Can Untargeted Metabolomics Be Utilized in Drug Discovery/Development?

Area of Science:

• Pharmacology
• Biochemistry
• Drug Discovery

Background:

• High attrition rates in drug discovery and development pose significant challenges to the pharmaceutical industry.
• Untargeted metabolomics offers a powerful analytical strategy to address these challenges.

Purpose of the Study:

• To review the practical value of untargeted metabolomics in advancing drug candidates.
• To highlight its role in target identification, biomarker selection, efficacy assessment, and toxicity prediction.

Main Methods:

• Overview of pharmaceutical and metabolomics workflows.
• Review and discussion of the pros and cons of key workflow components.
• Illustration using literature examples (2010-2016).

Main Results:

• Untargeted metabolomics can identify novel therapeutic targets and validate existing ones.
• It facilitates the selection of translational metabolite biomarkers for drug development.
• Metabolite signatures can elucidate drug efficacy mechanisms and predict toxicity.

Conclusions:

• Untargeted metabolomics provides critical decision-making value throughout the drug discovery and preclinical development process.
• Its integration can improve the efficiency and success rate of bringing new drugs to market.
• This approach supports the identification of novel targets, biomarkers, and understanding of drug action and safety.