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Parkinson's Disease is a disabling disorder affecting 1 in 500 people in the UK. King's College London (KCL) researchers discovered that non-motor symptoms are a key determinant of quality of life for patients with Parkinson's Disease but found these were rarely assessed or treated. KCL researchers developed the first patient-reported questionnaires allowing clear documentation of different non-motor symptoms. These KCL-designed scales empowered patients to report these symptoms and receive appropriate treatments and led to the implementation of patient outcome-based policy-making in England and internationally. They also placed important symptoms for a patient's quality of life as a key outcome in large-scale clinical trials for Parkinson's Disease.
Research into the genetic causes of Parkinson's disease by Professor Nick Wood's group at the UCL Department of Molecular Neuroscience, describing the mutations in the gene LRRK2, have led to the development of a new genetic test which is now available to patients and their families. This benefits them by providing a precise diagnosis, and an understanding of the risk of disease to relatives. The research has provided new insight into patterns of Parkinson's disease in particular ethnic groups, and given rise to improved public understanding and high profile philanthropy. This discovery has also opened up a new area of research into disease-modifying treatments in Parkinson's disease within the pharmaceutical industry, leading to new drug candidates.
Bio-inspired computer algorithms, developed by Dr Stephen Smith at the University of York, have been integrated with commercially available hardware that analyse patients' movements to diagnose and monitor a range of neurodegenerative conditions including Parkinson's disease and Alzheimer's disease. Clinical studies undertaken in the UK and USA report a diagnostic accuracy exceeding 90% - a 15% improvement on current clinical practice. A new spinout company, ClearSky Medical Diagnostics Ltd, has licenced four products exploiting this technology to nine health centres in the UK, USA, Singapore, Australia and UAE, transforming clinical testing with improved diagnosis and monitoring of patients in hospitals and their own homes.
Systemic lupus erythematosus (SLE) is a multi-system autoimmune disease that is subject to relapses (flares) and remissions. Measuring disease activity in multiple systems, some of which may be worsening while others are improving, is a challenge in the management of patients with SLE and also in the conduct of clinical trials of new drugs for the treatment of SLE. The British Isles Lupus Assessment Group (BILAG) disease activity index for measuring lupus was developed by Professors Paul Bacon and Caroline Gordon at the University of Birmingham and has been validated and implemented for clinical trials and routine clinical practice. The instrument is able to capture significant improvement or worsening in lupus disease activity on a system based approach, leading to improved management and treatment of patients. It is the preferred disease activity instrument for international SLE trials recommended by the US Food and Drug Administration and European Medicines Agency, demonstrating impact on health and welfare and public policy and health services.
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.
Our research has had a major impact on the way pharmaceutical trials in Alzheimer's disease are conducted. The Boundary Shift Integral technique, which we developed and validated, has changed commercial practice and has become the industry standard for measuring atrophy progression. Our methods have largely replaced previous manual measures and in 2008-13 were used in over 20 large international trials. This had significant economic benefits for several companies providing image analysis services. For UCL alone they generated over £5m of industrial contracts. Additionally, through licensing and collaboration, UCL's research contributed to IXICO establishing a significant market share in this important commercial area.
The underpinning research delivered the first concrete and specific insights into service organisation, content and provision for people with Parkinson's (pwP). As a result, the research has been able to directly influence and inform government and professional body aims, policies and guidelines, and inform educational and clinical practice. The research has therefore contributed to the wellbeing of pwP in the UK and across Europe. Parkinson's disease is a degenerative neurological disorder which is estimated to affect 6.3 million people worldwide, including 1.2 million people across Europe and 120,000 people in the UK. Most pwP experience negative changes to their voice, speech, language and swallowing.
Our biomarker research and underpinning technologies have commercially impacted upon the global R&D strategies of Unilever, Philips and Mars, realising new market areas for them, resulting in several million GBP invested in related R&D as well as "claim support" for products both in development and already available on shelves. Unilever have adopted biomarker outcomes as endpoints in clinical trials of new products, and Philips and Mars are developing with us saliva-based near-patient diagnostic tests for the human and small animal markets. We have also spun out two SME's: A) Oral Health Innovations (OHI) Ltd has developed online risk and disease analysis software for oral conditions, which was piloted, adopted and launched by Denplan, the UKs largest dental capitation plan operator (accessing 6500 dentists and 1.8 million patients), at the 2013 annual British Dental Association conference; and B) GFC Diagnostics makes SmokeScreen™ a non-invasive, sensitive and objective saliva test developed from our biomarker research at Birmingham University. Both technologies have already provided demonstrable social and commercial impact and given their uptake to date, will also deliver economic, environmental and health impacts.
Corticosteroids are the traditional mainstay of treatment for inflammatory conditions but their side effects are often severe, especially in children. Professor MacDonald's team researched alternatives to corticosteroids in childhood Crohn's disease. With Nestlé they developed a polymeric, milk-based formula feed (Modulen IBD) that was highly effective in inducing clinical remission. NICE guidance have changed to reflect these findings. The treatment is now first-line therapy for childhood Crohn's in UK and the rest of Europe and recommended in clinical guidelines in USA. We estimate that across Europe alone, 13,000 new cases of childhood Crohn's annually will be spared steroid therapy as a result of this work.
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.