Increased Adoption Of iPSC-Derived Human Cells For Use In Drug Discovery And Toxicity Testing

By Chris Parker, Vice President and Chief Commercial Officer, Cellular Dynamics International

Traditionally, discovering a pharmacological therapy for a disease involves first investigating the underlying biological processes of that condition and then identifying drug candidates that interfere with those processes.  From here, the most promising compounds are advanced to pre-clinical and clinical development. The goal of this “bench to bedside” approach is to translate bench research to point-of-care patient outcomes as quickly and safely as possible. With dwindling drug pipelines, pharmaceutical companies need new approaches that can re-energize their discovery efforts.  

Significant advancements in our understanding of molecular biology have allowed us to approach disease-specific therapeutic discovery from different angles. DNA sequencing technologies have advanced, providing scientists with the ability to sequence the human genome quickly and cheaper than ever before. This capability has enabled genome-wide association studies (GWAS) to associate disease phenotypes with patient genotypes, thus stratifying patient populations with the objective to identify specific genetic variants that are associated with a disease state. Understanding these genetic variants will allow scientists to identify which functional protein(s) contribute to the emergence of a particular disease, and further, which pharmacological interventions can produce clinically relevant results through highly targeted therapy (“personalized medicine”). While this approach holds much promise in the development of novel therapeutics, the potential of GWAS data is limited because a vast majority of identified genetic variants are not well understood.