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

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)

The clinical application of cell replacement therapy in the US is dependent on the FDA's approval, and the primary objective of the FDA is to protect patients from unsafe drugs and procedures. The FDA has a specific mandate for human gene and cell therapy and since the unexpected deaths in early trials of gene therapy trials the bar for safety in these areas is unusually high. This is a summary of the key findings from the FDA's report on human embryonic stem cell therapy (April 2008): "From the perspective of toxicology, the proliferative potential of undifferentiated human embryonic and embryonic germ cells evokes the greatest level of concern. A characteristic of hESCs is their capacity to generate teratomas when transplanted into immunologically incompetent strains of mice. Undifferentiated hESCs are not considered as suitable for transplantation due to the risk of unregulated growth. Before clinical trials are begun in humans, the issue of unregulated growth potential and its relationship to stem cell differentiation must be evaluated". In order to overcome the concerns about the safety of pluripotent stem cell therapy, we have designed a pipeline of quality control measures that can be applied to any cell type that is being considered for Investigational New Drug (IND) approval by the FDA. The technologies that we will develop under this award will allow rigorous selection and characterization of cells before they are tested as transplants. By reducing the possibility that stem cell therapies will be toxic or cause cancer in patients, we will remove the major barriers to advancement of these therapies to the clinic.

Statement of benefit to California (provided by applicant)

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