Addressing the Cell Purity and Identity Bottleneck Through Generation and Expansion of Clonal Human Embryonic Progenitor Cell Lines

The Early Translational Awards, which are funded annually, either lead to a drug candidate for an unmet medical need or address a bottleneck in the development of new therapies. The 16 Early Translational I awards worth $71,570,007 were awarded on 4/29/09. You can learn more about the awards by reading the RFA or reading the press release.

Public Abstract (provided by applicant)

Human embryonic stem (hES) cells and induced pluripotent (iPS) cells, such as reprogrammed skin cells, offer the potential to revolutionize medicine because they can replicate indefinitely and become virtually any cell in the body. They therefore have the potential to provide a limitless source of cells to replace cells lost to injury (spinal cord, skin wounds, etc.) or degenerative diseases like diabetes, Alzheimer

Statement of benefit to California (provided by applicant)

Safety is critical to the development of any new drug candidate and is even more essential when considering cellular therapies where cells can persist in the body for years. Thus, the primary benefit of our proposed project of generating well characterized cell lines and markers for their isolation is to provide a means of manufacturing sufficient quantities of cells with the needed purity to provide safer cell therapies. By providing California researchers with a bank of well characterized intermediate precursor cell lines and cell purification reagents we will help overcome the cell purity and identity bottleneck that is currently stalling the successful translation of basic stem cell research to clinical applications. A key beneficial outcome of our project will be to shorten the time it takes to get stem cell therapies from the research laboratory and into the hands of physicians to treat patients suffering from degenerative diseases and injuries. By accelerating the translation of research to drug approval more Californians that are currently in need of treatment will have the opportunity to benefit from stem cell therapies and Californians will see a more rapid return on their investment in the form of reduced health care costs. Another significant benefit of our proposed project is in the application of pluripotent stem cells for modeling diseases. Our work will provide the cost effective means to purify well characterized precursor cells from various sources of embryonic stem cells including reprogrammed skin cells of different genetic backgrounds and disease states. This will reduce the need for animal models and the cost of disease models for drug discovery. Finally, in addition to our cell bank and cell purification kits, we will provide Californians with a database of cell surface antigens that define the various intermediate cell types that occur during embryogenesis by their lineage and cell fate. This resource will provide California researchers with information that will help accelerate the pace of stem cell research. Thus, our proposed project will help bring stem cell therapies to Californians sooner by directly addressing the critical issue of cell safety and at the same time it will provide valuable resources for accelerating stem cell based drug discovery and our knowledge of human development.