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New therapies supported by clear evidence from clinical trials have resulted in outstanding improvements in survival and quality of life for people living with cystic fibrosis (CF). Elborn's clinical trials group has delivered a programme of crucial clinical trials which has impacted on clinical practice in CF. From 2009-2012 Elborn co-led a pivotal multicentre trial using Ivacaftor (Kalydeco TM), a transformative new drug which represents a paradigm shift as the first approved therapy that corrects the basic defect in CF. This therapy is an exemplar of personalised medicine and is prescribed for patients with the specific gene mutation in which this drug works.
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.
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.
In 2010, 8,483 cases of tuberculosis (TB) were reported in the UK, mainly in urban areas and with London and the West Midlands having the highest rates of disease (rate within Heartlands Primary Care Trust 80+ per 100,000). Research led by Professor Peter Hawkey at the University of Birmingham has resulted in the development of novel techniques for real time typing of Mycobacterium tuberculosis strains. The new cost effective and rapid methodology has been adopted by the three UK national reference laboratories and has resulted in significant improvements to the national TB typing scheme and TB infection management. An associated secure IT system has been developed to enable TB control teams to rapidly receive typing data together with an analysis of the local cases. This has influenced changes in clinical practice by reducing the need for contact tracing. Use of the new techniques developed at Birmingham has resulted in faster, more accurate identification of outbreaks of TB and this information has been used to significantly improve patient management.
Prior to the change in WHO recommendations which occurred following this study many patients in Africa and other developing countries were receiving an inferior regimen for the management of tuberculosis, a consequence of which meant that many had to be retreated. Since the implementation of the revised WHO Guidelines in 2010 almost all countries have now switched to the gold standard tuberculosis treatment regimen based on 6 months of isoniazid and rifampicin
Research at the University of Liverpool (UoL) has developed and proven a straightforward diagnostic test method for bacterial blood infections. This was urgently needed as sepsis is a medical emergency that lacks adequate and rapid diagnostic tests particularly for low cost early detection. UoL's research has demonstrated that a simple optical test that can be conducted during routine testing of coagulation is an effective diagnostic, prognostic and monitoring marker for sepsis that can be routinely applied in clinical settings. There are now established UK and international laboratory standards in place. In 2010 a spinout company was formed to exploit four patents and incorporate the technology into a point-of-care device suitable for all clinical settings. The company, Sepsis Ltd, has attracted £1.45m of investment.
The Cancer Research UK Formulation Unit at the University of Strathclyde performed the pharmaceutical research and development of new chemotherapy treatments for malignant brain and prostate cancer (temozolomide and abiraterone acetate). These two drugs are now marketed globally, with FDA approval for the US market in 1999 and 2011 respectively, and have directly improved the quality of life and increased survival rates during treatment for over a quarter of a million cancer patients annually since 2008. Temozolomide achieved $1 billion sales per annum in 2008, and Abiraterone global sales reached $1.45 billion by 2013. Both drugs have produced economic benefit to the charity Cancer Research UK through royalty payments.
This case study summarises a body of research on Multiple Sclerosis (MS) developed at Plymouth University under the leadership of Professor Zajicek and Professor Hobart. Hobart's work on linical outcome measurements has directly influenced clinical research, trials and drug licensing, especially in MS and Alzheimer's disease. The MS scales developed by Hobart have been endorsed by the United States FDA and are in demand by commercial organisations in the development and trialling of treatments for MS and have led to the licensing of new drugs. Zajicek has led the topical field in evaluating the potential benefits and risks of cannabis for treating MS, contributing to the evidence base behind the medical use of cannabinoids in general, and pioneering its global potential use to slow neurodegeneration.
Research co-led by Prof Roz Anderson, in collaboration with a multi-disciplinary team, resulted in a new chromogenic substrate for the rapid detection and specific identification of the bacterial pathogen, Pseudomonas aeruginosa, a `super-bug' that threatens many thousands of hospital patients annually, leading to poor clinical outcome and increased risk of mortality.
bioMérieux adopted the technology for a new product, ChromID® P. aeruginosa, for commercial realisation as a clinical microbiology test; it was launched in the EU, USA and Australia, supporting the company's commercial position as leaders in this field. This test has enhanced the care of patients, through more rapid detection of P. aeruginosa and earlier informed clinical decision- making.
Approximately 152,000 strokes and 49,000 stroke-related deaths occur in the UK every year; of these, 85% are caused by blockage of a blood vessel in the brain (acute ischaemic stroke). The economic burden of stroke in the UK is estimated at £3.75bn with hospital inpatient care accounting for 82% this cost. Since the 1990s advances in thrombolytic treatments (which dissolve blood clots) have limited the extent of damage and subsequent impairment; however their use has been restricted due to ambiguity between stroke onset and stroke symptom presentation. University of Glasgow research has challenged the restrictions associated with thrombolysis treatment which has significantly influenced the wider use and applicability of thrombolytic treatment. This research has influenced new guideline recommendations and emergency stroke care patterns, through the implementation of dedicated acute stroke centres, and contributed to the on-going improvement in stroke survival rates.