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The UCL Centre for Amyloidosis and Acute Phase Proteins conducts world-leading research and development that has rapidly fed through to patient care. These include advances in the diagnosis of amyloidosis and clinical characterisation of many new subtypes including genetic forms, development and application of new biomarkers to monitor disease activity and progress, new modalities of imaging and better treatment. The consequences have been new standards of clinical care adopted nationally and internationally, improved and more accurate diagnosis, steady improvements in the outcomes of this disease, major investment by the NHS and adoption of new clinical metrics by the pharmaceutical industry to enable research on specific new therapies currently in development.
Pioneering research led by the University of Aberdeen has directly resulted in the development of an investigational medicinal product for the long-term management and prevention of Alzheimer's disease, breaking new ground in the search for effective Alzheimer's treatments. Although not yet commercially available, this drug has already benefited more than 100 patients and their families. A new spin-out company created to develop the drug has created new jobs and attracted more than US$335 million in investment since 2008. Extensive media coverage of the research has generated increased public awareness of the disease and Aberdeen's cutting-edge research and ability to raise investment. The claimed impact is therefore that a new spin-out company was formed; investments from and collaborations with industry in research and development were generated; and new employment created.
Research by a team at Southampton into amyloid beta protein (A03b2) immunisation to treat Alzheimer's disease has been key to changing the way the global medical community understands and reacts to the disease. The first to observe that A03b2 immunisation clears A03b2 plaques, the team's studies were pivotal in initiating and informing the safe clinical trial development of 40 immunotherapy agents; investments of $3bn by the pharmaceutical industry; and 30 phase II and phase III studies. The research shaped US government policy on new safety measures for clinical trials and played a leading role in the doubling of UK funding to tackle Alzheimer's.
The drug galantamine (Reminyl) received approval for the treatment of early stages of Alzheimer's disease in 2001. However it was not made available on the NHS until March 2011, the effective onset date for the impact. The decision as to whether a treatment is available on the NHS is made by the National Institute of Health and Care Excellence (NICE), who sought additional clinical data and a rationale for the action of the drug. The mechanism of action was elucidated by Lancaster researchers that included chemists and biomedical scientists. These results were part of Alzheimer's Society's campaign to convince NICE to make the drug available on the NHS for early stage Alzheimer's. The resulting impact was direct, enhancing the quality of life for 100,000s of Alzheimer's patients (318,000 galantamine prescriptions were dispensed in the UK in 2012 [8]), with indirect impact on spouses, immediate family, and carers. The impact continues as new patients come into the pool.
Scientists in the MRC Cyclotron Unit within Imperial College pioneered quantitative Molecular Imaging methods for neuroscience drug development that have since been expanded through collaboration between Imperial and GlaxoSmithKline (GSK) scientists. Human Molecular Imaging has had significant commercial impact with adoption by the major pharmaceutical companies to reduce the risks and costs associated with early drug development. This led directly to the selection of the Imperial Hammersmith Hospital site for the world's first clinical imaging centre embedded in a pharmaceutical company. New GSK investment created new and highly skilled UK employment opportunities first at this GSK Clinical Imaging Centre (CIC) and then Imanova, Ltd., a specialised imaging CRO that was "spun out" from the CIC. Outcomes from studies commissioned by GSK in the CIC and later in Imanova have directly influenced GSK clinical development planning, strategy and drug candidate progression. More recently, outcomes from Imanova are influencing clinical development decisions of other pharmaceutical organisations in similar ways.
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.
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 (VeriPsychTM) 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.
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.
Psoriatic arthritis (PsA) is a chronic inflammatory disease of joints, skin and tendons that affects 0.5-0.8% of the population worldwide. PsA can cause substantial psychological and social problems and also causes increased risk of death from cardiovascular disease. Research conducted by Prof Iain McInnes at the University of Glasgow in partnership with leading pharmaceutical company, Janssen, has provided robust evidence of the clinical benefits and safety of the cytokine blocker ustekinumab, leading to its approval for use for PsA by the European Medicines Agency in July 2013. This was the first approval of a PsA drug with a new mode of action in a decade, providing a novel treatment for approximately 1.25 million PsA patients across Europe.
Galantamine (Reminyl®) is one of the drugs recommended by NICE for treatment of Alzheimer's disease (AD). Until recently, it was approved only for the moderate stage of AD. In 2011, NICE guidance was changed to recommend that this drug could also be prescribed for early-stage AD. This has had a major impact on the lives of AD sufferers. In published research arising from an Alzheimer's Society Project Grant, Prof. Allsop at Lancaster demonstrated that Galantamine inhibits Aβ aggregation and so should be prescribed as early as possible during the course of AD due to its potential disease-modifying properties. This research underpinned arguments made by the Alzheimer's Society who were one of the key players in pressing for the change in NICE recommendations.