Ensuring the safety of cell therapy: a quality control pipeline for cell purification and validation

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. Almost 20,000 Californians await organ transplants, and more than a million suffer from progressive degenerative diseases and injuries such as Alzheimer disease, Parkinson’s disease, nerve-muscle disease such as amyotrophic lateral sclerosis (ALS) and muscular dystrophy, liver disease, diabetes, and spinal cord injury. The possibility of applying cell replacement therapy to these problems could drastically improve the outlook for treatment for the victims. 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 to make these cells as safe as is possible for cell therapy, by ensuring that they retain normal, noncancerous qualities. California scientists have taken the first steps to clinical applications of pluripotent stem cells through their cutting edge research in developing new ways to derive these cells and to differentiate them into cell types that can be used to replace damaged tissues. We propose to take this research to the next step, to prepare the cells for clinical trials. We propose to develop a comprehensive pipeline of quality control technologies that will ensure the safety and purity of cells used first for preclinical testing and later for obtaining IND approval from the FDA for initiating human trials. These technologies can be used for any cell therapy, and will considerably reduce the barriers to development of safe, effective new treatments for incurable disease. This will have a positive effect on the health care of all Californians, reduce the cost of development of cell therapies, and create new opportunities for jobs and industry in the state.