National Center for Advancing Translational Sciences, NIH, Maryland, United States
Abstract: Recent advancements in technologies such as molecular and cellular biology, and omics analyses (RNAseq and mass spectrum based proteomic profiling) have propelled biomarker development into a new era. Molecular biomarkers are important indicators of therapeutics efficacy in clinical trials. We have employed omics analysis using human cells and organoids derived from patient iPS cells for biomarker development. We found that human cells and organoids are useful for disease modeling and are reliable sample sources for biomarker development. We identified a set of biomarkers from patient cells that are correlated with the disease subtypes categorized by its clinical severity. We also evaluated the efficacy of a clinical drug candidate in patient cells using a set of biomarkers identified by omics analyses. The results have demonstrated biomarker development using omics analysis and patient iPSC derived cells is an effective approach for identification of disease phenotypes and evaluation of therapeutics efficacy for development.