by a private foundation. It will soon be enrolling hundreds of patients in Atlanta, Baltimore, and elsewhere. His team is not the only one pursuing this question. Doctors at Harvard-affiliated Beth Israel Deaconess got money from another foundation to study the sepsis treatment protocol at 13 hospitals. Dr. Michael Donnino says his hospital has already treated 11 patients, and three other hospitals in Michigan and New York opened their studies for patients just this week.
MICHAEL DONNINO: We're ramping up very quickly here and looking forward to getting results.
HARRIS: He expects to have those in about a year. The parallel studies will help make whatever answer results all the more credible and robust, he says. Reproducibility is the keystone of science.
DONNINO: Having another trial out there, I think, is great.
HARRIS: Both trials have outside experts who will take a peek at the accumulating data from time to time. If the results are as dramatic as Dr. Marik gets in his hospital, or, on the other hand, clearly futile, the studies could be called off early, Coopersmith says.
COOPERSMITH: And, either way, it will change practice across the United States and across the world.
HARRIS: Richard Harris, NPR News.