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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

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

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

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

New businesses, commercial investment and adoption of new technology result from antigen-specific peptide immunotherapy development

Summary of the impact

By identifying a novel approach to treat allergy and autoimmune disease the University of Bristol has created a new field of research into antigen-specific peptide immunotherapy. Initial work carried out by Professor David Wraith at the University has since 2008 led to the creation of new businesses, (including the spinout company Apitope), generated 100s of millions of pounds of investment and underpinned both the adoption of new technology and the development of new products by the pharmaceutical industry. The commercial impact of this research into antigen specific immunotherapy is on-going and expanding.

Submitting Institution

University of Bristol

Unit of Assessment

Clinical Medicine

Summary Impact Type

Technological

Research Subject Area(s)

Medical and Health Sciences: Immunology, Neurosciences

Transforming Care for Women with Pelvic Organ Prolapse

Summary of the impact

A research programme of multi-centre clinical trials led by Professor Suzanne Hagen has established Pelvic Floor Muscle Training (PFMT) as an effective treatment for women with prolapse. Hagen's team has also successfully developed a Prolapse Symptom Scale and further tested a Prolapse Staging System to improve outcome measurement for women's health physiotherapists in the UK (20% and 15% clinical uptake respectively). The research has informed local, national and international guidelines and changed practice in 48% of UK physiotherapists. The research has also raised awareness of PFMT treatment for prolapse, with 70% of UK physiotherapists reporting an increase in prolapse referrals.

Submitting Institution

Glasgow Caledonian University

Unit of Assessment

Allied Health Professions, Dentistry, Nursing and Pharmacy

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Public Health and Health Services

Improving the Methodology, Ethics, and Implementation of Evidence-Based Medicine

Summary of the impact

Dr Jeremy Howick's research into philosophical issues concerning the nature of the evidence invoked in evidence-based medicine has led both to a revision of the standards for reporting trials, and to a redesign of the Oxford Centre for Evidence-Based Medicine `Levels of Evidence': one of the most widely used systems for ranking medical evidence, and thereby for deciding whether treatments are effective, in the world. His research into philosophical issues concerning the ethics of using placebos in clinical trials and in clinical practice has influenced practitioners as well as patients by helping to determine how treatments are developed and applied. Through his research in both of these areas he has enhanced public understanding of the use of placebos.

Submitting Institution

University of Oxford

Unit of Assessment

Philosophy

Summary Impact Type

Societal

Research Subject Area(s)

Medical and Health Sciences: Clinical Sciences, Pharmacology and Pharmaceutical Sciences, Public Health and Health Services

SIFT-MS: instruments for breath analysis and trace gas analysis in medicine and industry

Summary of the impact

A novel technique named Selected Ion Flow Tube-Mass Spectrometry, SIFT-MS, has been researched and developed by Professor David Smith and Professor Patrik Spanel at the Institute for Science and Technology in Medicine (ISTM), Keele University. This technique allows real time, on-line analysis of trace gases in air and exhaled breath. This development stems from basic research into the reactivity of ions in the gas phase, in which Smith & Spanel are recognised international experts, combined with a complete understanding of the engineering and technical requirements of successful analytical instrumentation. SIFT-MS instruments have been developed initially from large laboratory devices to the current, transportable commercial instruments that are produced by two independent manufacturers and sold worldwide. They are used in various fields of research, including immediate analysis of exhaled breath and urine headspace for clinical diagnosis and therapeutic monitoring, cell biology, and in environmental and food sciences. They have important practical use in the safety of customs workers in container ports and in prospecting for oil and gas. SIFT-MS instruments manufactured in the UK have been exported to Austria, Czech Republic, China and the USA.

Submitting Institution

Keele University

Unit of Assessment

General Engineering

Summary Impact Type

Technological

Research Subject Area(s)

Physical Sciences: Other Physical Sciences
Chemical Sciences: Analytical Chemistry, Physical Chemistry (incl. Structural)

Development of novel adaptive designs to improve efficiency in clinical trials

Summary of the impact

Clinical trials are costly to the pharmaceutical industry and public funding bodies, require major commitment from volunteer patients and take significant time to lead to patient benefit. Adaptive designs are one approach which seeks to improve the efficiency of such studies. Statistical research at Reading has led to novel methodology for the design and analysis of clinical drug trials within the framework of adaptive designs which has the potential to reduce the time taken for effective drugs to reach the market and thus benefit specific patient groups. To date the research has had impact in three major ways: i) it has been adopted by pharmaceutical companies as a means of improving the efficiency of their clinical trials, ii) the research has been cited in the regulatory guidance on adaptive clinical trial design, and iii) it has increased awareness by clinicians and other medical professionals of the potential benefit of the adaptive design methodology to their patient groups. Hence, the research has influenced industry, regulatory and health professionals with potential significant economic benefit and improved outcome for patients.

Submitting Institution

University of Reading

Unit of Assessment

Mathematical Sciences

Summary Impact Type

Political

Research Subject Area(s)

Medical and Health Sciences: Clinical Sciences, Oncology and Carcinogenesis, Pharmacology and Pharmaceutical Sciences

Gene therapy for immunodeficiency diseases

Summary of the impact

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.

Submitting Institution

University College London

Unit of Assessment

Clinical Medicine

Summary Impact Type

Health

Research Subject Area(s)

Technology: Medical Biotechnology
Medical and Health Sciences: Immunology

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