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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.
We were the first to show that human stem cells could be used to create functional organ replacements in patients. These transplants, first performed to save the life of an adult in 2008, and then repeated to save a child in 2010, have changed the way the world views stem cell therapies. We have opened the door to a future where conventional transplantation, with all its technical, toxicity and ethical problems, can be replaced and increased in range by a family of customised organ replacements, populated by cells derived from autologous stem cells. This has altered worldview, changed clinical practice and had key influences on UK policy.
Mouse disease models provide an invaluable tool to the medical sciences, underpinning the understanding of disease mechanisms and the development of therapeutic interventions. A new cultivation protocol for deriving mouse embryonic stem (ES) cells was developed by Dr Nichols between 2006 and 2009. This has facilitated the production of ES cells from disease model mice that can be manipulated in vitro and used to establish modified transgenic mice with the required genetic profile, in a single generation. This method reduces the number of mice needed, as well as associated costs and staff time, by 90%. Dr Nichols has trained industry delegates from international transgenics companies and transgenic facility managers in the new technology. As a consequence, a minimum of 26820 fewer mice have been used in experiments, and a minimum of £536k have been saved since 2009.
In 2008, Professors Martin Birchall and Anthony Hollander (University of Bristol) and a team of scientists and surgeons led from Bristol successfully created and then transplanted the first tissue-engineered trachea (windpipe), using the seriously ill patient's own stem cells. The bioengineered trachea immediately provided the patient with a normally functioning airway, thereby avoiding higher risk surgery or life-long immunosuppression. This sequence of events, which raised public interest and understanding around the world as a result of huge media coverage, acted as proof of concept for this kind of medical intervention. A new clinical technology with far-reaching implications for patients had passed a major test. This development demonstrated the potential of stem cell biology and regenerative medicine to eradicate disease as well as treat symptoms and has already led to the implantation of bioengineered tracheas in at least 14 other patients.
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.
Professor Andrew Webster's sociological research on developments in biomedical science has been impactful in shaping regulatory practice and influencing policy in relation to biobanking, stem cell research and regenerative medicine. In particular, his research has been used to: change donation procedures to the UK Biobank; influence regulatory decisions made by the UK Stem Cell Bank Steering Committee (UKSCBSC); contribute to regulatory practices associated with clinical trial design and adoption, and inform the UK government's investment strategy in regenerative medicine.
Stem cells play an important role in drug discovery and development of therapeutic interventions. Differentiation (and maintenance) of stem cells into specialised cells is achieved by controlled application of specific, expensive growth factors.
Dr Hyvönen has developed an efficient method for producing highly purified, bioactive human growth factors from E.coli, reducing costs by up to 10-FOLD. tHE TECHNOLOGY HAS BEEN LICENSED TO A major international manufacturer of growth factors (PeproTech Inc.), and to a UK-based specialist stem cell company (CellGS Ltd), enabling them to implement new products and business strategies. Through a departmental facility, material is also being sold to external companies and Cambridge Stem Cell Consortium members. In addition, Dr Hyvönen has made his expertise available to biotech companies through consultancy.
Fundamental and applied research at RVC has led to introduction of stem cell therapy supporting equine tendon regeneration, advancing equine clinical practice internationally. A resultant spin-out company has delivered revenue-generating veterinary clinical services internationally and is now developing new human treatments. The therapy offers improved health and welfare, particularly in racing, as treated horses are less likely to re-injure in comparison with those managed conventionally, and consequently less likely to be culled due to premature termination of their competitive careers. The acceptance by the Medicines and Healthcare products Regulatory Agency that the equine treatment data provide validation for a phase II human clinical trial without further preclinical studies represents a rare and significant outcome for veterinary research.
Impact: Economic. The EaStCHEM spin-out company Deliverics has commercialised biodegradable transfection reagents for both the "research tool" and the "RNAi therapeutics" markets (globally valued at £400M and £4 billion respectively). Beneficiaries are the pharmaceutical and biotechnology sectors, and clinicians. The turnover since 2010/11 is £330k and the company currently has five employees.
Significance: Deliveric's agents out-perfom existing materials in term of efficacy and reduced levels of toxicity. They are not hampered by the immunogenicity, manufacturing issues, and carcinogenicity previously seen for viral vectors used as delivery agents. This presents a wide ranging ability to deliver nucleic acids into cells and tissues for biological applications.
Research; date; attribution: EaStCHEM research (2008) led by Bradley reported a family of non-viral DNA delivery agents that offered a highly-efficient and non-toxic method of delivering siRNA/DNA into mammalian cells and tissues. Development and patenting of this technology led to the spin-out of Deliverics Ltd. in 2010.
Reach: International customer base (20 research groups and 10 companies) including specially appointed distributors in Spain (Albyn Medical), South Korea (CoreSciences), and US (Galen).
Novel biological scaffolds that regenerate with the patient's own cells have been researched, and patented and since 2008 developed, taken through successful clinical trials and commercialised. Economic impact within the REF period has been delivered through the University of Leeds spinout company Tissue Regenix plc, which has received £32M private investment, employs 35 people and is AIM listed, with a capital value of £70M. Health impact has been delivered through licensing and development by NHS Blood & Transplant Tissue Services. The biological scaffolds have demonstrated five years' successful clinical use in heart valve replacement and three years' clinical use as a commercial vascular patch.