There are millions of people in California who will benefit from the development of cell therapies for the treatment of diabetes, neurological disease, heart disease and other degenerative diseases. Because of their enormous developmental potential, pluripotent stem cells, which include embryonic stem cells and induced pluripotent cells, are considered to be a valuable source for the specific cell types required for cell therapies. A tremendous amount of effort has already been invested in developing stem cell-based therapies, and the first clinical tests of these therapies are expected to begin within a year. Under the regulations of the FDA, the first trials will be “Phase 1”, or safety trials, to determine whether the cells have any negative effects. Only after the safety of the cells has been assured can further tests be performed to determined whether the cells provide medical benefit. The FDA has major concerns about the use of pluripotent stem cell-based therapies, and will require considerable thoroughness in characterizing the cells before they are transplanted and in examining the first cell therapy patients for any signs of adverse effects. The agency is concerned that there is not enough known about how cells derived from pluripotent stem cells will behave after they have been transplanted- whether they will migrate away from the transplant site, continue to divide, be toxic to the patient, or become cancerous.
There are currently no sensitive methods to trace transplanted cells, and this is a critical bottleneck that must be overcome to be able to satisfy the safety requirements. We propose to develop methods that can detect small numbers of transplanted cells within tissues, and determine whether they are behaving normally or are becoming toxic or cancerous. The new methods are based in cutting-edge genomic research that has already enabled breakthroughs in deciphering the human genome and in determining the genetic causes of disease. The technologies will be developed as a partnership between a leading corporation in the development of molecular analysis tools, a highly regarded academic human embryonic stem cell laboratory, and a prominent genome research center. By the time that the Phase I safety studies are underway, we plan to have the tools in place to detect and analyze the transplanted cells, to determine whether the cells are behaving normally or if they pose a risk to the health of the patients.
Statement of Benefit to California:
Pluripotent stem cells hold the potential to revolutionize medicine and health care. Cell therapies may be the only viable treatment for devastating and presently incurable conditions such as diabetes, Parkinson's disease, spinal cord injuries, and many other diseases. California scientists have taken the first steps to clinical applications of pluripotent stem cells through their bold research to develop new ways to derive these cells and to differentiate them into cell types that can be used to replace damaged tissues. But there are tremendous challenges to overcome if cell therapy is to become a reality. Chief among these challenges is the need to be certain that the therapies are as safe as possible; the outcome of the first Phase I clinical safety trials will determine the future of this field of medicine. We propose to develop technologies to assess the outcome of the first clinical trials, by combining the skills of a leading corporation in the development of genomics and epigenomics analysis tools, a highly regarded academic human embryonic stem cell laboratory, and a prominent Genome Center. Our developments will help make cell therapy a standard medical practice, and will benefit California by significantly increasing the options available in the health care system, reducing the long-term health care costs to California, and attracting high-skill jobs and tax revenues.