Background Information on the Organisation
The MRC’s mission is to improve human health through world-class medical research. To achieve this we support research across the biomedical spectrum, from fundamental lab-based science to clinical trials, and in all major disease areas. We work closely with the NHS and the UK Health Departments to deliver our mission, and give a high priority to research that is likely to make a real difference to clinical practice and the health of the population. MRC’s mission statement is to:
- Encourage and support research to improve human health.
- Produce skilled researchers.
- Advance and disseminate knowledge and technology to improve the quality of life and economic competitiveness of the UK.
- Promote dialogue with the public about medical research.
The MRC is one of seven UK Research Councils, and receives annual ‘grant-in-aid’ funding from Parliament through the Department for Business, Innovation and Skills. Although government-funded, the MRC is independent in its choice of which research it supports.
The MRC supports and advances medical research in three main ways: through our own research facilities, by funding research centres in partnership with universities, and by providing research grants and career awards to scientists in UK universities and hospitals.
MRC website: www.mrc.ac.uk
Annual Research Budget
In 2013/14, the MRC spent £845 million on research.
Within this, MRC spent £328 million on over 440 intramural research programmes in MRC units and institutes, and provided £432 million in research grants to universities, medical schools and research institutions, including £104 million in MRC University Units. MRC spent a further £70 million on post-graduate and post-doctoral training awards.
Stem Cell Budget
MRC is the lead Government agency in funding stem cell research, though significant additional support is provided through three other UK research councils, the Biotechnology and Biological Sciences Research Council (BBSRC) the Engineering and Physical Sciences Research Council (EPSRC) and the Economic and Social Research Council (ESRC), as well as UK research charities and the Dept of Health (DH). The UK Government funded Technology Strategy Board (TSB) also funds stem cell research in the commercial sector. Funding across these various UK agencies is coordinated through the UK Regenerative Medicine Funders Forum (UKRMFF) with a rotating Chair across the member bodies.
- MRC is the major UK funder of stem cell research, contributing approximately 1/3 of the UK research portfolio in this area.
- During the financial year 2012/13 MRC spent £44M on stem cell research.
- MRC aims to commit £150M overall in regenerative medicine (in addition to research on basic stem cell biology) between 2011 and 2015.
Stem Cell Strategy
MRC’s strategic approach in stem cell research is to:
- support fundamental stem cell research in order to that provide insights into developmental biology, the control of cell renewal and differentiation, the mechanisms underlying pluripotency, and the aberrant processes that underlie disease pathology
- provide the necessary infrastructure to provide ethically-sourced stem cell lines to the research community, and promote the development of clinical-grade stem cells and therapeutic delivery;
- further translation towards the clinic through building enhanced links between basic and clinical stem cell researchers, and the promotion of preclinical research, experimental medicine and early phase clinical trials;
- provide support for capacity building, through investment in multidisciplinary training, the encouragement of early-stage clinical scientists, and the recruitment of high achievers from overseas;
- promote interdisciplinary research and cooperation across the domains of academia and industry.
In terms of ongoing strategy development at the UK level, the following activities should be highlighted:
Joint Research Council UK and Technology Strategy Board “A Strategy for UK Regenerative Medicine”
Published in March 2012, A Strategy for UK Regenerative Medicine sets out clear objectives and a delivery plan focused on translating our increased biological understanding into clinical impacts that will benefit both patients and the UK economy.
House of Lords Science and Technology Committee Inquiry into UK Regenerative Medicine.
The Inquiry was set up in July 2012 to examine what the UK is doing well in regenerative medicine
and any barriers to its future development and published in July 2013. Particular challenges identified include complexity of the regulatory landscape, support for clinical trials, manufacturing scale up for mid-late stage clinical development, and adoption by healthcare providers (NHS) including re-evaluation of reimbursement models.
