• microbiome;
  • Clostridium difficile;
  • microbiota;
  • fecal microbiota transplantation;
  • probiotics


Clinical Development Phases I-III Regulatory, Quality, Manufacturing

Scientific breakthroughs in deciphering the human gut microbiome and the clinical success of fecal microbiota transplantation (FMT) to treat recurrent Clostridium difficile infection (R-CDI) are driving therapeutic advances based on human gut microbiota. Due to the powerful therapeutic capacity of FMT and the keen interest for FMT-related products approved by regulatory agencies it is timely to review the growing field of therapeutics rooted in the human microbiome, emphasizing FMT but also considering probiotics, vaccines, bacteriophages, and bioactive products. The diminishing effectiveness of antibiotics and the increasing rates of antibiotic resistance have renewed interest in finding alternative methods to combat bacterial infections. Despite pharmaceutical investment in developing new and more effective antibiotics, infectious disease experts warn of a compelling need to develop antibacterial agents distinct from antibiotics. Probiotics have been recognized as beneficial to human health for over one hundred years. The most powerful probiotic of all, the gastrointestinal microbiota, houses approximately 100 trillion species of bacteria, many of which produce a wealth of potent components, such as antimicrobial bacteriocins, metabolites, vitamins, and bacteriophages. The success of human gut microbiota in treating R-CDI and restoring gut homeostasis has highlighted the power of “nature's complete probiotic” and is propelling fecal microbiota along a therapeutic biologic regulatory path. Clinical use of FMT in R-CDI has also taught us that other conditions, e.g., ulcerative colitis, characterized by superinfected and dysbiotic microbiomes may benefit from restoring gut homeostasis with normal microbiota, leading to active efforts to develop therapeutics from the human gut microbiome.