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Clinicians and scientists at UCL have been central to the design and management of single centre and multi-centre lymphoma trials within the UK and internationally. The trials have enabled a balanced approach to the non-Hodgkin lymphomas (NHL), supporting more conservative strategies in certain well-defined situations but also providing evidence for the value of very intensive therapy in appropriate patients. These trials have contributed to patient survival, quality of life and appropriate resource utilisation.
Sustained research by the University of Oxford's Mahidol Oxford Tropical Medicine Research Unit in Thailand (MORU) has been the driving force behind the current World Health Organization recommendations for the management of acute and chronic infection in patients with melioidosis. This research has motivated improvements in treatments and provided new strategies to identify at-risk populations, enabling clinicians to make early diagnoses. Melioidosis is a major cause of severe illness in parts of Southeast Asia and there are increasing numbers of cases in India, China, and Brazil.
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.
COPD affects up to 3.5 million people in the UK and costs the NHS £700m pa. Over the last 15 years, research by Professor Calverley and colleagues at the University of Liverpool (UoL) has impacted significantly on the care of COPD patients. Specifically, this group showed that routine testing of COPD patients for the presence of bronchodilator reversibility was unreliable and did not predict clinical outcomes. This changed international guideline recommendations in 2007 and the Quality Outcomes Framework payments to GPs in 2009. They showed that oral corticosteroids accelerated recovery from exacerbations and that anti-inflammatory drugs, whether inhaled corticosteroids or PDEIV inhibitors, reduced exacerbations by 25% with a subsequent fall in the number and length of hospitalisations. This led to changed NICE guidance for corticosteroids in 2010 and drug registration with EMA and FDA for the PDEIV inhibitor treatment in 2011. Treatment in UK and Western Europe has changed as a result of this research.
The University of Oxford's International Subarachnoid Aneurysm Trial (ISAT) changed clinical practice worldwide by showing that endovascular coiling is a more effective and safer treatment than neurosurgery following subarachnoid haemorrhage, with fewer complications and improved quality of life. Subarachnoid haemorrhages account for 1 in 14 strokes and are caused by bleeding in and around the brain; approximately 85% occur when cerebral aneurysms rupture. ISAT was the first trial to compare neurosurgery, or neuroradiological endovascular coiling in patients with ruptured cerebral aneurysms causing acute subarachnoid haemorrhage.
Research by the University of Southampton has helped transform the understanding and treatment of chronic lymphocytic leukaemia (CLL), the most common leukaemia, affecting around 2,400 patients each year in the UK and 17,000 in the USA. Southampton's widely cited studies revealing the existence of two subsets of CLL have been crucial in giving clinicians and patients in the UK and overseas a much clearer indication of the likely disease course. The predictive information is now included in all clinical trials and in international guidelines, delivering greatly improved care. The research has also inspired the development of a new drug given "breakthrough" status by the Food and Drug Administration in the United States.
Researchers at the University of Bristol have developed tests to track low-level leukaemia — `minimal residual disease' (MRD) — in children with acute lymphoblastic leukaemia (ALL) down to levels thousands of times lower than detectable by light microscopy. These tests have become the gold standard for monitoring of leukaemic response in clinical trials. MRD testing has been shown in 2013 to allow safe de-intensification of treatment for one-fifth of patients treated nationally, with substantial savings in toxicity and treatment-related expense. The same techniques have also improved worldwide understanding of how disease clearance is related to success after haemopoietic stem cell transplantation.
Research conducted at UCL/UCLH over the last 20 years has enabled the identification of adults with acute leukaemia who are most likely to benefit from the use of stem cell transplantation, i.e. those with acute leukaemia in first remission. The treatment is highly intensive, potentially toxic and expensive high-dose chemotherapy followed by haemopoietic stem cell transplantation, and is inappropriate for some patients. The work has made a major contribution to the development of guidelines worldwide for the treatment of this disease. Improved patient selection for transplantation results in improved survival, less toxicity with improved overall quality of life, and a more appropriate use of NHS resources.
Alemtuzumab, a humanised therapeutic antibody, is a major addition to the repertoire of immunosuppressive agents used for organ and stem cell transplants. Administered as an induction agent in a short course of treatment, alemtuzumab reduces the incidence of graft rejection without preventing recovery of the patient's ability to fight infection. Alemtuzumab also decreases graft versus host disease, a vital factor in the treatment of aplastic anaemia and acute leukaemias. Furthermore, its important role in minimising immunosuppressive therapy helps prevent treatment-associated problems for the patient. Currently used off-licence for transplants, alemtuzumab improves patient survival and healthcare.
Professor Platt and colleagues at the University of Oxford have developed the drug miglustat, the first oral therapy for rare lysosomal storage diseases. These are primarily neurodegenerative diseases that affect 1 in 5,000 live births, always leading to premature death. In 2009, miglustat became the first treatment to be licensed for treating neurological manifestations in Niemann-Pick disease type C (NPC). It is now prescribed for the majority of NPC patients worldwide, and has led to significant improvements in both life expectancy and quality of life. Miglustat was approved for type 1 Gaucher disease in 2002 and, since 2008, has proved an effective treatment for patients previously stabilised with enzyme replacement therapy; miglustat has the additional benefit of improving bone disease. Sales of miglustat since 2008 have generated CHF 315 million in revenues for Actelion, the company sublicensed to sell the drug.