Submit Manuscript  

Article Details

Can Untargeted Metabolomics Be Utilized in Drug Discovery/Development?

[ Vol. 17 , Issue. 24 ]


Gary W. Caldwell* and Gregory C. Leo   Pages 2716 - 2739 ( 24 )


Untargeted metabolomics is a promising approach for reducing the significant attrition rate for discovering and developing drugs in the pharmaceutical industry. This review aims to highlight the practical decision-making value of untargeted metabolomics for the advancement of drug candidates in drug discovery/development including potentially identifying and validating novel therapeutic targets, creating alternative screening paradigms, facilitating the selection of specific and translational metabolite biomarkers, identifying metabolite signatures for the drug efficacy mechanism of action, and understanding potential drug-induced toxicity. The review provides an overview of the pharmaceutical process workflow to discover and develop new small molecule drugs followed by the metabolomics process workflow that is involved in conducting metabolomics studies. The pros and cons of the major components of the pharmaceutical and metabolomics workflows are reviewed and discussed. Finally, selected untargeted metabolomics literature examples, from primarily 2010 to 2016, are used to illustrate why, how, and where untargeted metabolomics can be integrated into the drug discovery/preclinical drug development process.


Drug Discovery, Preclinical, Clinical, Metabolome, Metabolomics, GC/MS, LC/MS, NMR, Platform Comparison, Targeted, Untargeted, Footprinting, Flux, Univariate, Multivariate.


Janssen Research and Development, 1400 Welsh and McKean Roads, Spring House, PA 19477, Janssen Research and Development, Welsh and McKean Roads, Spring House, PA 19477

Graphical Abstract:

Read Full-Text article