How to Achieve a Good Phage Therapy Clinical Trial?

Abstract

Modern and well-conducted clinical trials in phage therapy are counted on the fingers of a single hand. Needless to say, individual case treatments are numerous, especially in Eastern European countries like Russia, Georgia, or Poland. But most of the scientific evidence trials are missing or considered useless because of the current, pragmatic, and daily medicinal practice of phage therapy in these countries. However, in Western countries, phage therapy disappeared from pharmacopoeias for most than three decades and bacteriophages are unknown to the young generations of clinicians. The clinical knowledge data base needs to be rebuilt according to the most recent evaluation criteria.

This book chapter aims at providing supportive clinical and patient case data, but especially to provide practical recommendations for performing a phage therapy trial in line with Good Clinical Practices (GCP). A special focus is given on one-size-fits-all clinical trials in comparison to the recent regulatory evolution leading to personalized phage therapy treatments after preliminary diagnostic (phagogram). In addition, the authors are trying to open perspectives to speed up the recruitment process and reduce the length of phage therapy trials, by taking an example on the evaluation of personalized treatments in immuno-oncology.

The choice of primary endpoint tailored to the self-propagation properties of bacteriophages is also studied, in the context of Minimum Inhibition Concentration (MIC) standard used for antibiotic evaluation and often recommended for other anti-infective products, even if inappropriate.