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UOA05-07: A paradigm change in the treatment of multiple sclerosis

Summary of the impact

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.

Submitting Institution

University of Oxford

Unit of Assessment

Biological Sciences

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Clinical Sciences, Immunology, Neurosciences

Public Understanding of Multiple Sclerosis Research

Summary of the impact

Research undertaken by Professor Woodroofe has impacted on a range of beneficiaries: people with multiple sclerosis (MS), their families and carers, and health professionals, including nurses, physiotherapists and doctors. This has been achieved through an array of innovative dissemination activities involving shared learning among researchers and beneficiaries. Through these activities beneficiaries gained a greater knowledge and deeper understanding of the disease process in MS, which they subsequently shared within their own networks. Woodroofe's research on MS has been published in leading international journals making an important contribution to the field and underpinning the impact achieved.

Submitting Institution

Sheffield Hallam University

Unit of Assessment

Biological Sciences

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Immunology, Neurosciences

Translating research into novel immunotherapies delivers scientific and economic gains for the pharmaceutical/biotechnology sector in drug discovery

Summary of the impact

Research into novel immunotherapies has given rise to a novel drug (EtxB), which is now in Phase II clinical trials, and to a profitable contract research company partnering with the pharmaceutical industry to develop their compounds. Trident Pharmaceuticals was formed around patents filed by the University of Bristol, has received investment of [text removed for publication], successfully completed Phase I trials (2011) and is in the midst of Phase IIa trials in humans with inflammatory disease (2013). KWS BioTest arose as a result of the underpinning research and experience gained from developing EtxB, and is now a leading contract research organisation working with pharmaceutical and biotechnology companies developing novel treatments for human disease. KWS has directly contributed to the development of therapies at more than 75 different companies, employs 28 people, has exported [text removed for publication] and was 2012 winner of a Biomedical iNet Award for outstanding business achievement.

Submitting Institution

University of Bristol

Unit of Assessment

Clinical Medicine

Summary Impact Type

Technological

Research Subject Area(s)

Medical and Health Sciences: Immunology

Multiple Sclerosis: developing treatment and improving outcomes

Summary of the impact

This case study summarises a body of research on Multiple Sclerosis (MS) developed at Plymouth University under the leadership of Professor Zajicek and Professor Hobart. Hobart's work on linical outcome measurements has directly influenced clinical research, trials and drug licensing, especially in MS and Alzheimer's disease. The MS scales developed by Hobart have been endorsed by the United States FDA and are in demand by commercial organisations in the development and trialling of treatments for MS and have led to the licensing of new drugs. Zajicek has led the topical field in evaluating the potential benefits and risks of cannabis for treating MS, contributing to the evidence base behind the medical use of cannabinoids in general, and pioneering its global potential use to slow neurodegeneration.

Submitting Institution

Plymouth University

Unit of Assessment

Clinical Medicine

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Clinical Sciences, Neurosciences, Public Health and Health Services

Minimal residual disease assessment in acute lymphoblastic leukaemia allows safe individualisation of chemotherapy and reduction of treatment toxicity

Summary of the impact

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.

Submitting Institution

University of Bristol

Unit of Assessment

Clinical Medicine

Summary Impact Type

Technological

Research Subject Area(s)

Medical and Health Sciences: Cardiorespiratory Medicine and Haematology, Oncology and Carcinogenesis

Cannabis as a source of medicines

Summary of the impact

Research at the University of Aberdeen has directly contributed to the development of the cannabis-based medicine, Sativex®, which was licensed in the UK in 2010 for relieving neuropathic pain and spasticity of multiple sclerosis (MS), removing the need for patients to self-medicate with illegal, "unregulated" cannabis. The research has both enhanced patient welfare and promoted collaboration with industry. Several other countries have also approved Sativex®. Apart from such direct benefits, the research has also increased understanding of the benefits of cannabis-based medicines among the general public, and the main researcher has advised the Home Office on pertinent legislation. Therefore the claimed impact here includes benefits to health and welfare guidelines and on the public understanding of science. In addition industry has invested in research and development and a new product has been commercialised.

Submitting Institution

University of Aberdeen

Unit of Assessment

Biological Sciences

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Neurosciences, Pharmacology and Pharmaceutical Sciences

Natalizumab: a potent treatment for highly active relapsing-remitting multiple sclerosis

Summary of the impact

Multiple Sclerosis (MS) is the most common disabling neurological disease of young adults in the UK, affecting 1 in 800 of the population. In most patients the early years are characterised by relapse and remissions; relapses are often disabling and permanent disability occurs when remissions fail to recover fully. Research at the UCL Institute of Neurology — from early MRI studies through phase 1-3 clinical trials — has resulted in the licensing of natalizumab for highly active relapsing remitting MS. Natalizumab is now widely used to treat such patients with very good efficacy and close monitoring. Natalizumab is a potent treatment that has reduced relapse rate by two-thirds and relapse-related disability by 50%. By July 2013, over 115,000 patients around the world had received this treatment.

Submitting Institution

University College London

Unit of Assessment

Psychology, Psychiatry and Neuroscience

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Clinical Sciences, Neurosciences

Therapeutic hypothermia saves thousands of babies each year across the developed world from severe disability or death

Summary of the impact

Since 2010, infants around the world have been saved from death or severe disability as a result of research conducted by Professor Marianne Thoresen and her team at the University of Bristol. Translational research conducted between 1998 and 2010 by the Thoresen group showed that mild cooling of newborn children who had suffered a lack of oxygen during labour and delivery reduced death and disability by over 50%. Extensive publication on this treatment and practical training for neonatal staff, led by the Bristol team since 2008, has transformed the management of neonatal brain injury. By 2010/11, therapeutic cooling had been adopted as standard treatment throughout the developed world, saving thousands of children from death or severe disability, including cerebral palsy and epilepsy. Therapeutic hypothermia also saves the NHS and UK families about £200 million/year in care and compensation costs.

Submitting Institution

University of Bristol

Unit of Assessment

Psychology, Psychiatry and Neuroscience

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Clinical Sciences, Neurosciences, Paediatrics and Reproductive Medicine

A clinical science programme delivering the most effective therapy for multiple sclerosis - Coles and Compston

Summary of the impact

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.

Submitting Institution

University of Cambridge

Unit of Assessment

Psychology, Psychiatry and Neuroscience

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Clinical Sciences, Neurosciences

UOA05-08: The use of alemtuzumab in stem cell and organ transplantation

Summary of the impact

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.

Submitting Institution

University of Oxford

Unit of Assessment

Biological Sciences

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Clinical Sciences, Immunology

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