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The noted astronomer Carl Sagan had a saying. Extraordinary claims require extraordinary evidence. Last week, a physician made the extraordinary claim that he had an effective treatment for sepsis, sometimes known as blood poisoning. It kills around 300,000 Americans every year, so even a modest improvement in survival could save tens of thousands of lives. NPR's Richard Harris looks at how scientists are now planning to test this extraordinary claim.
RICHARD HARRIS, BYLINE: Sepsis is the leading cause of death in the hospital. It's an overwhelming inflammation usually triggered by an infection. Because it's so often deadly, people took notice when a well-regarded critical care physician at Eastern Virginia Medical School announced he'd found an effective treatment. Paul Marik's protocol involves intravenous steroids and vitamins which he says saved many of his patients. Former Food and Drug Administration Commissioner Robert Califf says, prove it.
ROBERT CALIFF: I mean nothing has worked despite all the great ideas that people have had, often ballyhooed with observational data like this, claiming that it's a big effect. So I'd say the odds are still that it doesn't work, but every once in a while, something works in an unexpected way.
HARRIS: With 800 people a day dying of sepsis in the United States, it would be best to know as rapidly as possible whether Dr. Marik has found a significant improvement to sepsis treatment. The way to do that is to run a carefully conducted study, a randomized controlled trial where some people get the new treatment, others get standard treatment and neither patients nor doctors know which is which.
CALIFF: It would take a very small randomized trial done by people independent of him to prove it. And then the whole world would benefit.
HARRIS: Knowledge is power, but this won't be quick. Even under ideal circumstances, it could take two or three years to plan, fund and run a trial like that. Some doctors aren't waiting. David Carlbom, medical director of the sepsis program at Harborview Medical Center in Seattle, heard Marik give a talk about his protocol and decided to offer it to his patients.
DAVID CARLBOM: Because of the potential benefit and I think very few harms, I talk to families when I'm caring for a septic shock patient and discuss this with them and get their consent to trying this therapy.
HARRIS: His view after treating five patients is strictly impressionistic, not scientific. But at least some patients responded well.
CARLBOM: I don't know if it's the medicine. Maybe it's just us taking good care of them. Maybe it's just them and their disease. But I was a little surprised that they actually sort of did seem to heal faster from their septic shock.
HARRIS: Another early adopter is trying this method at the University of South Florida in Tampa. Dr. Mark Rumbak is an old friend of Marik's and heard about the treatment months ago. Rumbak says he's tried it on 30 or 40 patients, most of whom did well.
MARK RUMBAK: Not only are they doing better, but they do better quickly. Within 24, 48 hours, you already see the patients responding. It's actually quite nice. It's quite dramatic. They respond fairly quickly.
HARRIS: But again, these are anecdotes, so both of these doctors would like to see the results of careful studies. Many others say they'll wait for that data before changing how they treat sepsis. Dr. Gordon Bernard at Vanderbilt has been studying sepsis for 30 years.
GORDON BERNARD: I'm in the camp that says, you know, I've seen things that looked interesting many, many times over the decades that I've been doing this, and none of them have panned out. And so this is just one more.
HARRIS: Bernard says wishful thinking could have led Dr. Marik to conclude that only 1 out of his 150 patients had died of sepsis. More people did die but reportedly of other causes.
BERNARD: You could spend all day long adjudicating some of these cases as to whether they died of sepsis or something else.
HARRIS: That question would be answered in a rigorous study. Califf, now at Duke University, says it doesn't always pay to be skeptical. He remembers that it took more than a decade for doctors to believe Australian scientists who discovered that stomach ulcers can be treated with antibiotics.
CALIFF: We always have to be aware that every once in a while, something works, and we don't want to miss that opportunity.
HARRIS: Dr. Marik says he's hoping to move forward quickly with a controlled clinical trial. Richard Harris, NPR News.
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