It's hard not to get excited about news of a potentially effective treatment for sepsis, a condition that leads to multiple organ failure and kills more people in the hospital than any other disease.
But there have been so many false promises about this condition over the years, it's also wise to treat announcements — like one published online by the journal, Chest — with caution.
The study, from Eastern Virginia Medical School in Norfolk, Va., reported some remarkable success in treating patients who were at high risk of sudden death.
The story began in January, 2015, when Dr. Paul Marik was running the intensive care unit at Sentara Norfolk General Hospital. A 48-year-old woman came in with a severe case of sepsis — inflammation frequently triggered by an overwhelming infection.
"Her kidneys weren't working. Her lungs weren't working. She was going to die," Marik said. "In a situation like this, you start thinking out of the box."
Marik had recently read a study by researchers at Virginia Commonwealth University in Richmond. Dr. Berry Fowler and his colleagues had shown some moderate success in treating people who had sepsis with intravenous vitamin C.
Marik decided to give it a try. He added in a low dose of corticosteroids, which are sometimes used to treat sepsis, along with a bit of another vitamin, thiamine. His desperately ill patient got an infusion of this mixture.
"I was expecting the next morning when I came to work she would be dead," Marik said."But when I walked in the next morning, I got the shock of my life."
The patient was well on the road to recovery.
Marik tried this treatment with the next two sepsis patients he encountered, and was similarly surprised. So he started treating his sepsis patients regularly with the vitamin and steroid infusion.
After he'd treated 50 patients, he decided to write up his results. As he described it in Chest, only four of those 47 patients died in the hospital — and all the deaths were from their underlying diseases, not from sepsis. For comparison, he looked back at 47 patients the hospital had treated before he tried the vitamin C infusion and found that 19 had died in the hospital.
This is not the standard way to evaluate a potential new treatment. Ordinarily, the potential treatment would be tested head to head with a placebo or standard treatment, and neither the doctors nor the patients would know who in the study was getting the new therapy.
But the results were so stunning, Marik decided that from that point on he would treat all his sepsis patients with the vitamin C infusion. So far, he's treated about 150 patients, and only one has died of sepsis, he said.
That's a phenomenal claim, considering that of the million Americans a year who get sepsis, about 300,000 die.
"So that's the equivalent of three jumbo jets crashing every single day," Marik said.
An effective treatment for sepsis would be a really big deal.
"If it turns out in further studies that this is true, and we can validate it, then this will be an unbelievably huge deal," said Dr. Craig Coopersmith, a surgery professor at Emory University School of Medicine. "But right now we should treat it as a preliminary deal that needs to be validated."
Coopersmith, a top sepsis researcher, is cautious for a reason. There have been hundreds of exciting results from sepsis studies that failed in follow-up research.
A result "can look really exciting when you do it on a group in one hospital with one set of clinicians, and then when you try to validate with a larger group in multiple centers — thus far we've been unsuccessful with anything," Coopersmith said.
Marik may face extra skepticism because the main ingredient is vitamin C, which has attracted a great deal of skepticism over the years. Fowler at VCU was concerned about that when he wanted to launch a study about the role of vitamin C in sepsis.
"Honestly, when we submitted the grant to the NIH, I was fully expecting them to say 'vitamin C? Really?' " Fowler tells Shots.
But he'd made the case that vitamin C wasn't simply a once-trendy antioxidant. When the vitamin is injected, as opposed to taken orally, it can influence the immune system's response to inflammation, he told Shots.
The NIH gave him a $3.2 million grant to run a carefully controlled study of vitamin C to treat sepsis, with all the usual conditions: It includes placebos; the scientists don't know who's getting the active drug; and it's being conducted at several universities.
That study could wrap up later this year. Fowler says some patients in the trial will probably have been given corticosteroids, as Marik's patients were, but that's not a formal part of the study.
Coopersmith said the extraordinary results reported from Norfolk clearly need that kind of careful scrutiny. Marik said he agrees, though he's obviously not waiting for those results to emerge.
Taxpayers and drug companies have spent billions of dollars searching for an effective treatment for sepsis. Drug companies could reap billions in profits if they can develop a successful treatment for this common and often fatal disease.
But profit is not the motive here, Marik says. The ingredients cost about as much as a single dose of antibiotics.
"I obviously have no vested interest," he says. "Nobody's going to make money from this — so this is a very anti-capitalistic thing!"
You can reach Richard Harris at rharris@npr.org.
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