Future of FMT May Depend on New AGA Registry

Paul Feuerstadt, MD

A Two-Year Study

FMT and Clostridium Difficile

The first participant has been enrolled in the American Gastroenterological Association’s (AGA) fecal microbiota transplantation (FMT) study, planned to be the largest of its kind.Paul Feuerstadt, MD, of the Gastroenterology Center of Connecticut, enrolled the first study participant and will follow up with this patient 4 times throughout the next 2 years. The patient will inform researchers about their condition annually over the course of the study, and Dr. Feuerstadt will share how the patient responds to treatment.The only approved use of FMT is for recurrent Clostridium difficile (C. difficile) infection. A 2013 US Food and Drug Administration (FDA) enforcement discretion ruling allowed doctors to provide FMT without filing the standard investigational New Drug Application (NDA) to treat C. difficile infection, where it’s 90% effective. FMT is also being tested in clinical trials for other indications.

A Frontline Treatment

“The study will also gather data for all usage of FMT for a variety of indications including C. difficile infection, Inflammatory Bowel Disease, Irritable Bowel Syndrome, chronic constipation, obesity, diabetes, metabolic syndrome, non-alcoholic fatty liver disease and neurologic diseases," Feuerstadt said.While FMT is currently the last resort when antibiotic drugs have failed to bring C. difficile under control, it could eventually become the new frontline treatment.

"It’s likely that with time and more experience, FMT will move earlier in the treatment of C. difficile infection," Feuerstadt said when asked about FMT. “Currently, it’s recommended that FMT is performed for the third recurrence or fourth total episode of C. difficile. Some providers are already using this earlier in the treatment algorithm with success, but more information is needed regarding the safety and efficacy before formal recommendations are made to use this earlier in the treatment.”

Treating Other Diseases

This study published in the Annals of Internal Medicine found that multiply recurrent C. difficile cases are increasing at over fourfold the general rate of the infection. Of the 500,000 people in the US who are infected with C. diff annually —at least half will be reinfected at least once. C. diff-related costs are over $5 billion a year.FMT has shown promise for Crohn’s disease and ulcerative colitis, but modifying our intestinal microbiome could help address a broad variety of other conditions.

“Will be able to track usage and outcomes of these interventions and hopefully advance our knowledge and understanding of how this technique might benefit other disease states,” added Feuerstadt.

FDA Approval

FMT may eventually treat Parkinson’s disease, depression, anxiety, and autism by affecting serotonin levels through the gut-brain axis. In 1 experiment, fecal matter from individuals with major depressive disorder had an effect on depression-like behaviors when transplanted into germ-free test animals, when compared to the significantly more positive response to fecal material from healthy humans. “FMT as its own entity being performed independently by practitioners using donors locally identified and screened will likely not be approved by the FDA since there are so many different methodologies and screening methods," Feuerstadt said regarding FDA approval. "It is likely that one of the pharmaceutically produced formulations will achieve FDA approval following the typical pathway including phase 1, phase 2 and phase 3 trials. These include Openbiome, Rebiotix, SERES or Crestovo pharmaceuticals. The key element to this AGA data repository is that it broadly tracks the epidemiologic usage of FMT, the outcomes and any potential short- and long-term side effects.”

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