MRC–TSB iPSC Grand Challenge Workshop:
Jointly hosted by MRC and TSB, the workshop discussed the emerging opportunities and challenges in iPSC research and sought to identify how the UK research community could best work together to progress the field. MRC has now published guidance for researchers, highlighting key issues to consider when working in this field and when seeking funding.
Regenerative Medicine Regulatory Workshop:
This workshop, considering regulatory lessons and challenges from emerging cell-based therapies, was held at the Medicines and Healthcare products Regulatory Agency (MHRA) in October 2012 with sponsorship from the Academy of Medical Sciences, Association of the British Pharmaceutical Industry, Economic and Social Research Council, Medical Research Council and MHRA. The workshop
addressed current regulatory complexity in the UK/EU, regulatory uncertainty in therapeutic development and regulatory needs to support
International Workshop on Regulatory Pathways for Cell Therapies, Sept 2013
International differences in regenerative medicine regulation are seen as a potential risk to the field’s development. In 2013 a group of organisations, including the MRC, sponsored the ‘International Workshop on Regulatory Pathways for Cell Therapies’ to ascertain differences and potential areas of convergence in the regulatory approach of different international regulators, with the Report published in July 2014. Other members of the sponsor group included: California Institute for Regenerative Medicine, Alliance for Regenerative Medicine, Economic and Social Research Council, Stem Cell Therapy Catapult, Canadian Centre for Commercialization of Regenerative Medicine.
MRC’s overarching strategy for biomedical research is set out in its Strategic Plan 2014-2018, ‘Research Changes Lives’
Key Stem Cell Centres/Investments
- UK Regenerative Medicine Platform: 5 technology platforms to address key translational bottlenecks in regenerative medicine supported by the Engineering and Physical Sciences Research Council (EPSRC), MRC, and Biotechnology and Biological Sciences Research Council (BBSRC), with aligned research programmes in musculoskeletal (together with Arthritis Research UK), ophthalmic and liver diseases – see below for further detail.
- MRC Centre for Regenerative Medicine in Edinburgh (Director: Charles ffrench-Constant)
- Wellcome Trust/MRC Cambridge Stem Cell Institute in Cambridge (Director:
- UK Stem Cell Bank London – an international resource for ethically sourced human stem cell lines (including clinical grade lines) deposited by research groups from both the UK (as a condition of license for hESC derivation) and overseas. By November 2014 over 130 hESC lines have been approved for deposit in the Bank, from six different countries (341 hESC lines have been approved for use in UK Research, listed in the UK Stem Cell Registry). The Bank includes 33 UK clinicalgrade (GMP) hESC lines.
- Cell Therapy Catapult: The UK government’s Technology Strategy Board (TSB) has established a £50m Cell Therapy Catapult to provide technology, expertise and technical leadership and offer support to companies working on cell therapies, enabling the UK to reinforce its position as a world leader in the science and delivery of healthcare solutions. Access will be provided to equipment and research and development capabilities that may otherwise be out of the reach of companies in this emerging field. The Catapult will aid the rapid commercialization of the outputs of world class research in cell biology, stem cells, clinical medicine and bioengineering, and will build on strong government funding in regenerative medicine and related areas. Highlight this year includes Securing an additional £55m for the establishment of a UK large-scale cell manufacturing centre which will provide vital large-scale manufacturing facilities, helping the UK to retain manufacturing activity, attract inward investment and boost exports.
- EPSRC Centre for Innovative Manufacturing in Regenerative Medicine in Loughborough (Director: David Williams).
- EPSRC, BBSRC and TSB Medical Technologies IKC in Leeds (Director: John Fisher)
- MRC National Institute for Medical Research (NIMR) London – the Division of Stem Cell Biology and Developmental Genetics has programmes that study progenitor cells in early embryonic development and sex determination.
- MRC Clinical Sciences Centre (CSC) London – Section of Epigenetic Development and Cancer has an emphasis on stem cell development and its consequences on gene regulation, DNA replication and chromosome organisation.
- MRC Molecular Haematology Unit Oxford – studies how haematopoletic stem cells develop and differentiate.
