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.
FMT was being performed in healthcare settings with success before the FDA intervened. Clinicians and patients are calling for the facilitation of FMT; an intervention for many who have very few options.
The January study ratified what some in the medical community have known anecdotally for years: this treatment works much of the time and works quickly. In 1958, Dr. Ben Eiseman, Chief of Surgery at Denver General Hospital published his experience with fecal enemas in four patients with pseudomembranous colitis, likely related to a Clostridium difficile infection. Applying his experiences as an outdoorsman to his patients, he applied his observation of ill animals who consumed the feces of their well counterparts to inspire his trial of fecal enemas in his human patients. While he is remembered for a lifetime of surgical endeavors, mentorship, and teaching, this case series is credited with being the first published description of FMT in a peer reviewed publication in the United States.
With sporadic reports, FMT began to achieve a following as antibiotic associated diarrhea and C. difficile became a more frequent malady. Despite its effectiveness, many are initially repelled by the process. But after suffering for months, many desperate patients who have been treated ineffectively in other ways began to accept FMT and seek it out. Reports of cures and instantaneous improvements in quality of life began to pepper web sites and discussions on patient self-help blogs.
Why this sudden crack-down on a procedure that has restored health and function to those previously incapacitated? Understandably, the FDA wants to prevent the potential complications from do-it-yourself treatments and home-based clinics that they envision practicing unscrupulous and unsterile procedures. With increasing awareness of its benefits for diarrhea that recurs, both patients and providers have shared its successes in social media and peer reviewed publications, creating the need for more thorough study of the treatment, not more roadblocks to its use. As broader spectrum antimicrobial use has become more widespread for superbugs, more and more patients are experiencing antibiotic associated diarrhea, including C. difficile infections. The emergence of a virulent C. difficile strain has forced practitioners to consider alternative management strategies; FMT is a natural -- one that many practitioners have used or would like to use in future.
Who is the ideal stool donor? How should donor stool be tested? How should recipients be monitored? How should stool be administered? Who is an appropriate candidate? None of the crucial questions of good medical practice will be answered with regulations that make FMT difficult to perform and impossible to evaluate. I am a doctor who treats infectious diseases, with a focus on those in the gut, and my greatest concern is not that one of my patients will get an infectious disease from FMT. It is that I will be unable to offer them the best care and they will seek treatment from the black market, where their risk for complication is unknown.