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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.
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.
Researchers from King's College London (KCL) designed and trialled a series of Oral Mucosal Disease Severity Scoring Systems (ODSS) that are now used routinely in clinical assessment of both serious and common oral diseases. They have changed clinical practice and significantly improved patient care and quality of life. For example, using ODSS has changed the first line treatment for orofacial granulomatosis from drugs to diet control, optimising treatment and definition of disease phenotypes. ODSS has achieved national and international impacts by providing objective evidence for the efficacy of treatments and is now incorporated into international guidelines of good practice and core training for oral medicine specialists.
We established a comprehensive international collaboration to develop, validate and apply new scales for the identification and quantification of non-motor symptoms and signs in Parkinson's disease (PD). This was intended to provide tools to assess response to treatment, help define the clinical prodrome of PD and provide a means to evaluate novel therapies designed to modify the course of disease. The scales have been produced, validated and published. They have been incorporated as end points in international clinical trials for PD and have been introduced by specialist societies and NHS commissioners as a standard of care for PD patients.
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.
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.
The University of Liverpool (UoL) research identified corticosteroid treatment for more than 3 consecutive months as a risk for serious sepsis in Crohn's disease and an indicator of poor practice; there are 115,000 Crohn's disease patients in the UK. Following this, national audits of Inflammatory Bowel Disease (IBD), also under UoL leadership, showed reduction in inappropriate long term steroid from 46% of Crohn's disease patients in 2006 to 21% in 2010. These audits led to widespread adoption of National Service Standards for the Care of Patients with IBD. Death and hospital readmission rates for IBD patients were subsequently significantly reduced.
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.
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.
The Dermatology Life Quality Index (DLQI) questionnaire is a clinical and research tool, which has fundamentally shifted dermatology from being doctor-centred to patient-centred. Previously, no standard method to quantify the impact of skin disease on patients existed. The DLQI was created by interviewing people with skin disease and made clinically useful through development and validation of score bands. NICE/SIGN require UK dermatologists to use the DLQI when assessing severe psoriasis and hand eczema. DLQI is used in national psoriasis guidelines in 14 countries, is available in 91 language translations, has been used in 678 clinical research studies and generated £881,236 in royalties to Cardiff University.