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

我们对人类多能干细胞（hPSCs）生物学的日益了解，为大量创伤性损伤、遗传缺陷和退行性疾病患者提供了前所未有的机会，可以产生细胞来修复或替代任何类型的细胞。它还为研究人员提供了一个强大的研究工具，以开发人体组织模型来研究人类疾病，开发新药并调查其安全性。以有效和稳健的方式在基础科学和医学中实现这些进步的途径是建立一个坚实的结构，从道德来源、可靠和适当表征的源材料（细胞和对照）开始。它还需要各种方法，使关键的科学和医学发展能够为新的医疗产品和干细胞的新应用提供渠道。UKSCB在英国再生医学计划中的一个关键功能将是提供精心准备和经过道德来源的人类干细胞系，这些干细胞系是在英国专门为临床使用而生产的。UKSCB将把这些胚胎干细胞（hESC）库作为集中的英国资源，用于细胞治疗（即符合英国和欧洲法规），并将其提供给英国和国际细胞治疗界。具有一系列对研究人员有价值的遗传和物理特征的干细胞系也将被UKSCB储存并提供给国际研究界。这一银行计划还将通过释放其自身的饲养细胞库存用于常规干细胞培养来支持研究界。UKSCB还将提供一个转化研究项目，重点关注：1)改善银行流程；2)干细胞培养临床使用的标准化和安全问题。UKSCB研究的第一个主题将通过以下途径增强其细胞库功能：1)优化干细胞系的培养扩增、表征和冷冻保存方法；2)培养用于临床级干细胞系的新人类饲养细胞。在标准化和安全性的第二个主题中，UKSCB将使用尖端的基因组测序技术来开发临床级细胞中微生物剂和突变的筛选方法，并开发方法和控制材料，以协助建立用于干细胞培养的可重复性和可接受性的指标。该研究计划由UKSCB的核心MRC资助，将由现有的合作和额外的资金支持，并将通过目前正在评估的新拨款申请大幅扩展（特别是在自动化和改善干细胞表征方面）。其他工作将包括基于UKSCB在干细胞培养、保存、表征和监管问题方面的经验和专业知识的积极培训、教育和建议计划。这将通过与英国和国外的其他干细胞库和干细胞研究机构合作获得补充资金。通过与其他国家的研究人员和干细胞库的积极合作，以及其在干细胞库和细胞治疗监管科学问题方面的领导地位，UKSCB将保持和发展其国际形象。这将用于促进外国实验室和公司与英国再生医学界之间的联系，以促进合作和外来投资。总之，UKSCB提案将为英国胚胎使用监管提供基本组成部分，这为英国赢得了坚实而有力的道德监管声誉。此外，它将确保在技术、安全和伦理问题上获得至关重要的基础支持，这对于将研究方法转化为“首次人体”研究的早期阶段至关重要，以确保通往新细胞疗法的安全可靠途径。

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.