Twenty years ago, a respected colleague asked me to perform a “poop transplant” on a local schoolteacher with multiply recurrent C. difficile infection. I was incredulous and skeptical, but my colleague (a friend but a forceful one) persisted until I ultimately acquiesced.
Soon I was in a hazmat suit blenderizing stool and performing my first (subsequently named) fecal microbiota transplant, or FMT. My patient was cured, and I began two decades of care of thousands of patients with refractory C. difficile infection, a therapy that might seem counterintuitive (stool as medicine?) but in fact, is quite biologically rational: C. difficile thrives in a disturbed, damaged colonic microbiome. The transplant repairs the damage so the bacteria no longer thrive.
A few years later, I traded in the blender for frozen investigational FMT from OpenBiome, a nonprofit stool bank in Boston, due to its safety, standardization, and ability to provide frozen “on demand” highly screened human stool for delivery in the colon. I’ve since done hundreds of such procedures, witnessing firsthand the unparalleled benefits they bring to patients with these devastating infections.
Now, the Food and Drug Administration may restrict access to investigational FMT in a way that could potentially jeopardize the health of many severely vulnerable CDI patients.
Clostridioides difficile infection (CDI or often C. diff) is a potentially catastrophic bacterial gut infection that affects 500,000 Americans each year. Among those afflicted, nearly 120,000 will have severe or “fulminant” CDI. There is a mortality rate of more than 10% in elderly patients. Twenty thousand children will contract it each year as well, a number that is increasing.
For more than a decade, investigational FMT has been the standard of care for severe, fulminant, and pediatric CDI patients. Although the FDA approved two live biotherapeutic products for treating adult recurrent CDI in 2022, neither drug is indicated for these specific (and most vulnerable) patients.
The investigational new drug status is poised to expire on Oct. 31. Unless the FDA extends enforcement discretion, these seriously ill patients will go from having a proven and effective treatment to being left behind.
I’m not alone in this opinion. Guidelines from the American College of Gastroenterology (ACG), the American Gastroenterological Association (AGA), the Infectious Diseases Society of America (IDSA) and the Society for Healthcare Epidemiology of America (SHEA) all recommend investigational FMT for severe or fulminant CDI patients not responding to antibiotics.
Until the FDA approves an effective and safe live biotherapeutic product indicated for these patient populations, I urge them to continue to make investigational FMT available for these truly most vulnerable patients. Many of their lives literally depend on it.
Neil Stollman, M.D., is chief of the Division of Gastroenterology at the Alta Bates Summit Medical Center and works at the East Bay Center for Digestive Health, both in Oakland, California.