Summary of the impact

Research at the University of Nottingham has defined the clinical phenotype and management of lymphangioleiomyomatosis, a rare and often fatal multisystem disease affecting 1 in 200,000 women worldwide. The group has led the development and evaluation of new therapies and diagnostic strategies which are now part of routine clinical care. The research has underpinned the transformation of this previously under recognised and untreatable disease into a condition recognised by respiratory physicians, with international clinical guidelines, patient registries, clinical trials, specific treatments and a UK specialist clinical service.

Summary of the impact

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.

Summary of the impact

Psynova Neurotech is a prize-winning spin-out company founded by Professors Sabine Bahn and Chris Lowe from the University of Cambridge. It focuses on the commercialization of novel blood-based biomarker tests for conditions like schizophrenia, depression and bipolar disorder. Psynova and its partner company Rules Based Medicine (now Myriad RBM Inc.) launched the first commercially available Aid for the Diagnosis of Schizophrenia (VeriPsych^TM) in 2010. In June 2011, Psynova and Rules Based Medicine were acquired by Myriad Genetics Inc. for £50 million. In February 2011 Psynova Neurotech and Professor Bahn were announced winner of the ACES best European Life Science spin-out award.

Summary of the impact

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.

Summary of the impact

CANTAB-Paired Associates Learning (PAL) was developed to detect early memory problems in Alzheimer's disease; and was recently (in 2012) launched by Cambridge Cognition (floated on the London Stock Exchange in April 2013) as a mobile (iPad) application (CANTABmobile™) suitable for use in GP clinics. This and other cognitive tests from the CANTAB battery have also been employed in 77 clinical trials since 2008, involving hundreds of sites world-wide, by most of the major pharmaceutical companies and by biotech, device and nutraceutical companies. CANTABmobile™ currently has 166 licensed user-practitioners including six clinical commissioning groups implementing the national initiative for early diagnosis.

Summary of the impact

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.

Summary of the impact

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.

Summary of the impact

Visensia^TM is a bedside `early warning' system, deployed in many hospitals in the UK and US, which automatically analyses hospital patients' vital signs, produces simple-to-read scores, and alerts healthcare staff to any deterioration in a patient's condition. It resulted from research in this Department, commercialised by Oxford BioSignals Ltd (£1.5m sales to date, and 137 licences sold since 2010). Visensia^TM reduces the number of patients already in hospital who suffer an unexpected cardiac arrest or need an unplanned transfer to intensive care. The US Food and Drug Administration (FDA) approved the system's use after a 1000-patient clinical trial. There were no unexpected fatal cardiac arrests on the wards where the clinical trial took place in the three years after Visensia^TM. was deployed.

Summary of the impact

The capacity for cognitive function may be missed by clinical examination in severely disabled survivors of acquired brain injuries, resulting in individuals being mislabelled as being in the vegetative state (VS). Work from David Menon and John Pickard has shown that functional brain imaging provides a more consistent and less observer-dependent means of detecting and quantifying such cognitive capacity. As a result of this work, the use of functional imaging has been integrated into clinical protocols as the basis for: identifying patients with such covert cognition; prognosticating on outcome; defining a rational framework for patient selection in clinical trials; and exploring the use of brain-machine interfaces to improve communication with such patients.

Summary of the impact

1. Practitioner/professional service impact. Our work on clinical outcome modelling has influenced the Royal College of Physicians' (RCP) new standard for the assessment of the severity of acute illness (known as the "National Early Warning Score" or NEWS). The specific recommendation is for adoption by NHS bodies, but is already being adopted internationally.
2. Health impact. The chairman of the RCP working party estimated that our work could result in the saving of thousands of lives per year.
3. Economic impact. Our work is incorporated in the VitalPAC system developed by The Learning Clinic Ltd (TLC), and currently deployed to more than 20 hospitals.