What is the role of the Human Microbiome?
The microbial community that lives on and in the human body exerts a crucial role on human metabolism, function and immunity. The beneficial impact of the microbiome on human health is under intensive research, as is demonstrated by the increasing scientific literature published during the last 10 years. Assessment at PubMed, the main scientific literature data base, showed that publications focused on these themes (microbiome and human health) was less than 10 publications a year in 2007 and reached around 500 publications in 2018.
Microbiome Therapeutics Pipeline, R&D Innovation
The extensive research on the human microbiome is leading to the discovery and development of novel microorganisms derived from our microbial symbionts. This tendency can be detected in the cumulated number of patents related to microbiome and cancer research within the microbiome space that can be extracted from consolidated multiple patent databases (Fankhauser et al, 2018).
It is not surprising, therefore, that there is growing interest in pursuing ways to treat or reduce the risk of disease by altering the microbiome. There are multiple approaches that are proposed to alter the microbiome including (but not limited to) prebiotics, live microorganisms, small molecules and faecal transplantation. Assessment of these approaches requires both pre-clinical and clinical studies, conducted to a standard which is acceptable to both the scientific and commercial community. This work should be conducted to ICH-GCP standard.
What are Human Microbiome Therapeutics?
Traditional Probiotics confer health benefit on the host that are preventative, rather than therapeutic, and are not based on microbiome analysis or understanding of individual microbiome imbalances. The next generation of probiotics may belong to less familiar and formerly uncharacterized strains of microorganism with unusual and in some cases valuable properties. As our understanding of the microbiome develops, there is a growing interest in the application of microbiome technologies in personalised medical foods which bridge the gap between the food and the pharmaceutical markets (O’Toole et al, 2017, Sun et al, 2016).
These new strains aimed at improving disease and symptoms thereof have been termed recently “Live biotherapeutic products (LBP). They are medicinal products containing live micro-organisms (bacteria or yeasts) for human use. LBP are administered orally or vaginally and are available in different forms. LBP may contain one or multiple microbial strains from the same or different species of micro-organisms.
Microbiome Therapeutics, the Regulatory framework
Regulatory institutions such as EFSA and FDA require scientific evidence when associating a health claim to Microbiome based products, and the European Directorate for the Quality of Medicines (EDQM) play a major role in ensuring the quality of biotherapeutics, including biosimilars, thereby contributing to overall patient safety. The Ph. Eur. standards are designed to meet the needs of stakeholders, including industry, Official Medicines Control Laboratory (OMCLs) and regulatory authorities.
Microbiome Clinical Trials, a necessary step to clinical validation
The evidence of the efficacy and tolerability needs to be obtained through human interventional studies, that need to be designed following the guidance provided by these institutions. The main requirements of the study outcomes are the characterization of the strain, the identification of the health benefit and the demonstration of the same in a normal healthy or pre-disease target population.
The quantity of living bacteria or yeasts is determined by counting viable and culturable micro-organisms of the intended strain(s).” Fecal microbiota transplantation (FMT) and products intended as gene therapy agents are not covered by the same standards.