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The Abraham solvation parameter approach developed at UCL has become integral to the work carried out by drug discovery teams at [text removed for publication] and other major pharmaceutical companies, as well as research and development groups at international chemical companies including Syngenta and [text removed for publication]. It enables chemists to predict physicochemical and biochemical properties of chemicals, including drugs and agrochemicals, rapidly and efficiently, without the need to conduct time-consuming experiments. The method helps drug discovery teams to identify and optimise the most promising compounds, and often results in fewer compounds being made before a candidate is selected, saving time and resources. The approach has been integrated into software used for drug discovery [text removed for publication].
As a result of our discoveries of a new splice variant (ASIC1b) and a new member (ASIC4) of the ASIC family, and elucidation of their roles in pain caused by tissue acidity, several pharmaceutical companies are now working on ASIC-targeted analgesics and one company has been set up specifically to focus on this work. ASIC-related therapies for a wide variety of conditions are now in clinical trials, with substantial patient involvement. Our work has allowed new therapeutic applications to be conceived for already existing prescribed compounds, and for naturally-occurring compounds, that act on ASICs. Thus, our research on ASICs has had clinical and commercial impact.
Haemophilia, an inherited bleeding disease, is treated by frequent and extremely expensive infusions of recombinant versions of the missing factors. Advances in gene therapy have now been achieved at UCL, with successful treatment of Haemophilia B in 10 severely affected patients. The novel factor IX expression cassette has been patented and licensed to an industrial partner (UniQure). Savings to the NHS in excess of £1.5m have already been made and increase every month. Pre-clinical advances have also been made in Haemophilia A, and the factor VIII expression cassette has been patented and licensed to an industrial partner (BioMarin).
Alternatives to acute admission in mental health are crucial, not least because of the high cost of inpatient care. We have carried out a major research programme that includes the only randomised controlled evaluation of crisis resolution teams and the only major UK study of crisis houses, which are community-based, residential alternatives to hospital admission. This programme demonstrated the efficacy of community treatment and has significantly influenced decision making at a local and national policy level, including commissioning guidance and three sets of NICE guidelines. This has contributed to changes in the way acute services for severely mentally ill adults are configured in the NHS, and internationally.
Our research on alternatives to medication in the treatment of childhood epilepsy has resulted in increasing rates of surgery with better outcomes, and a new clinical service — the national Children's Epilepsy Surgery Service (CESS) — being commissioned in England and Wales. We have also developed an evidence base for ketogenic dietary therapy, resulting in an increase in service provision. Many more patients are benefiting from this therapy, which is now recommended in NICE guidelines. Throughout our programme of research we have engaged with charities and patient groups to disseminate the results of our research as widely as possible.
The UCL Centre for Amyloidosis and Acute Phase Proteins has designed and developed new chemical entities targeting serum amyloid P component (SAP), C-reactive protein (CRP) and transthyretin, for novel therapeutic approaches to amyloidosis, Alzheimer's disease, cardiovascular and inflammatory diseases. The UCL spin out company, Pentraxin Therapeutics Ltd, founded by Sir Mark Pepys to hold his intellectual property (IP), has licensed two programmes to GlaxoSmithKline (GSK). These highly synergistic, collaborative multi-million pound developments, strikingly exemplify new working relationships between academia and the pharmaceutical industry.
Clare Stanford's group has opened up a new line of research for drug treatment of Attention Deficit Hyperactivity Disorder. Based on this work, UCL Business has been awarded an EU patent for the NK1R `knockout' mouse as an investigative tool and pharmaceutical screen. Cerebricon, a subsidiary of Charles River, has taken an exclusive licence to market this mouse and advertise it on their website. Our studies have also enabled us to identify a new genetic locus in which abnormalities are linked to ADHD in humans, and to identify a new drug candidate for treating ADHD.
Research by Dr Andrew Martin at the UCL Research Department of Structural & Molecular Biology has led to a series of antibody-related tools being made available for free use over the Web. One of these, Abysis, has been visited over 360,000 times by over 8,000 users. Abysis has also been released under a commercial license and has been purchased by companies ranging from small biotechs to large pharma for use in their antibody therapeutic development pipelines, allowing them to identify unusual features of their sequences and to improve strategies for humanisation. Martin has also acted as an expert witness for drug companies in patent disputes.
Research by Marianne Odlyha and her group at the Department of Biological Sciences, Birkbeck, University of London, has led to the development of minimally invasive analytical methods and portable tools (dosimeters) for assessing damage to historical artefacts. These dosimeters are now in use at locations around the world, including the Tate Gallery's store rooms, English Heritage properties (Apsley House) and museums in Ghent, Cracow and Mexico. Methods for assessing damage, and for mitigation of pollutant impact on objects in museum enclosures, have been disseminated to conservation professionals through workshops and training courses held across Europe. The assessment and prevention of damage is vital to conserve the cultural as well as the monetary value of artefacts.
Our research into the epidemiology of childhood visual impairment has transformed understanding of the epidemiology, outcomes and impact of childhood visual impairment in the UK. As a result, changes have been made to the Newborn and Infant Physical Examination Programme and the National Vision Screening Programme. Our work has underpinned the development of new quality standards for paediatric ophthalmology services issued by the Royal College of Ophthalmologists. Our studies on congenital cataract have improved the management of this condition nationally. We have involved visually impaired service users and patient groups directly in our research to improve quality of life measures and patient engagement.