Fecal Transplants and the FDA ?>

Fecal Transplants and the FDA

by David Tuller, DrPH

Gastrointestinal infections with the drug-resistant bacteria C. difficile have become epidemic in health care settings in recent years, posing a serious threat to weakened and immunocompromised patients. People often suffer from these infections after being treated with antibiotics that wipe out much of their existing gut bacteria, allowing C. difficile to thrive. A different antibiotics regimen resolves most cases of C. difficile, but one in five patients experience multiple bouts, often accompanied by severe nausea, diarrhea and abdominal pain.

At worst, the illness can lead to dehydration, malnutrition and death. The Centers for Disease Control and Prevention (CDC) estimates that 14,000 Americans a year die from it. That’s why the recent good news about a gross-sounding but common-sense treatment, fecal transplants, has arrived just in time. Fecal transplants involve making a preparation from the stool of a healthy person and placing it—along with all the bacterial species it contains—in the gastrointestinal tract of someone else. They’ve proven to be surprisingly effective.

In January, the New England Journal of Medicine published the first randomized trial comparing fecal transplants with antibiotics for treating intractable C. difficile infections. In the study, transplants of donor feces cured 15 out of 16 patients while standard antibiotics cured only three cases out of 13. In fact, the trial itself was ended early because interim results so clearly proved that fecal transplants were more effective against this illness than the current arsenal of antibiotics.

The results weren’t surprising. Many doctors had previously reported success with the treatment—the first mention in the medical literature dates back to 1958. However, the study findings offered the kind of proof only available from a randomized trial. And the apparent effectiveness of the procedure is consistent with emerging understandings about the powerful, health-sustaining role played by the hundreds of bacterial species that thrive in the gut of a healthy individual.

In May, the Food and Drug Administration (FDA) held a meeting about fecal transplants and announced that doctors would need to apply for special permission each time they performed the procedure—a cumbersome and time-consuming process.

In June, after an outcry from doctors who have used fecal transplants to treat C. difficile, the agency reversed itself and indicated it would pursue a hands-off policy, with two reasonable caveats: that doctors should offer fecal transplants only to patients with intractable cases and that they must explain that the treatment is still investigational. (Another study, this one presented at October's annual meeting of the Infectious Diseases Society of America, suggests that ingesting a pill containing a concentrate of fecal bacteria may also alleviate persistent C. difficile. This is an experimental therapy and, as such, not available to consumers.)

The FDA made the right call. Normally, of course, new treatments should undergo more rigorous testing before being introduced. And there clearly is room for legitimate concerns: evidence could emerge that some doctors are hyping fecal transplants as a cure for all sorts of conditions, for example, or the procedure could itself lead to the spread of other pathogenic bacterial species.

But given the current context of an epidemic of life-threatening C. difficile infections that cannot be resolved with standard regimens, the agency is smart to allow doctors to perform a minimally invasive procedure that has proven remarkably successful to date even as research continues.