UK Stem Cell Bank Application for Phase IV Funding 2014 - 2017

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Our growing understanding of the biology of human pluripotent stem cells (hPSCs) provides unprecedented opportunities to generate cells to repair or replace any cell-type for large numbers of patients with traumatic injury, genetic defects and degenerative diseases. It also provides researchers with a powerful research tool to develop human tissue models to study human disease, develop new drugs and investigate their safety. The pathway to deliver these advances in basic science and medicine in an efficient and robust way is to establish a solid structure that starts with ethically sourced, reliable and appropriately characterised source materials (cells and controls). It also requires methodologies to enable key scientific and medical developments to feed a pipeline for new medical products and novel applications of stem cells. A key function of UKSCB in the UK regenerative medicine programme will be to make available carefully prepared and tested stocks of ethically sourced human stem cell lines, produced in the UK specifically for clinical use. The UKSCB will prepare these as a centralised UK resource of well characterised human embryonic stem cell (hESC) banks for cell therapy (i.e. compliant with UK and European regulation) and make them available to the UK and international cell therapy community. Stem cell lines with a range of genetic and physical characteristics of value to researchers will also be banked and made available by UKSCB to the international research community. This banking programme will also support the research community by releasing stocks of its own feeder cells to use in routine stem cell culture. The UKSCB will also deliver a translational research programme focused on 1) improving banking processes and 2) standardisation and safety issues for clinical use of stem cell cultures. The first theme in UKSCB research will enhance its cell banking function through i) optimisation of methods for culture expansion, characterisation and cryopreservation of stem cell lines, and ii) generation of new human feeder cells for culture of clinical grade stem cells lines. In the second theme on standardisation and safety, UKSCB will use cutting edge genome sequencing techniques to develop screening methods for microbial agents and mutations in clinical grade cells and also develop methods and control materials to assist in establishing metrics for reproducibility and acceptability of stem cell cultures for use. The research planned under core MRC funding for the UKSCB will be supported by existing collaborations with additional funding and will be expanded significantly (particularly in automation and improving stem cell characterisation) through new grant applications currently under evaluation. Other work will include an active programme of training, education and advice based on the UKSCB's experience and expertise in stem cell culture, preservation, characterisation and regulatory issues. This will be developed with supplementary funding obtained through collaboration with other stem cell banks and stem cell research institutes in the UK and abroad. The UKSCB international profile will be maintained and developed through active engagement with researchers and stem cell banks in other countries and its leadership in stem cell banking and regulatory science issues for cell therapy. This will be used to facilitate links between foreign laboratories and companies and the UK regenerative medicine community to promote collaboration and inward investment. In conclusion, the UKSCB proposal will provide fundamental component of UK regulation on the use of embryos which has gained the UK a reputation for solid and robust ethical regulation. In addition, it will ensure there is vital underpinning support on technical, safety and ethical issues that is critical for the early stages of translation of research methods into 'first in man' studies to assure a safe and secure pathway to new cell therapies.