Recent Research Achievements and Outputs
Selected press releases since 2013 ISCF Meeting:
Scientists regenerate immune organ in mice (8 April 2014). Scientists for the first time used regenerative medicine to restore the function of the thymus in aged mice, a discovery that could pave the way for future human therapies.
Artificial skin layer grown in lab (25 April 2014). An international team has developed the first lab-grown epidermis – the outermost skin layer – with a functional permeability barrier akin to real skin. Grown from human induced pluripotent stem cells (iPSCs), it offers a cost-effective alternative lab model for testing drugs.
Researchers grow ‘seed’ of spinal cord tissue in a dish (26 Aug 2014) Scientists have for the first time managed to turn stem cells into the specialised cells that go on to form spinal cord, muscle and bone tissue in the growing embryo. Their discovery could lead to a new way of studying degenerative conditions such as spinal muscular atrophy, which affects the nerve cells in the spinal column, and may pave the way for future treatments for this and other neuromuscular conditions.
Scientists reset human stem cells to earliest developmental state (11 Sep 2014) Scientists have successfully ‘reset’ human pluripotent stem cells to the earliest developmental state – equivalent to cells found in an embryo before it implants in the womb (7-9 days old). These ‘pristine’ stem cells may mark the true starting point for human development, but have until now been impossible to replicate in the lab.
To walk again (October 2014) An international team involving Dr Pawel Tabakow, a Polish
neurosurgeon and Geoff Raisman (UCL) demonstrate that transplantation of olfactory
ensheathing cells can restore some function in human spinal injury. This work originated from Raisman’s work at NIMR supported by MRC.
Funding and Strategic Initiatives
UK Regenerative Medicine Platform (UKRMP): This £25M investment by the Engineering and Physical Sciences Research Council (EPSRC), MRC, and Biotechnology and Biological Sciences Research
Council (BBSRC) has two stages.
Stage I (£20M) has created five Hubs to provide UK centres of expertise/knowledge with the necessary critical mass to address key translational challenges and provide new tools, protocols and resources with broad applicability that can be utilised by other UK research groups in academia and
- Cell behaviour, differentiation and manufacturing
Director: Professor Peter Andrews, University of Sheffield
Focus: to lay foundations for a generic translational pipeline from the derivation of clinical grade pluripotent stem cells to potential cell therapies, building upon emerging technologies to optimise processes for consistent, scalable stem cell manufacturing that minimize the appearance of genetic and epigenetic variants, and meet the requirements of clinicians, regulatory authorities and industry for safe and cost-effective applications.
Engineering and exploiting the stem cell niche
Director: Professor Stuart Forbes, University of Edinburgh
Focus: to exploit therapeutically our understanding of the biology of stem niches.
Safety and efficacy, focussing on imaging technologies
Director: Professor Kevin Park, University of Liverpool
Focus: to provide a clearer understanding of the potential hazards (and associated risks) with RMTs and to develop new methodologies to assess their risk, so that scientific stakeholders are able to accelerate these new medicines into the human population with full confidence.
Acellular (smart material) approaches for therapeutic delivery
Director: Professor Kevin Shakesheff, University of Nottingham
Focus: to develop platform technologies that use materials and drugs to orchestrate cells to regenerate tissue.
Director: Professor Fiona M Watt, King’s College London
Focus: to discover how to harness the immune system for improved outcomes in regenerative medicine.
See also Centre for the Computational and Chemical Biology of the Stem Cell Niche (under the regenerative Medicine capital awards scheme below).
