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Knowledge of the three-dimensional structures of macromolecules is a prerequisite for understanding their function at the atomic level, an essential component of modern drug development. Most structures are determined by X-ray crystallography: the majority using molecular replacement (MR, which exploits known structures of related proteins), and about half of the remainder using single-wavelength anomalous diffraction (SAD). The Phaser crystallographic software, developed by Read and colleagues, implements powerful new likelihood-based methods for MR and SAD phasing and has made a large impact, accelerating over the period 2008-2013. At the pharma giant, AstraZeneca, Phaser is considered the "tool of choice" for solving structures by MR.
Collaboration between Imperial College Departments of Mechanical Engineering and Surgery led to the development of active constraint robot solutions which augment surgeon skills so that joint replacement components are implanted accurately and successfully. This led to the founding of Acrobot to develop innovative surgical technologies. Acrobot was acquired by Stanmore Implants Worldwide in 2010. An orthopaedic stereotaxic instrument, based on Imperial research, obtained US Food and Drug Administration (FDA) clearance in 2013. This has led to Mako-Surgical purchasing Stanmore Implants Acrobot technology in April 2013.
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).
The electrocardiogram (ECG) is one of the most commonly used medical tests which assist in diagnosing heart disorders worldwide. However, diagnosis relies on accurate interpretation of ECG recordings. Studies by University of Glasgow researchers have led to changes to international guidelines for ECG-based diagnosis of a heart attack (myocardial infarction; MI) and have led to significant refinements to the automated ECG analysis software called the `Glasgow Program'. Commercialisation of the Program since 2008 has resulted in its incorporation into some of the market-leading medical devices, with approval of the Glasgow Program by the FDA and more than 40,000 devices sold worldwide, potentially aiding millions of patients around the world. The Program assists hospital doctors, family practitioners and others such as first responding emergency services, e.g. ambulance and fire services, with the reliable interpretation of ECGs, enabling rapid and accurate diagnosis and treatment of patients with a variety of heart problems.
Research by the University of Southampton has helped transform the understanding and treatment of chronic lymphocytic leukaemia (CLL), the most common leukaemia, affecting around 2,400 patients each year in the UK and 17,000 in the USA. Southampton's widely cited studies revealing the existence of two subsets of CLL have been crucial in giving clinicians and patients in the UK and overseas a much clearer indication of the likely disease course. The predictive information is now included in all clinical trials and in international guidelines, delivering greatly improved care. The research has also inspired the development of a new drug given "breakthrough" status by the Food and Drug Administration in the United States.
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.
High-profile cases of growth hormone misuse by leading international sports stars have made the development of an effective detection test a main priority of global anti-doping organisations. A research team headed by the University of Southampton developed a new test, adopted at the 2012 Olympic Games, which identified two drugs cheats at the London Paralympic Games just weeks after it was first introduced. The subsequent bans serve to act as a powerful deterrent to other athletes and help restore public confidence in fair competition. The World Anti-Doping Agency has announced its commitment to rolling out the test internationally.
As a result of University of Sheffield research in 1995-2002, a new gold standard treatment for major bleeding on warfarin has been established, ensuring the more effective treatment of tens of thousands of patients requiring emergency anticoagulation reversal each year in the UK alone. The treatment, using prothrombin complex concentrate (PCC) was demonstrated to be superior to fresh frozen plasma (FFP), the standard alternative at the time, and two PCCs have now been licensed for this indication in the UK.
UK and international guidelines now recommend PCC over FFP.
Trauma is a rapidly increasing global healthcare problem which is predicted by the World Health Organisation (WHO) to overtake infectious disease globally by 2020. The discovery of the acute coagulopathy of trauma (uncontrolled bleeding) and the subsequent establishment of the clot stabiliser tranexamic acid (TXA) as a treatment for this condition has led to a change in national and international trauma management protocols. British armed forces and the US military implemented the use of the drug soon after the results were published. Every injured British or American soldier now receives this treatment. The use of TXA has been included in national and international guidance for trauma care.