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Research conducted by Professor TM Cox has led to several advances in the management of lysosomal storage disorders; i) development of miglustat (Zavesca®); now available throughout the world (EMA and FDA approved) for adult patients with Gaucher's disease and throughout the European Union and five other countries worldwide for adult and pediatric patients with Niemann- Pick type C disease, ii) development of the potential successor eliglustat; now in Phase 3 clinical trials, iii) identification of a biomarker for Gaucher's: CCL18/PARC, now incorporated into NHS standard operating procedures for monitoring therapeutic intervention. His pre-clinical research into gene therapy for Tay-Sachs disease also helped establish the NIH-funded Gene Therapy Consortium and gain the FDA's pre-IND approval for clinical trials in 2013, which together have raised public awareness of this disease.
Jayne's team have co-ordinated a sequence of randomised clinical trials, that have defined the standard of care for ANCA vasculitis treatment and shaped national and international guideline statements, NHS national commissioning guidance and an on-going NICE assessment. Together with Ken Smith his group have pioneered the use of the B cell-depleting agent rituximab, in vasculitis, contributing key evidence that led to its licence approval (USA and EU) for this indication. Ken Smith's group supported by Jayne's clinical team have discovered novel therapeutic biomarkers, patented and being assessed in Phase II clinical studies, that promise to deliver "personalised medicine" in this and related conditions. These activities have harmonised the management of vasculitis, are improving patient outcomes, and have provided a resource for on-going scientific and clinical studies.
King's College London (KCL) research has made a major contribution to improving the quality of life for patients who have anaemia linked with chronic kidney disease. Studies undertaken by KCL researchers established that intravenous iron supplementation was required in anaemic patients with advanced kidney disease, in whom oral iron therapy was ineffective, and defined the best regimes for administration of intravenous iron. Subsequent KCL work on drugs that stimulate production of red blood cells (erythropoiesis) defined the target levels of haemoglobin to aim for in chronic kidney disease patients. Most recently, KCL researchers made the key discovery that the novel drug peginesatide for the first time enables the rescue of patients who develop a rare and potentially fatal reaction against erythropoietin (which is the commonest treatment for anaemia in chronic kidney disease). These KCL research studies have had a significant impact by making a major contribution to national and international clinical guidelines, including UK NICE guidelines and the 2012 National Kidney Foundation KDIGO Clinical Practice Guideline for Anemia in Chronic Kidney Disease.
Research led by University of Oxford scientists has resulted in widespread use of the humanised therapeutic antibody, Campath (alemtuzumab), in patients with chronic lymphocytic leukaemia (CLL). Licensed by both the European and American regulatory authorities in 2004 for the treatment of CLL, Campath is used as first-line treatment for patients with aggressive forms of the disease and following relapse. It can induce long-term clinical remission even in cases resistant to other drugs. Campath has now been used in approximately 15,000 patients, and has generated revenues of approximately £750 million from the licensed treatment of CLL.
Starting from a mechanism-based hypothesis, Alastair Compston and colleagues in Cambridge have led the academic development of Alemtuzumab as a highly effective therapy for relapsing-remitting multiple sclerosis through Phase 1, 2 and two Phase 3 trials (1991-2012). The impacts to date are demonstration of the importance of the therapeutic `window of opportunity' in treating multiple sclerosis; a product licence in the European Union (September 2013) for the commonest potentially disabling neurological disease of young adults; expansion of the work-force in industry to develop and market this initiative; and an estimated several-fold increase in revenue to the University of Cambridge (and other beneficiaries) from total royalties of £18.6M from 1997 to date.
Researchers at the Dunn School of Pathology at the University of Oxford have played a major role in the development of an effective and innovative treatment for the chronic debilitating disease multiple sclerosis (MS). Research arising from the work of immunologists in Oxford, and partner neuroscientists in Cambridge University, has shown that low dose treatment with the lymphocyte depleting antibody alemtuzumab can break the cycle of disease in MS. Alemtuzumab acts by re-setting the immune system, leading to long-term arrest or remission, without increasing the risk of infection or malignancy. Large-scale studies since 2008 have shown that treatment is more effective and better tolerated than conventional forms of therapy. In June 2013, the European Medicines Agency's Committee for Medicinal Products for Human Use recommended that the drug be licensed for people with active relapsing-remitting MS. The research by Oxford University and its collaborators into the use of alemtuzumab in MS has been shown to benefit patients; it offers hope to millions of sufferers worldwide; and has had a major impact on the pharmaceutical industry.
Psynova Neurotech is a prize-winning spin-out company founded by Professors Sabine Bahn and Chris Lowe from the University of Cambridge. It focuses on the commercialization of novel blood-based biomarker tests for conditions like schizophrenia, depression and bipolar disorder. Psynova and its partner company Rules Based Medicine (now Myriad RBM Inc.) launched the first commercially available Aid for the Diagnosis of Schizophrenia (VeriPsychTM) in 2010. In June 2011, Psynova and Rules Based Medicine were acquired by Myriad Genetics Inc. for £50 million. In February 2011 Psynova Neurotech and Professor Bahn were announced winner of the ACES best European Life Science spin-out award.
Psoriatic arthritis (PsA) is a chronic inflammatory disease of joints, skin and tendons that affects 0.5-0.8% of the population worldwide. PsA can cause substantial psychological and social problems and also causes increased risk of death from cardiovascular disease. Research conducted by Prof Iain McInnes at the University of Glasgow in partnership with leading pharmaceutical company, Janssen, has provided robust evidence of the clinical benefits and safety of the cytokine blocker ustekinumab, leading to its approval for use for PsA by the European Medicines Agency in July 2013. This was the first approval of a PsA drug with a new mode of action in a decade, providing a novel treatment for approximately 1.25 million PsA patients across Europe.
Researchers from St Georges have evaluated and optimised anti-fungal therapy for cryptococcal meningitis, the commonest cause of adult meningitis in sub-Saharan Africa. They have developed a "screen-and-treat" strategy to prevent the development of clinical disease in HIV-positive patients, and with collaborators developed and tested a novel point-of-care diagnostic test. These advances have led to changes in and development of a series of international guidelines and application of these new strategies in parts of Africa. A case for reduced costs of amphotericin was advanced by the group who were instrumental in reducing these costs in South Africa, allowing wider drug provision.
This case study outlines the impact of novel omega-3 fatty acid therapy for sickle cell disease on health and policy. 128 patients on the treatment since 2010, and another 300 who started to receive it in June 2012 have seen remarkable improvements in health and quality of life as assessed by reductions in hospital admission and absence from work/school due to the disease. A panel of experts set up by the Ministry of Health of Sudan to evaluate the evidence recommended the integration of the therapy in the management of the disease in a policy report dated December 20, 2012. The Ministry has accepted the recommendation.