With the successful establishment of this Platform, a £6m investment from the UKRMP in partnership with Arthritis Research UK supports five cutting-edge research consortia at UK research institutions. People suffering from a range of diseases will benefit from these multidisciplinary translational regenerative medicine programmes:
- Professor Charles Archer, Swansea University – Generating durable and resilient repair of cartilage defects using tissue-specific adult stem cells – a systematic, therapeutic approach;
- Professor Pete Coffey, University College London – Scalable production of RPE cells from induced pluripotent stem cell under GMP conditions for cellular replacement therapy of the dry form of Age-related macular degeneration (AMD), a significant cause of blindness in the elderly;
- Professor Andrew McCaskie, University of Cambridge – Stepwise Translational Pathway for Smart Material Cell Therapy (SMART STEP), which will be focused on osteoarthritis;
- Professor Salmeron-Sanchez, University of Glasgow – Synergistic microenvironments for non-union bone defects, with the aim of increasing natural bone regeneration and healing capacity;
- Dr David Hay, The University of Edinburgh – The development of 3-dimensional implantable liver organoids, with the aim of providing alternative renewable solutions to organ transplantation in the treatment of human liver disease.
The first Annual Report for the UKRMP is available through the UKRMP website.
£20M injection of capital funding into regenerative medicine: (2013) to support research addressing the key translational bottlenecks identified through the Strategy for UK Regenerative Medicine. The funding complements recent strategic investments, including the UK Regenerative Medicine Platform, building on existing institutional strengths to provide or enhance national capabilities to meet the challenges identified in the UK strategy. Highlights include a £5m investment (matched by University of Edinburgh) in a new “Centre for the Computational and Chemical Biology of the Stem Cell Niche” and a £2.8M Preclinical in vivo functional imaging platform at the University of Bristol
Human Inducible Pluripotent Stem Cell Initiative “HIPSCi”: Jointly funded by WT and MRC, this is a £13M initiative to create a catalogue of high-quality deeply genotyped and phenotyped induced pluripotent stem cells (iPS cells) from normal and disease carrying individuals. The initiative will provide a knowledge-base to underpin the use of such cells in studying the effects of genes on
health and disease and create a new iPS cell bank.
The first call for disease cohorts was launched this year with 7 cohorts identified in addition to Bardet-Biedl ciliopathy and “Maturity onset diabetes of the young” as pathfinders:
- Usher syndrome (neurones/retina)
- Rare hereditary cerebellar ataxia
- Mitochondrial diseases (neuronal and cardiomyocyte)
- Hypertrophic cardiomyopathy
- Inflammatory bowel disease (macrophage/epithelia)
- Hereditary spastic paraplegia
- Kabuki syndrome (neuronal disease)
BHF Cardiovascular Regenerative Medicine Centres: BHF have committed £7.5M to support three centres of excellence in cardiovascular regenerative medicine aligned with the UKRMP (Oxford Centre for Mending Broken Hearts, Director: Paul Riley; BHF Centre of Regenerative Medicine, Edinburgh, Director: David Newby; BHF Centre of Research Excellence, Imperial College, Director: Michael Schneider).
China UK Stem Cell Partnership Initiative provides matched funding from the MRC and NSFC for significant 3 year research projects, focused on basic and preclinical research of relevance to the longer term development of stem cell based therapies for human disease and disorders and follows from a successful partnership development programme launched in 2012 to build collaborative research links between UK and Chinese labs. It is expected five projects will be confirmed shortly, covering skin/wound healing, remyelination, amyotrophic lateral sclerosis, cardiovascular disease and neurogenesis.
DBT-MRC Joint Centre Partnerships Supported through the UK Newton Fund and the Department of Biotechnology (DBT), Government of India to encourage the strengthening of UK – India research partnerships, grants will be made available to centres or consortia of existing partners in UK and India to further develop collaborative research and support exchange of MRC- and DBT-funded researchers in
both countries. Applications are encouraged across a number of areas, including translational regenerative medicine in neuroscience.
Translational Research Funding
MRC/TSB Biomedical Catalyst: Regenerative Medicine Research Programme: The Biomedical Catalyst has two funding streams relating to stem cell research; The Regenerative Medicine Research Committee provides funding for projects in the early translational space. In the past 5 years the program has awarded >£30m, encompassing projects from providing xeno-free clinical grade hESC lines to four phase 1 trials of autologous stem cell therapies. The Major Awards Committee (with MRC supporting academic led proposals and TSB supporting industry led research) considers later stage translational projects, awarding >£8m in 2013/14. These include the £2.8m Regenvox project to build an artificial human larynx, led by Martin Birchall at UCL.
