Since the discovery of induced pluripotent stem cells (iPS cells) in 2006, a great deal of basic research has been done to understand how to produce, manipulate, and utilize the cell type. iPS cells are revolutionizing regenerative medicine, because they represent a potential route for producing patient-specific stem cells for research applications or clinical use. iPS cells also show great promise for phamaco-toxicological screening, by allowing disease modeling and safety assessment of potential new drugs.

iPS Cells in Drug Discovery and Disease Modeling

iPS cells have the potential to transform drug discovery by providing physiologically relevant human cells for compound identification, target validation, compound screening, and tool discovery. They can be used to produce a wide variety of mature human cell types, allowing potential drug compounds to be screened in high-throughput systems using human cells.

Additionally, iPS cells allow for the creation of patient specific cell populations, facilitating in vitro drug testing that replicates the disease conditions of the patient population of interest.  This approach facilitates early determination of whether a specific genetic population will respond well to a drug candidate, a process known as “personalized medicine.”

Currently, all but one of the clinical trials underway with iPS cells involve the creation and evaluation of iPS cell lines from specific patient population to determine whether the cell lines will be a good model for a disease of interest in a specific patient population. The only exception to this is Cynata’s clinical trial underway for GvHD in the UK that involves a MSC-derived iPS cell product. [Read more…]