Derivation of scalable clinical grade human embryonic stem cells under current Good Manufacturing Practice conditions using conventional and single blastomere biopsy protocols

Funding Type: 
New Cell Lines
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 
New human embryonic stem cell lines for the clinical repair of damaged or missing tissues. Human embryonic stem cells have great therapeutic potential because of their ability to grow indefinitely and their potential to produce almost any cell type and tissue in the body. This makes it theoretically possibly to produce an inexhaustible supply of cells, tissues, and organs to repair damaged or diseased tissues in patients. However, several problems exist that must be resolved before human embryonic stem cells can be used for human therapies. Almost all the currently available human embryonic stem cell lines have been grown under conditions using animal products. This is problematic because the human embryonic stem cells may have been contaminated with animal viruses, prions or other animal pathogens making them unsuitable or, at least, suboptimal for therapeutic use in humans. We propose to develop a cell culture system to grow human embryonic stem cells that does not use animal products. Furthermore, in conventional methods used to develop new embryonic stem cell lines, the embryo is typically sacrificed, which raises an ethical concern about the destruction of the embryo. We propose to develop new human embryonic stem cell lines by removing a single cell from the embryo to develop a new embryonic stem cell line. Since human embryonic stem cell can divide indefinitely, we can expand that single cell into a new embryonic stem cell line. By removing only a single cell from the embryo, the embryo will remain alive, obviating the ethical concerns about sacrificing embryos to produce new human embryonic stem cell lines. Finally, many cell types in the body do not divide (duplicate themselves) well when grown in tissue culture plates, thus, making it difficult to produce enough cells for use in patients. We propose to develop new tissue culture systems to grow human embryonic stem cell in mass cultures so that they can be expanded in sufficient quantities and then converted into the desired cell type for use in patients.
Statement of Benefit to California: 
California, like much of the United States, is facing a staggering challenge to its health care system. The large investments made in recent decades by the National Institutes of Health (NIH) have largely ignored the problems of age-related degenerative disease. As a result, increasingly physicians are treating the chronic, debilitating, and therefore expensive diseases associated with aging. This is made all the worse by the demographic wave caused by the entry of the Baby Boomers into retirement. It is estimated that by the year 2010, the Baby Boomers will be 25 percent of the population of California. By 2020 they will be approaching 64 years of age. As a result, the percentage of the elderly in California is expected to grow from 14 percent in 1990 to 22 percent in 2030. (Source: California Department of Finance, Population Projections 1993). Many of the chronic devastating diseases of an aging population are the degenerative diseases. Generally speaking, degenerative diseases are those diseases caused by the loss or dysfunction of cells. Examples include osteoarthritis (loss of cartilage cells that protect the ends of the bones), Parkinson’s disease (the loss of dopaminergic neurons), osteoporosis (dysfunction of osteoblasts), macular degeneration (dysfunction of retinal pigment cells) and so on. More significantly, the loss or dysfunction of cells in the heart (or the vessels that supply the heart with blood) results in heart disease, the most frequent cause of death in California. In 2001 (the most recent year data is available) heart disease caused 68,234 deaths (29% of all the deaths in the state). Stroke is also a vascular disease and the third leading cause of death in California. In 2001, stroke caused 18,088 deaths (8% of all of the deaths in the state). Regenerative medicine represents the effort of cell biologists to invent a new approach to the problem of degenerative disease. Human embryonic stem (hES) cells have the potential to become all of the cells in the human body, and their unique properties give researchers the hope that from these primitive cells new therapies can result that may be available in time for the looming health care crisis. It is estimated that are over 200 cell-types in the adult human and hES cells are capable of making all of these. However, to turn this new technology into actual therapies that can alleviate human suffering, researchers need new tools to generate large numbers of purified cell types. This proposal describes a project to derive new embryonic stem cells lines that are clinical grade and suitable for therapeutic use in regenerative medicine.

© 2013 California Institute for Regenerative Medicine