In January of this year, the New England Journal of Medicine reported that just one stool donation treatment was effective for 13 out of 16 suffering from colitis caused by Clostridium difficile -- one of the most intractable and crippling gut diseases out there. Two of the people whose symptoms didn't respond to the first treatment but received a second infusion (from a different donor) got better then. There were no reported complications. Independent reviewers were so impressed with these results that the study was stopped so that stool donation could be offered to everyone in the study's control group.
When stool is used to prevent, cure, or treat a disease or condition, the FDA considers it to be a drug.
This procedure is a fecal microbiota transplant (FMT), more commonly known as a fecal transplant. FMT instills healthy stool from a donor into someone with a gut condition that has destroyed his or her healthy bacteria. Stool donors can be a family member, friend, or a healthy volunteer whose donated stool to helps to repopulate the gut with healthy bacteria. C.difficile is associated with the deaths of 14,000 Americans a year and incapacitates thousands more. This study further confirms what anecdotal evidence has shown for some time--this treatment works.
So why is the FDA asking that it be kept from those who need it the most by requiring consent forms and lengthy approvals? In a letter dated April 25, 2013, the director of the FDA Center for Biologics Evaluation and Research initially stated that an Investigational New Drug Application, or IND for short, will be required for all FMT procedures. This process makes it considerably more cumbersome and legally risky for a physician to perform. As was pointed out in an personal essay in the New York Times last Sunday, the IND discourages providers from offering FMT even when they know that the benefits outweigh the cumbersome approval process.
When stool is used to prevent, cure, or treat a disease or condition, the FDA considers it to be a drug. A woman does not need an IND to breastfeed a child; human milk promotes growth and development -- certainly a health outcome. Blood is freely administered without the IND process and thousands of lives are saved each year as a result. Why then should an IND be required to administer stool, which is a bodily product just as milk and blood?
After protest from the medical community and the patients whom it serves, the FDA relaxed its requirement for an IND slightly, allowing FMT to be performed in a somewhat wider range of cases--but still not enough to make the procedure one that an informed physician can use easily in his or her arsenal. While this updated guidance may reduce the number of people who must wait for 30 days with abdominal pain, cramping, diarrhea and a reduced quality of life while a federal agency decides that donor stool can or cannot be given, it is a Band Aid solution that does not address the underlying issue of how to classify FMT. In the age of the internet it's likely that many patients won't wait for additional updates from the FDA -- there are plenty of do-it-yourself websites. But should this treatment be a do it yourself endeavor? As a physician, I think not. I know that the FDA agrees. FMT is best be performed under the direction of a healthcare provider.