Log in
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.
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.
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.
Between 2008 and 2011 researchers at the Department of Veterinary Medicine (DVM) undertook the first randomized double-blinded clinical trial of a cell therapy for spinal cord injury (SCI). The trial involved transplantation of autologous olfactory ensheathing cells into domestic dogs with chronic pelvic limb paraplegia as a consequence of clinical SCI. The results indicated a significant improvement in locomotor function. This study has had a major impact on 1) the public awareness of the use of veterinary disease in biomedical research, 2) public awareness of SCI and approaches to its treatment, and 3) current programmes, both veterinary and human, for SCI treatment.
Research at the UCL Institute of Child Health (ICH) has led to the successful treatment of children with primary immunodeficiency diseases for whom there was little chance of "cure" by the only other possible means: haematopoietic stem cell transplantation (HSCT). Beginning in 2002, we have treated 32 patients with four different primary immunodeficiency disorders. In total we have treated 12 patients with severe combined immunodeficiency (SCID-X1), 13 patients with adenosine deaminase deficient severe combined immunodeficiency (ADA-SCID), 5 patients with chronic granulomatous disease (CGD) and 2 patients with Wiskott-Aldrich syndrome (WAS). Most of the patients have been successfully treated and are at home, off all therapy. We are now starting to develop this technology to treat a wider range of related disorders.
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.
Research by Smales has led to IP that protects novel technologies for mammalian recombinant cell line development. Based upon mass spectrometry and in silico modelling approaches, the technology has permitted the development of highly efficient cell lines for monoclonal antibody production in the commercial environment at Lonza Biologics. This IP has three important benefits to the pharmaceutical and biotechnology industries:
(a) It allows key biopharmaceuticals to be made using substantially less resource and with an overall higher efficiency.
(b) It reduces the time from transfection to production of cell banks.
(c) It accelerates bioreactor evaluation and the ability to predict cell line performance at the bioreactor scale early in cell line construction.
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.
Research from the Department of Materials Science and Engineering led to healthcare impact through treatment of burns patients and those with chronic non-healing wounds using the culture and expansion of the patient's own skin cells. This impact was achieved by establishing a product, MySkin®, as the UK's first and only commercially available complete service for the culture and delivery of patient's skin cells. It is now used in 11 out of the UK's 12 major burns units for patients in danger of death from extensive burns. MySkin® benefits patients, clinicians and nurses and was Biomedical Product of the Year in 2008 (see Sky News video (2008) on www.Ilika.com).
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.