The Untapped Therapeutic Potential of Microbiomics

The microbiome, the complex ecosystem of microorganisms living in and on us, plays a vital role in our health. While the gut microbiome is the most well-known, microorganisms also inhabit the skin, saliva, female reproductive tract, and even areas once thought to be sterile, such as blood and urine.

In recent years, the field of microbiomics has matured significantly, uncovering critical links between the microbiome and various clinical outcomes. Beyond localised effects, many systemic influences such as the gut-brain axis highlight the microbiome's broad therapeutic relevance, even in areas that might initially seem unrelated.

The microbiome’s role in human health presents a vast therapeutic potential, but unfortunately, it remains largely unrealised. One major obstacle lies in the lack of regulatory precedence for handling the unique data and methodologies inherent to the science of microbiomics. This is confounded by microbiomics, unlike more conventional clinical studies, being characterised by:

• A lack of standardisation and reference material 
• Highly multivariate results
• Big data
• Complex algorithms with implicit scientific assumptions

This divergence from conventional clinical studies creates some unique challenges in bridging the gap between the science of microbiomics and the requirements of the regulatory bodies when conducting a clinical trial.

The first efforts for standardising microbiomics research are currently underway, but key challenges remain:

• How do we validate our laboratory methods when no reference material with known nominal values are available for calibration?
• How do we manage the big data and required high-performance computer systems in a manner compliant with regulatory requirements?
• How do we assess the accuracy of the complex profiling algorithms typical of microbiomics, especially when considering the numerous uncultivated microbial taxa?

These questions are not just theoretical; they represent practical hurdles that must be addressed in order to unlock the full therapeutic potential of microbiomics.

This is a frontier field, and those who take the lead in aligning microbiome clinical trials with regulatory standards will likely shape the future of the industry. Establishing a robust approach could open the door to developing new treatments as well as enhancing pre-existing treatments, and set the foundation for microbiomics as a cornerstone of modern medicine.

To understand more about microbiomics, join us for our course on Microbiomics in Clinical Trials.

Published on Jan 13, 2025 by Ella Thomas