UK Stem Cell Bank Application for Phase V Funding 2017-2020

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Regenerative medicine is a new and exciting field in clinical science with great promise for the treatment of traumatic injury and a range of currently incurable serious human diseases. Recent years have seen dramatic progress in this area, generating a diverse and complex range of potential therapies for which there is strong pressure to progress to clinic without delay. Such rapid development means that there is an ever shortening 'proving' period between translation and application of stem-cell based medicines in which to gain key understanding of the crucial issues for product safety and efficacy. Regenerative medicine development is still largely done by SMEs and academic research groups, often with limited resources and lack of internal regulatory science support. Close interaction within the regenerative medicine community to utilise experience from expert groups, is therefore crucial to long term success for this industry in the UK. The UK Stem Cell Bank (UKSCB) has established a long and effective track record in supplying quality assured and ethically sourced starting cells for advanced therapies and providing scientific advice and training to industry, academia and policy makers. As clinical trials in cell therapy roll-out internationally, such support activities will be vital in avoiding wasted time and resource on poor quality starting materials and to assist the smooth and safe progression of new cell therapy products to widespread use and acceptability.The ultimate intent of the UKSCB is to sustain crucial ethical and scientific rigour in the use of human stem cell lines, whilst utilising its expertise and knowledge to enhance UK translational research to facilitate the safe and effective delivery of advanced cell therapies.The primary objectives of the UKSCB are:1) To curate samples of each ethically approved human embryonic stem cell (hESC) line and assure suitability of appropriately derived lines for clinical use under UK and EU regulation.2) To enhance the efficiency, capacity and utility of the UKSCB clinical grade (EUTCD-Grade) cell line resource by further streamlining of banking procedures. This will include closer coordination with expert UK and international research groups to expand data sets to promote the value of UKSCB-held lines for human application.3) To deliver a translational research programme supported by NIBSC core funding focused on: 1) developing enhanced banking processes and 2) crucial standardisation and safety issues for clinical use of EUTCD-grade lines. In particular the UKSCB will seek to:a) Continue to improved reproducibility of qualified methods for culture expansion, characterisation and cryopreservation of hESC cell lines including naïve hESCs and those from gene edited embryosb) Implement better defined banking conditions for clinical grade stem cells lines on synthetic culture surfacesThe second UKSCB research theme on standardisation and safety will:a) Establish, as part of a NIBSC-led work-stream, a robust and internationally acceptable advanced risk assessment process for vCJD contamination of stem cells together with detection protocols for EUTCD-grade linesb) Develop methods and reference materials to assist in establishing metrics for reproducibility and acceptability of stem cell cultures.4) To utilise maintain close collaboration with cutting edge researchers and key industry partners to leverage delivery of UKSCB scientific objectives for the benefit of UK regenerative medicine and ensure the UKSCB maintains its reputation as the premier source of high quality stem cell lines for clinical application.5) To deliver an ongoing programme of training, workshops and advice based on UKSCB and NIBSC expertise in stem cell bioprocessing and regulatory science.6) To develop a wider programme of communications to enhance connectivity with the stem cell and regenerative medicines communities.