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Harnessing Stem Cells for Drug Discovery: Part 3

Stem cells are being used to develop robust, reliable disease models that are advancing our understanding of underlying disease progression and mechanism.

Stem cells have become an important resource in drug discovery. In the previous blogs for this series we discussed enabling technologies in culturing of stem cells and how stem cells are contributing to compound screening and selection. Here we discuss how stem cells are being used to model disease and elucidate disease mechanisms.

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Stem cells in disease modeling

Deciphering tissue differentiation or disease progression in vivo is a challenge. Historically, investigations required developing an appropriate sampling procedure and assay portfolio to examine key events, signaling pathways, and morphological changes. The ability to examine tissue changes in culture is becoming more sophisticated as the process of cell reprogramming between fibroblasts into induced pluripotent stem cells (iPSCs) and then into specific lineages is being refined. The combination of in vitro stem cell differentiation and improved imaging technology is moving toward a real time understanding of how differentiation processes occur. As we understand normal processes, analysis of the process with varying conditions can extend our understanding of disease mechanisms. Of particular interest is the ability to use human cells with known genetic lesions as the source of the cells. Perhaps opening the door to investigating how a specific gene mutation can cause disease.

As the use of stem cells in disease modeling becomes mainstream, the availability of stem cell derived disease models from providers is increasing. This provides a platform for discovery that has improved reproducibility and quality control, perhaps decreasing the cost of drug discovery as the best compound to proceed to development as a therapeutic can be identified faster.

Several vendors that offer services through Scientist are leading the way in providing stem cell technology. For example, Cell Dynamics International (CDI) offers custom cell lines for many tissues including cardiomyocytes, neurons, hepatocytes, endothelial cells and others. CDI can also create cell lines for a particular genetic mutation, an individual with adverse drug responses, or a unique disease model. Similarly, Lonza offers services from tissue acquisition to cell line production to create biologically relevant cell models.

The potential therapeutic applications for stem cells seem limitless with prospects for replacing cells, tissues and organs. A surprising twist in stem cell use has been their rise in drug discovery and development pipelines. While many of us have expected great things from stem cells for years, their greatest contribution may be through their in vitro use for therapeutic development and understanding disease mechanisms.

  1. Cromley, J. Stem cells rapidly gaining traction in research and drug discovery. Drug Discovery World. Summer 2013.