Derivation of clinical grade human embryonic stem cell lines for therapeutic applications

In future it might be possible to use stem cells to treat diseases for which we currently have no cure. These include, heart disease (the biggest killer in the UK), Juvenile (type 1) diabetes, and degenerative neurological conditions such as Parkinson s and Motor Neurone disease. But before we can test potential therapies on patients we must create cell lines that are safe (free of infectious agents and possible animal contamination) and genetically stable. At the moment embryonic stem cells can only be used for reseach. This project aims to efficently produce embryonic stem cells to laboratory standards (called Good Manufacturing Practice, GMP) that allow them to be used for treatment of patients. The cell lines will be deposited in the UK Stem Cell Bank and will be freely available to the medical community.

A major barrier to the progress of using human embryonic stem cells (hESCs) for regenerative medicine is the efficient generation of cell lines to a grade suitable for producing differentiated cell types to administer to patients in clinical trials. The production of such cell lines requires compliance to standards of Good Manufacturing Practice (GMP) and must therefore meet stringent ethical and quality assurance criteria to comply with regulations that govern clinical trials in the UK under the EU Tissue and Cell Directive (EUTCD, 2006). The latter creates a common framework that ensures high standards in the procurement, testing, processing, storage, distribution and import / export of tissues and cells across the EU community. The FDA sets an equivalent standard in North America. We have established a stem cell derivation facility, embryo network and validated protocols to meet this need and here propose to generate a minimum of five master banks of clinical grade cell lines. Our previous record in generating research hESC lines would indicate that we are likely to readily exceed this quota and produce 10 ?15 clinical grade lines over three years. The lines will be deposited with the UK Stem Cell Bank (UKSCB) for distribution to the medical community and used also to advance our ongoing collaborative research programmes to develop applications for macular eye disease and immune tolerised coronary stents to the stage of phase I/II clinical trials within the next two years.