European Union Activities led from UK
EBiSC: 35M euro Public private partnership in iPSC, funded under the EU Innovative Medicines Initiative (IMI). EBiSC is designed to address the increasing demand by induced Pluripotent Stem Cell (iPSC) researchers for quality-controlled, diseaserelevant research grade iPSC lines, data and cell services. Its goal is to demonstrate an operational banking and distribution service of iPSC lines after 3 years and to establish subsequently for Europe a centralised, not-for-profit bank providing all qualified users with access to scalable, cost-efficient and customised products.
STEMBANCC: Public private partnership in iPSC. Also funded under IMI, STEMBANCC aims to generate and characterise high quality human iPS cell lines to study a range of chronic diseases (e.g. diabetes and dementia) and test for drug efficacy and safety.
The main objectives of StemBANCC are to
- provide access to well characterized, genetically diverse iPSCderived cell types for pharma and academia in Europe, focusing on 500 subjects in 8 disease groups, spanning neurological and psychiatric disorders and diabetes
- provide cell-based assays for predictive toxicology (focus on liver, heart, kidney)
- establish assays for the study of disease biology and compound efficacy & toxicity for drug screening assays
- optimize protocols for the appropriate culture, differentiation, expansion and
maintenance of iPSC-derived cell-types
- foster the implementation of a central test facility for the maintenance of the biobank beyond the funded period.
Changes to Stem Cell Related Policies
No changes in 2013/14.
For further information, see the UK Code of Practice for the Use of Human Stem Cell Lines, which lays out the governance and regulatory structures in the UK for human stem cell research and for the operation of the UK Stem Cell Bank, as well as providing guidance on best practice.
Other relevant developments affecting UK stem cell research.
EU Citizen’s “One of Us” Initiative: In May 2014 Wellcome Trust together with MRC and other UK and international funding organisations and other patient groups joined forces to press the European Commission (EC) to issue a statement dismissing the ‘One of Us’ EU Citizens’ Initiative that called for an end to funding of activities that presuppose the destruction of human embryos, including stem cell research. http://www.wellcome.ac.uk/About-us/Policy/Spotlight-issues/EU-funding-for-stem-cellresearch/index.htm. This was gratefully supported by a joint statement to the EC from many of the members of the ISCF http://www.stem-cell-forum.net/wpcontent/uploads/The-International-Stem-Cell-Funders-Forum-Statement-Europe-2014.pdf. Following representation the EU decided not to change the current legislative approach as it pertained to funding such research http://europa.eu/rapid/press-release_IP-14-608_en.htm.
European Court of Justice Ruling on patenting of hESC lines: This 2011 ruling bans the patenting of inventions involving hESCs and follows a challenge by Greenpeace to a patent filed by German scientist Professor Oliver Brüstle in 1997 to protect a line of cells he developed to treat nervous system defects. The ruling applies only to the patentability of hESC lines and associated processes, and will have no immediate impact on the regulatory framework in the UK. MRC and other UK funders have reaffirmed their commitment to continue to support hESC research.
To note, EU Horizon 2020 funding programme will also retain the same policies relating to support for hESC research as Framework Programme 7 (FP7; no change) – see also EU “One of Us” Initiative above.
National (or Regional) Stem Cell Network or Contact Point
MRC Contact Point
Dr Paul Colville-Nash, Programme Manager for stem cells and developmental biology
Other Major Funders of Stem Cell Research
- Biotechnology and Biological Sciences Research Council (BBSRC)
- Engineering and Physical Sciences Research Council (EPSRC)
- Economic and Social Research Council (ESRC)
- Technology Strategy Board (TSB)
- Wellcome Trust
- Cancer Research UK
- British Heart Foundation
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