Human embryonic stem cells have enormous potential for expanding our knowledge of human disease, for developing safer, more effective drugs, and for therapeutic solutions to incurable disease. However, forward progress has been severely slowed by the lack of available hESC lines. Since President Bush's Executive Order on August 9, 2001 banned NIH funding for any hESC lines derived after that time, there has been little funding available for derivation of new lines, with the consequence that the most widely studied hESCs were developed seven years ago . A great deal has been learned about hESCs in the last seven years; most importantly, we have learned that the available hESC lines develop genetic abnormalities during culture, and that the methods used for derivation of the NIH-approved lines are likely to have caused genetic abnormalities. There is also a great need for hESC lines that pharmaceutical companies can use to speed the pace of drug development. The goals of this research program are to increase the number and diversity of hESC lines available to researchers and for clinical applications, and to study the genetic stability of the cells as they expand in culture. Ultimately, we plan to determine whether the genetic changes affect the ability of hESC cells to become certain types of adult cells, and whether genetic abnormalities increase the probability that the cells will form tumors after transplantation. We will thoroughly analyze the new cell lines and provide both the cells and this information to the scientific and clinical communities.
Statement of Benefit to California:
Californians are a large and diverse population that poses unique challenges for the future of medical care. Fortunately, California has a tradition of taking the lead in technology and medical breakthroughs and following through from the first idea to the final product. A major goal for California's supporters of stem cell research is development of stem cell-based products that have medical use, and the mandate for the research community is to provide the best possible fundamental information to help guide clinical applications. We have already laid the groundwork for research that encompasses both federally approved and non-approved human embryonic stem cells (hESC) by establishing a privately funded resource to collect excess embryos that have been donated for research. This embryo bank, which has multiple layers of ethical oversight, currently has more than 1000 donated embryos. We propose to use this resource to generate new hESC lines that reflect the genetic diversity of California. Our long term goals are 1. to speed the development of drugs that are safe and effective for all people, regardless of their ethnicity, and 2. to make human embryonic stem cells as safe as is possible for cell therapy, by ensuring that they retain normal, noncancerous qualities.