Advertisement

The AGA’s Fecal Microbiota Transplantation National Registry: An Important Step Toward Understanding Risks and Benefits of Microbiota Therapeutics

Published:January 28, 2017DOI:https://doi.org/10.1053/j.gastro.2017.01.028
      Fecal microbiota transplantation (FMT) is the delivery of stool from a healthy donor to a recipient with the goal of mitigating disease by modifying the structure and/or function of the gut microbiome. This shotgun approach has proven to be a highly effective therapeutic modality for recurrent Clostridium difficile infection (CDI).
      • Kelly C.R.
      • Khoruts A.
      • Staley C.
      • et al.
      Effect of fecal microbiota transplantation on recurrence in multiply recurrent Clostridium difficile infection: a randomized trial.
      • van Nood E.
      • Vrieze A.
      • Nieuwdorp M.
      • et al.
      Duodenal infusion of donor feces for recurrent Clostridium difficile.
      The availability of the therapeutic substrate (ie, stool), together with its ease of administration, has advanced the practice of gut microbiota manipulation in patients more rapidly than our scientific understanding. In addition, the early adoption and expansion of FMT in clinical practice has so far bypassed the standard investigatory pathway (eg, large randomized trials) that typically contributes important short- and long-term safety data. From a scientific standpoint, the success of FMT in the treatment of CDI is an important proof of concept; modification of the gut microbiota can be an effective therapeutic modality in humans. Furthermore, this notion might extend to disease processes other than infection, such as the inflammatory bowel diseases and obesity.
      • Kelly C.R.
      • Kahn S.
      • Kashyap P.
      • et al.
      Update on fecal microbiota transplantation 2015: indications, methodologies, mechanisms, and outlook.
      In contrast, associations between the gut microbiota and other human disorders (eg, obesity, diabetes, cancer) raise legitimate safety concerns about the use of FMT that extend beyond pathogen transmission.
      To read this article in full you will need to make a payment
      AGA Member Login
      Login with your AGA username and password.
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect

      References

        • Kelly C.R.
        • Khoruts A.
        • Staley C.
        • et al.
        Effect of fecal microbiota transplantation on recurrence in multiply recurrent Clostridium difficile infection: a randomized trial.
        Ann Intern Med. 2016; 165: 609-616
        • van Nood E.
        • Vrieze A.
        • Nieuwdorp M.
        • et al.
        Duodenal infusion of donor feces for recurrent Clostridium difficile.
        N Engl J Med. 2013; 368: 407-415
        • Kelly C.R.
        • Kahn S.
        • Kashyap P.
        • et al.
        Update on fecal microbiota transplantation 2015: indications, methodologies, mechanisms, and outlook.
        Gastroenterology. 2015; 149: 223-237
        • Kelly C.R.
        • Kunde S.S.
        • Khoruts A.
        Guidance on preparing an investigational new drug application for fecal microbiota transplantation studies.
        Clin Gastroenterol Hepatol. 2014; 12: 283-288
      1. Food and Drug Administration (FDA). Public workshop: Fecal microbiota for transplantation. 2013; Available: www.fda.gov/biologicsbloodvaccines/newsevents/workshopsmeetingsconferences/ucm341643.htm. Accessed September 26, 2016.

      2. Food and Drug Administration (FDA). Enforcement policy regarding investigational new drug requirements for use of fecal microbiota for transplantation to treat Clostridium difficile infection not responsive to standard therapies. 2013; Available: www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Vaccines/UCM361393.pdf.

        • Hecht G.
        AGA's approach to the microbiome.
        Gastroenterology. 2014; 146: 1119-1121
      3. American Gastroenterological Association (AGA). Clinical research studies. Available: www.gastro.org/patient-care/clinical-research-trials. Accessed September 26, 2016.

        • Aroniadis O.C.
        • Brandt L.J.
        • Greenberg A.
        • et al.
        Long-term Follow-up study of fecal microbiota transplantation for severe and/or complicated Clostridium difficile infection: a multicenter experience.
        J Clin Gastroenterol. 2016; 50: 398-402
        • Baxter M.
        • Ahmad T.
        • Colville A.
        • et al.
        Fatal aspiration pneumonia as a complication of fecal microbiota transplant.
        Clin Infect Dis. 2015; 61: 136-137
        • De Leon L.M.
        • Watson J.B.
        • Kelly C.R.
        Transient flare of ulcerative colitis after fecal microbiota transplantation for recurrent Clostridium difficile infection.
        Clin Gastroenterol Hepatol. 2013; 11: 1036-1038
        • Kelly C.R.
        • Ihunnah C.
        • Fischer M.
        • et al.
        Fecal microbiota transplant for treatment of Clostridium difficile infection in immunocompromised patients.
        Am J Gastroenterol. 2014; 109: 1065-1071
      4. Sinai AppLab. 2016; Available: http://sinaiapplab.org/.

        • Debelius J.W.
        • Vazquez-Baeza Y.
        • McDonald D.
        • et al.
        Turning participatory microbiome research into usable data: lessons from the American Gut Project.
        J Microbiol Biol Educ. 2016; 17: 46-50
        • Petrof E.O.
        • Khoruts A.
        From stool transplants to next-generation microbiota therapeutics.
        Gastroenterology. 2014; 146: 1573-1582