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There are 10 million new infections and one million deaths from tuberculosis annually and there is an increase in resistant diseases. Yet there have been no new anti-tuberculosis agents developed for forty years. TB drug development is expensive because of the time taken for the organism to grow and because trials are expensive and the sample size is high. The biomarker and mathematical methods developed at St Andrews address these problems by making preclinical development faster and cheaper and is being used by three commercial companies and eight drug development groups. These methodologies shorten the time taken to complete trials and reduce cost.
Lung cancer is the commonest cause of cancer-related mortality worldwide. The University of Manchester (UoM) Lung Cancer Group has generated insights that underpin new standards of care in the treatment of advanced, metastatic small cell (SCLC) and non-small cell lung cancer (NSCLC), contributed to the results required for licensing of new drugs and secured approval for new treatment regimens now in routine clinical use internationally. Key contributions include an increase in survival of 23% in advanced NSCLC with the use of chemotherapy and doubling one-year survival from 13% to 27% in patients with incurable, extensive stage SCLC by the use of prophylactic cranial irradiation. The Group's research has impacted on outcomes for thousands of patients worldwide.
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.
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.
A research team, led by Professor John Robertson, was joined by Professor Herb Sewell as lead collaborator. They developed a blood test that permitted early detection of lung cancer in high risk patients, allowing earlier and more successful treatment. The EarlyCDT-Lung test was commercialised by the university spin-out, Oncimmune, and launched in 2010. It is in clinical use in North and South America, in private clinics in the UK and in some Middle East countries, generating employment and revenues for the company, and is starting to bring mortality and lifestyle benefits to patients and their families.
Research on professionals' discussions about clinical trials of cancer therapy has identified the major barriers to patient recruitment to clinical trials. This research was used to create an educational intervention to improve patient experiences and willingness to participate in a variety of clinical trials worldwide, resulting in increased participation in prostate, colorectal, renal and breast-cancer trials. It also involved educating members of UK cancer teams to the best ways to approach, communicate and maximise trial planning.
Research at the University of Manchester (UoM) has led a step-change in respiratory care for airway disease from oral to novel inhaled therapies targeted at asthma and chronic obstructive pulmonary disease (COPD) patients worldwide. UoM researchers carried out >250 studies, partnered industry to deliver >15 new inhaled drug formulations to market and were the first to test novel CFC-free inhalers. UoM led the development of global guidelines that influence better diagnosis and management of airways diseases. Through leadership within the Montreal Protocol since 1995, UoM researchers coordinated the safe global transition to CFC-free inhalers for ~200m patients with asthma and COPD, whilst protecting the ozone layer and climate.
Research from the University of Nottingham on aminoglycoside antibiotics in cystic fibrosis (CF) has changed clinical practice and improved patient safety internationally. There are over 70,000 people with CF worldwide. Most require frequent and prolonged intravenous courses of aminoglycoside antibiotics (which can cause kidney damage) to treat chronic lung infection with Pseudomonas aeruginosa. This infection may lead to respiratory failure and death. Our research has influenced national and international guidelines, and changed practice, such that once-daily aminoglycosides (less toxic to the kidneys) are now used. We have also stopped the use of gentamicin, in favour of less toxic aminoglycosides.
We have made substantial contributions to the diagnosis of lung disease by providing tools to assess and interpret lung function accurately across the entire lifespan. These contributions include: effects of lung disease being more clearly distinguished from those of normal growth, development and aging; increased understanding of the early determinants of adult respiratory disease and improved diagnosis of chronic obstructive lung disease. Commercially available equipment for assessing lung function in infants and preschool children has been developed based on our work and our recently developed multi-ethnic, all-age lung growth charts have been endorsed internationally and are now in widespread use.
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.