Centers for disease control and prevention
Microbiome clinical trials - Which positioning for Europe?
Among the ~2,100 microbiome clinical trials listed on the clinicaltrials.gov global database from 2000 to 2017, 35% of the studies are conducted in Europe, followed by 33% in the USA. China is third with only 5% of the overall number of studies. Nonetheless, the number of Chinese clinical studies is thought to be underestimated. Indeed, after merging the previous database with the Chinese Clinical Trials Registry, the number of Chinese microbiome clinical studies is twice higher representing about 10% of the overall microbiome studies.
Europe was responsible for the initiative of the first trial mentioning microbiome in 2000, while the USA tackled the subject in 2006 and China in 2009. Moreover, Europe represents 32% of the publications on the microbiome listed on PubMed from 2006 to 2017, versus 23% for the USA and 9% for China.
Microbiome research in Pharma industry and therapeutic applications
Initially, microbiome therapeutic areas of interest have been gastrointestinal diseases (irritable bowel syndrome, inflammatory bowel disease, and Crohn’s disease) and metabolic diseases (obesity and diabetes). They respectively represent 20% and 15% of the overall number of microbiome trials performed in Europe and the USA (2000-2017 period).
In recent years, the potential applications for microbiome have been extended to other therapeutic areas. Based on the number of clinical trials (Clinicaltrials.gov), the therapeutic areas that progress the fastest are central nervous system diseases, infectious diseases and oncology The number of clinical trials in oncology, including a microbiome analysis, is growing at a fast pace in Europe with +41% p.a. since 2011, but it is still behind the USA in volume. Indeed, microbiome trials in oncology already represent 7% of the overall number of trials conducted since 2000 in the USA while it represents just 2% in Europe.
The first microbiome-based therapeutic treatments are already emerging, such as the fecal microbiota transplantation (FMT). Most of the interventions consist of introducing the microbiota from a healthy donor into a patient. The FMT is successfully used to treat Clostridium difficile infections (CDI) but it could also be applied to counteract gut dysbiosis following heavy treatments like chemotherapy. For instance, Maat Pharma, OpenBiome, or Rebiotix are biotechs developing FMT solutions. It requires the access to a biobank with characterized feces from healthy donors. However, the legislation around FMT is still to be defined. In the USA, it is classified as a biologic product and a drug only for CDI. In Canada, it is registered as a new biologic drug and its use is restricted to clinical trial. There is no regulation at the European level, up to date the countries are setting their own rules. In France, it is considered to be a drug and aside from exceptional circumstances, FMT should only be administrated under a clinical trial. However, in the UK, the National Institute for Health and Care Excellence (NICE) allows the use of FMT in the NHS (National Health Service) for patients with recurrent CDI who are not responding to traditional therapies. With more clinical trials, regulations about FMT therapeutic solutions should be revisited in the future.
Besides the FMT, the Pharma industry is also interested in other microbiome therapeutic solutions, such as the ‘phagotherapy’, the antimicrobial peptides, and therapeutic adjuvant.
Pharma industry interest on Microbiome and human health is booming. Europe is at the forefront of the human microbiome research, as it promotes the emergence of new biotech companies developing microbiome-based products. One major focus is to develop new advanced therapies to fight infectious diseases, especially when multidrug-resistant (MDR) bacteria are involved. FMT is one promising approach. Other ‘human microbiome therapeutics’ are explored such as the phagotherapy or antimicrobial peptides (AMPs). Moreover, microbiome research in oncology and central nervous system diseases are becoming one of the major research focus of the pharma industry. A lot of research still needs to be done to understand the individual responses and variabilities but regulatory issues are also a real challenge
Marc-Olivier Bévierre (Cepton Strategies), Etienne Casal (Biofortis – Mérieux NutriSciences), Murielle Cazaubiel (Biofortis – Mérieux NutriSciences), Françoise Le Vacon (Biofortis – Mérieux NutriSciences), Charles Savoie (Cepton Strategies), Alessandra De Martino (Biofortis – Mérieux NutriSciences).
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1 - Clinicaltrials.gov as of January 2018
2 - Chinese Clinical Trial Registry as of January 2018
3 - PubMed as of January 2018