Abstract

Metabolomics is emerging as a promising systems biology approach for many research fields including functional genomics, disease diagnosis, nutrition science, and drug discovery. Following rapid development in academic and research institutes, metabolomics is drawing an attention in the pharmaceutical industry. This review aims to highlight the practical value of metabolomics in (a) potentially validating more novel therapeutic targets and indications, (b) facilitating decision-making on the advancement of therapeutics in the drug development process, and (c) leading to better patient stratification and cost-effective clinical trials, through the application of LC/MS/MRM-based targeted metabolomics studies at a relatively low cost in pharmaceutical companies. This paper provides a brief history of the development of metabolomics and common strategies for conducting metabolomics studies. The pros and cons of the most important technologies and the major components of metabolomics studies are reviewed and discussed. Finally, selected metabolomics study examples are reviewed to illustrate how metabolomics can be used to simultaneously capture underlying biochemical changes associated with pharmaceutical interventions, and effectively produce more accurate and/or alternative efficacy and ADR biomarkers, thereby, greatly extending our knowledge of disease, protein function and drug action and maximally benefiting the pharmaceutical industry.

Keywords: Biomarker, adverse drug reactions, drug efficacy, drug discovery and development, mass spectrometry, metabolomics, metabonomics, nuclear magnetic resonance, clinical trials, TCA intermediates, gas chromatography / mass spectrometry (GC/MS)