Metabolomics and Pharmacometabolomics: Advancing Precision Medicine in Drug Discovery and Development.
Eleni V Stolaki, Konstantina Psatha, Michalis Aivaliotis
Abstract
Open AccessMetabolomics and pharmacometabolomics are at the forefront of precision medicine, serving as powerful tools in drug discovery and development. These approaches help address critical challenges in the field, including high clinical trial failure rates, adverse drug reactions, and interindividual variability in drug response. Comprehensive metabolome profiling enables the elucidation of disease mechanisms, identification of drug targets, optimization of therapeutic strategies, and assessment of drug safety and efficacy. It also supports more informed clinical trial design. This review highlights the pivotal role of metabolomics in advancing precision medicine and aims to broaden the perspectives of emerging scientists entering this complex field. Key analytical techniques-namely mass spectrometry and nuclear magnetic resonance spectroscopy-are discussed for their respective strengths and limitations in metabolite identification, quantitation, and structural elucidation. Additionally, analytical separation technologies such as liquid and gas chromatography, ion mobility spectrometry, capillary electrophoresis, and supercritical fluid chromatography are explored for their potential to enhance metabolome coverage, improve analytical efficiency, and reduce costs. Ongoing advancements in instrumentation and computational tools are helping to overcome major challenges in metabolomics, including metabolome complexity, data analysis and integration, and biomarker validation. These developments continue to expand the applications of metabolomics and pharmacometabolomics in both preclinical and clinical research. Ultimately, this review underscores their translational potential in facilitating drug discovery, mitigating risks in clinical trials, and shaping the future of precision medicine.