Twenty years ago, I was faced with a unique and somewhat unconventional medical request from a colleague – to perform a “poop transplant” on a schoolteacher suffering from recurrent C. difficile infection. Initially skeptical, I eventually agreed and embarked on my first fecal microbiota transplant (FMT) procedure. To my surprise, the patient was cured, sparking my interest in this innovative treatment approach.
Over the years, I transitioned from blending stool in a hazmat suit to using frozen investigational FMT from OpenBiome, a nonprofit stool bank in Boston. The standardized and safe nature of this frozen FMT allowed me to treat hundreds of patients with refractory C. difficile infections successfully. Witnessing the remarkable benefits of FMT firsthand, I became a strong advocate for its use in treating these devastating infections.
However, recent developments have raised concerns about potential restrictions on access to investigational FMT by the Food and Drug Administration (FDA). This regulatory change could have significant implications for severely vulnerable C. difficile patients who rely on this therapy for their survival.
C. difficile infection is a serious bacterial gut infection that affects hundreds of thousands of Americans each year, with a mortality rate of over 10% in elderly patients. Investigational FMT has been the standard of care for severe and fulminant CDI patients for more than a decade, providing a proven and effective treatment option for those who do not respond to antibiotics.
While the FDA has approved live biotherapeutic products for adult recurrent CDI, these drugs are not indicated for the most vulnerable patient populations, including those with severe or fulminant infections. As the investigational new drug status for FMT is set to expire, there is a pressing need for continued access to this life-saving treatment for those who depend on it.
Leading medical organizations, including the American College of Gastroenterology (ACG) and the Infectious Diseases Society of America (IDSA), have recommended investigational FMT for severe CDI patients. Until the FDA approves a safe and effective alternative for these populations, it is crucial to maintain access to investigational FMT for those whose lives depend on it.
In conclusion, as a gastroenterologist with extensive experience in treating C. difficile infections, I urge the FDA to extend enforcement discretion for investigational FMT and ensure that severely vulnerable patients continue to have access to this life-saving therapy. The stakes are high, and the well-being of these patients should be our top priority.
Neil Stollman, M.D., Chief of the Division of Gastroenterology at Alta Bates Summit Medical Center and the East Bay Center for Digestive Health in Oakland, California.
