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Research on Congenital Adrenal Hyperplasia (CAH) at the University of Sheffield has resulted in both health and commercial impacts. The research has led to a new drug treatment, Chronocort®, being developed for CAH. Chronocort® has been tested in CAH patients with the positive outcome of improved disease control.
Commercial impact arose from the creation of a spin-out company, Diurnal Ltd, in 2004 which has raised investment of £3.8M since 2008, including £0.4M from pharmaceutical industry sources, and (as an SME partner) a €5.6M Framework 7 grant to develop a paediatric treatment for CAH. Diurnal has created five new jobs and has contracts with six UK companies worth £2.7M.
Dialysis has revolutionised the management of End Stage Kidney Disease (ESKD), but the benefits of this invasive, demanding treatment may not be clear-cut for elderly, frail patients with other serious comorbidities. University of Hertfordshire and East and North Hertfordshire NHS Trust researchers have led the development of Conservative Management, an alternative to dialysis for some patients, providing multidisciplinary support and careful symptomatic management until death. The research shows that quality of life is maintained, survival may not be significantly compromised, and preferred place of death is more often achieved than for counterparts on dialysis. Conservative Management programmes have been adopted across the UK and elsewhere, influencing the care of many patients.
Congenital myasthenic syndromes (CMS) are inherited neuromuscular disorders caused by defects at neuromuscular junctions, which are often a result of acetylcholine receptor gene mutations. A subset of CMS patients (around 14% in the US and Europe) have limb-girdle myasthenia (LGM). This disease can be highly disabling with symptoms including increasing weakness of skeletal muscles. As a result of collaborative work between Newcastle and Oxford, it was determined that many LGM patients have a mutation of the Dok-7 gene (unrelated to the acetylholine receptor), and do not, therefore, respond to standard CMS treatments. Since then, a number of additional mutations have been discovered, and genetic testing is now available for the majority of known LGM-causative genes. Crucially, Dok-7 patients, and those with other non-receptor related mutations, can now be diagnosed accurately and treated effectively, with ephedrine and salbutamol (in the US, albuterol). This significantly improves these patients' quality of life by enabling them to walk and breathe unassisted.
The discovery of an early Acute Traumatic Coagulopathy (ATC, a syndrome of abnormal clotting after trauma) by Professor Brohi's team in 2000, and subsequent work building on that pivotal discovery, has led to [A] a new understanding of why patients bleed to death after severe injury and resulted in [B] a fundamental change in resuscitation strategy for acute bleeding patients (`Damage Control Resuscitation') that has led to [C] a 250-300 per cent improved survival in massively bleeding trauma patients. Discovering the character and mechanism of ATC has led to [D] new research in diagnostics and therapeutic opportunities to further improve outcomes. These rapid changes have led to [E] new forums for professional education and [F] improved public understanding of science and medicine.
Warfarin is an anti-coagulant drug prescribed to tens of millions of people in the UK and US who are at high risk of developing blood clots. Because individual sensitivity to warfarin varies in the population there is a risk of overdosing the drug and causing serious bleeding and even stroke in many people when starting treatment. In 1999 researchers at Newcastle University were the first to demonstrate a statistically significant link between a person's genotype and the appropriate dose of warfarin. In 2010 the US Food and Drug Administration (FDA) mandated inclusion of a table of dose recommendations based on genotype in the warfarin prescribing information leaflet accompanying the drug. Newcastle research forms the basis of the 2009 international standard algorithm for gene-guided dosing of warfarin. This approach has been adopted by large US medical centres and the FDA states that it will prevent 17,000 strokes a year in the US.
Work on better management and identification of Medically Unexplained Symptoms (MUS) has led to the introduction of new treatments in primary care in England through the Improving Access to Psychological Treatment (IAPT) programme as well as having an impact on service planning and commissioning. These approaches have also been implemented into the routine training and practice of General Practitioners (GPs) in parts of Europe. A clinical and economic evaluation of a psychosocial approach to chronic fatigue syndrome using general nurses and development of a cognitive behaviour therapy approach has changed general practice and enhanced the patient experience for those with MUS.
Between 1996 and 2013 researchers at Swansea University evaluated service initiatives and changing professional roles associated with the management of patients with debilitating gastrointestinal disorders. This work showed the clinical and cost effectiveness of two main innovations: open access to hospital services for patients with inflammatory bowel disease; and increased responsibility for nurses, particularly as endoscopists. Our evidence has had a broad, significant impact on: national policy through incorporation in NHS strategies, professional service standards and commissioning guides; service delivery through the provision of increasing numbers of nurse endoscopists and the wide introduction of nurse-led open access to follow-up; and patient care, as documented in sequential national audits in 2006, 2008 and 2010.
The research has led to a clear and accepted definition of dignity which have been adopted by health care organisations and professionals. It has raised the awareness of professionals and carers of the needs and means to enhance patient's dignity. Resources have been developed and used to train and support staff to improve attention to patient dignity. The research has also influenced changes in national policy such as reducing use of mixed sex accommodation, and changes to healthcare practices such as improved hospital gown design and use of bed curtain clips and notices. The resources on patient dignity continue to be disseminated and adopted and inspired the Velindre Cancer Centre group which won the 2013 Nursing Times Dignity Award.
Research at the UCL Institute of Ophthalmology over the last 15 years has developed new treatments for management of uveitis and its sight-threatening complications, which have subsequently become standard practice. Our work, in previously untreatable disease, has allowed restoration of vision in many patients and prevention of further visual loss in others. Many patients have been able to reduce systemic medication, limiting adverse effects of treatment.
Research led by Professor Brown has led to widespread changes in clinical practice regarding the management of Hypertension. Following his demonstration that patients' response to drugs for Hypertension is variable (in a systematic manner), subsequent clinical guidelines acknowledged the variability among patients, and changed from recommending the same treatment for all patients, to an algorithm based on the Cambridge AB/CD rule. The simplicity of the AB/CD rule led to popularity among doctors, and adoption by national bodies — British Hypertension Society, NICE, and foreign guidelines, and by textbooks of Medicine. The guidelines arising from his research have contributed to improved health outcomes in the UK. Specifically, NICE's simple and rational guidance how to reach strict targets for blood pressure is credited with changing the UK from the poorest to best performing country in Europe.