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Research by Professor Parmjit Jat (first at the Ludwig Institute for Cancer Research, then part of UCL; later at the UCL Institute of Neurology) established and applied the critically important scientific concept of conditional immortalisation to a wide variety of cell lines, enabling cells to be grown indefinitely in vitro but differentiate upon altering the growth conditions. Two companies were established in partnership with Jat to exploit this research, ReNeuron (now worth £63.5m and publicly traded on the London AIM market) and XCellSyz (now part of Lonza AG). More than 20 patents based on Professor Jat's work have been issued. Reagents based on his research have been evaluated, licensed and used by 17 companies worldwide: Amgen, Amylin, Boehringer Mannheim, Cell Genesys, Chiron, Eli Lilly, Genentech Inc., Genetics Institute, Immunex, Johnson & Johnson, Medarex, Novartis, Ortho Pharm., Pfizer Inc., Regeneron, ReNeuron, Takeda, EMD Serono, and XCellSyZ/Cambrex Bioscience/Lonza.
Impact on commerce: Five stem cell culture products derived from UoE research have been brought to a global market since 2009 through the US based company StemCells Inc. StemCells Inc strategically acquired Stem Cell Sciences plc (SCS), with its licensed portfolio of UoE patents, to position themselves as a world leader in cell-based medicine. This enabled them to develop media and reagent tools in order to pursue nearer-term commercial opportunities. These products include the gold standard media for embryonic stem cell culture, iSTEM.
Beneficiaries: Commercial companies and users of the stem cell culture products.
Significance and Reach: iSTEM is the gold standard media used worldwide by researchers for maintaining mouse ES cells in their basal, non-differentiated state. Products are sold worldwide through global life sciences companies.
Attribution: All research was carried out at UoE between 1994 and 2006 (published up to 2008), led by Prof Austin Smith. Collaboration with Prof Philip Cohen, University of Dundee, on one paper (2008).
Neural stem cells offer enormous therapeutic potential for stroke but they require regulatory approval. Researchers at King's College London (KCL) devised a technology to immortalise stem cells, generated clinical-grade neural stem cell lines and demonstrated efficacy in an animal model of stroke. KCL research underpins the first approvals in the UK for a therapeutic stem cell product. This led to an industry-sponsored clinical trial of a stem cell therapeutic that has demonstrated vital improvement in all the first five stroke patients treated. KCL research has made a significant impact by considerably reducing the timetable for delivering potential therapies which will affect the life sciences industry and the process now in place acts as a model for other technology developments in this area.
Research on stem cells has led to an explosion of interest in the field of regenerative medicine, with the potential for new clinical interventions and treatments. Pioneering research in Sheffield led to the founding of a spin-out company, Axordia, in 2001, focussed on the applications of human embryonic stem cells (hESC) in medicine. Several hESC lines (including SHEF-1) were generated in Sheffield by Axordia, which was sold to Intercytex in 2008 for £1.68M. These Sheffield-derived hESC lines were then sold on to a major pharmaceutical company, Pfizer, for £0.75M in 2009. As a result, a clinical grade derivative of SHEF-1 has been developed and approved for clinical trials for treating age-related macular degeneration (AMD). In addition, Sheffield research has led to the licensing and sales of key hESC marker antibodies for stem-cell quality control. Finally, Sheffield researchers have informed emerging regulatory guidelines about the safety of hESC regenerative medicine applications by authoring reports and providing evidence to a Parliamentary committee. The case study has significant impact on commerce, health and welfare and public policy.
Clinical research from UCL established `salvage therapy' and autologous transplantation protocols for use in relapsed and resistant Hodgkin lymphoma and demonstrated the efficacy of such approaches. These treatments are now widely used standards of care. A reduced intensity transplant (RIT) regimen, incorporating alemtuzumab to reduce graft-versus-host disease, was also developed and a potent graft-versus-tumour effect was demonstrated. RIT treatments are now increasingly used in patients failing an autologous transplant and in those patients deemed to have a high risk of autograft failure, as determined by pre-transplant CT/PET scanning. We estimate that 5,000 patients have been cured in the REF period as a result of our research.
Research on stem cells has led to an explosion of interest in the field of regenerative medicine, with the potential for new clinical interventions and treatments. Pioneering research in Sheffield led to the founding of a spin-out company, Axordia, in 2001, focussed on the applications of human embryonic stem cells (hESC) in medicine. Several hESC lines (including SHEF-1) were developed by Axordia, which was sold to Intercytex in 2008 for £1.68M. These Sheffield-derived hESC lines were then sold on to a major pharmaceutical company, Pfizer, for $0.75M in 2009. As a result, a clinical grade derivative of SHEF1 has been developed and approved for clinical trials for treating Age Related Macular Degeneration (AMD). Finally, Sheffield researchers have informed emerging regulatory guidelines about the safety of hESC regenerative medicine applications by authoring reports for government and research councils.
Seven patients with avascular necrosis of the femoral head and bone cysts have been treated successfully with skeletal stem cell therapy, developed by Southampton researchers, resulting in an improved quality of life. This unique multi-disciplinary approach linking nano-bioengineering and stem cell research could revolutionise treatment for the 4,000 patients requiring surgery each year in the UK and reduce a huge financial burden on the NHS. The work has been granted three patents and the team are in discussions on development of the next generation of orthopaedic implants with industry.
Fundamental to effective treatment of diabetes is the understanding of complex mechanisms regulating the function and demise of insulin-secreting pancreatic beta-cells. Inherent limitations relating to pancreatic beta-cell supply coupled with short functional life in culture prompted the challenge to establish model clonal human beta-cells. Ulster exploited an innovative approach to first establish clonal rodent beta-cells. Further development of our novel technology resulted in the generation, patent protection, and commercialisation of world-first electrofusion-derived functional human beta-cells. Our unique and valuable beta-cell lines have been licensed to multi-national pharmaceutical companies for diabetes drug development and further commercialised by sales through ECACC (now Public Health England) to directly impact on both bio-industry and the international research community by providing a limitless supply of high quality model beta-cells for translational research and diabetes drug development.
Impact: Public engagement and education, influence on public ethical and scientific policy.
Significance: The first demonstration of cloning from an adult mammalian somatic cell has stimulated rolling religious, ethical, cultural, political and scientific debates. Dolly has become a scientific icon entering the public and educational lexicons in addition to scientific ones.
Beneficiaries: Human society, culture, education.
Attribution: Wilmut and colleagues (Roslin Institute, UoE), undertook somatic cell nuclear transfer and used it to perform the first successful cloning of an adult mammal.
Reach: Worldwide: Dolly became a scientific icon that is recognisable all around the world, representing a major public engagement with bioscience. For example; cloning principles are part of high school education including the International Baccalaureate (implemented in >3600 schools on five continents).
Basic and translational research undertaken since 1993 by UCL Virology has defined the natural history and pathogenesis of cytomegalovirus (CMV) infection and disease. As a consequence of our work, rapid diagnosis and pre-emptive therapy are now available worldwide for this important infection. We have provided a national reference service for strains of CMV resistant to current antiviral drugs and for diagnosis of congenital CMV infection.