Novel pharmaceutical discovery and development is an expensive, difficult, and inefficient process, primarily due to the lack of models that accurately present the appropriate condition or that reflect the appropriate response. Ascertaining potential efficacy and off-target toxicity is a large portion of the development process, often accomplished through the use of nonhuman animal models.
Translating the results of such experiments to the human condition is a challenging and imperfect science. Human pluripotent stem cell technology offers the potential to reduce the burden of these factors and a primary goal of Cellular Dynamics International (CDI) is to harness this technology in order to decrease both the time and cost of bringing newer, more effective, and safer therapeutics to market.
Stem cells are pluripotent cells that give rise to all the cells of the body and thus provide a potential in vitro source of any cell type. Human embryonic stem (hES) cells have already been used to generate fully differentiated cells that can be used in both drug discovery and toxicity testing, and automated methods are being applied to their culture and differentiation, thus enabling a potentially unlimited supply of any cell type and reducing the need for animals as test material.
Furthermore, the advent of induced pluripotent stem (iPS) cell technology, whereby fully differentiated cells are reprogrammed to a stem cell-like state, makes it possible to generate any cell type from any genetic background. This article will provide brief examples of current practices and future directions for the use of stem cells in drug discovery.