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

再生医学是临床科学中一个新的令人兴奋的领域，在治疗创伤性损伤和一系列目前无法治愈的严重人类疾病方面具有巨大的前景。近年来在这一领域取得了巨大的进展，产生了多样化和复杂的潜在治疗方法，因此有很大的压力立即进入临床。如此快速的发展意味着，在干细胞药物的转化和应用之间的“证明”期越来越短，在此期间，人们需要对产品安全性和有效性的关键问题有关键的了解。再生医学的发展仍然主要由中小企业和学术研究团体完成，通常资源有限，缺乏内部监管科学支持。因此，再生医学社区内的密切互动，利用专家组的经验，对英国这个行业的长期成功至关重要。英国干细胞银行（UKSCB）在为先进疗法提供质量保证和道德来源的起始细胞方面建立了长期有效的记录，并为工业界，学术界和政策制定者提供科学建议和培训。随着细胞治疗临床试验在国际上的推广，这种支持活动对于避免在劣质起始材料上浪费时间和资源以及帮助新细胞治疗产品顺利安全地发展到广泛使用和可接受性至关重要。UKSCB的最终目的是在人类干细胞系的使用中保持至关重要的伦理和科学严谨性，同时利用其专业知识和知识来加强英国的转化研究，以促进先进细胞疗法的安全和有效交付。UKSCB的主要目标是：1）根据英国和欧盟的规定，对每一个伦理上批准的人胚胎干细胞（hESC）系的样本进行管理，并确保适当衍生的细胞系适用于临床使用。2）为了提高效率，通过进一步简化银行程序，提高UKSCB临床级（EUTCD级）细胞系资源的容量和效用。这将包括与英国和国际研究小组的专家进行更密切的协调，以扩大数据集，促进UKSCB持有的人类应用产品线的价值。3）提供由NIBSC核心资金支持的转化研究计划，重点是：1）开发增强的银行流程和2）临床使用EUTCD级产品线的关键标准化和安全性问题。特别是，UKSCB将寻求：a）继续提高hESC细胞系（包括幼稚hESC和来自基因编辑胚胎的hESC）培养扩增、表征和冷冻保存的合格方法的重现性b）在合成培养表面上为临床级干细胞系实施更好定义的建库条件UKSCB关于标准化和安全性的第二个研究主题将：a）作为NIBSC领导的工作流程的一部分，针对干细胞vCJD污染的稳健且国际上可接受的先进风险评估过程以及EUTCD级生产线的检测方案b）开发方法和参考材料，以帮助建立干细胞培养的再现性和可接受性的指标。4）利用与尖端研究人员和主要行业合作伙伴保持密切合作，以利用UKSCB科学目标的交付，为英国再生医学和确保UKSCB保持其作为临床应用高质量干细胞系的首要来源的声誉。5）根据UKSCB和NIBSC在干细胞生物处理和监管科学方面的专业知识，提供持续的培训，研讨会和建议计划。6）制定更广泛的交流计划，以加强与干细胞和再生医学界的联系。

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.