Current Therapeutic Options for Gut Microbiome Restoration Are Limited 2.0 update

Current Therapeutic Options for Gut Microbiome Restoration Are Limited

Icon of bacteria

The aim of microbiome restoration is to repopulate a diverse gut microbiota to treat disease. For recurrent C. difficile infection, one historic approach has been fecal microbiota transplant (FMT).

Data suggest FMT is efficacious for the treatment of recurrent CDI and reestablishes biodiversity in the gut.1


However, several limitations are found across clinical studies, diagnosis, and treatment.


There is variability across clinical trials—cure rates were lower in randomized controlled trials than in open-label studies (67.7% vs 82.7%, respectively; P<.001)2,3

This inconsistency is due to considerable heterogeneity among randomized controlled trials, with marked differences in study structure, control groups, fecal transplant materials, and outcome assessments.4

Also, most studies assessing the benefits of FMT are retrospective case series or systematic reviews of contrasting sources of microbiota and limited safety data.5-7

Likewise, the lack of product standardization and administration methods has created a situation where a regulated, safe, and effective product is critically needed.8

  • In fact, as recently as March 2020, the FDA issued a warning of the potential risk of serious or life-threatening infections following investigational use of an FMT product supplied by a US stool bank company9

Patients ENROLLED IN CLINICAL TRIALS may not be a true reflection of patients seen in the real world

  • Strict inclusion and exclusion criteria in randomized controlled trials lead to inclusion of a small portion of patients from daily clinical practice, limiting generalizability of results to patients seen in clinical practice2

CDI testing is inconsistent in clinical practice

There is no gold standard: sensitivity and specificity of current tests for CDI are highly variable. Moreover, in the United States, there is no consensus on best diagnostic test for CDI.10,11

  • C. difficile colonization is common and can complicate diagnosis since its toxins can be detected but aren’t necessarily causing disease12
  • Patients can suffer and resolve an active infection, but harbor spores, putting them at risk for another potentially deadly infection12
  • Colonization does not require treatment13

Ferring is committed to exploring the potential of a microbiome-based therapeutic that is studied in a comprehensive population reflective of routine clinical practice for the treatment of recurrent C. difficileicile infection.