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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.
Rheumatoid arthritis is a debilitating inflammatory condition, affecting around 500,000 people in the UK and around 0.5-1% of the adult population worldwide. Using novel techniques to study human synovium, Professor Sir Marc Feldmann and Professor Sir Ravinder Maini from the Kennedy Institute of Rheumatology identified a therapeutic target, TNFα, for treatment of rheumatoid arthritis. Following successful clinical trials, showing the safety and effectiveness of this new target, anti-TNFα antibodies have now become the gold standard treatment for severe rheumatoid arthritis worldwide. In addition to dramatically impacting patient care, anti-TNFα antibodies represent the largest group of therapies against rheumatoid arthritis on the market, with annual sales currently exceeding US$24.4 billion.
An estimated 1% of UK adults suffer from rheumatoid arthritis and the long-term pain and disability associated with it, Historically, however, treatments focused on relieving symptoms and did not control the arthritis itself or prevent disability. An extensive series of clinical trials and associated research programmes at King's College London (KCL) over 20 years has now significantly improved treatment recommendations and thus quality of life for thousands of rheumatoid arthritis patients in the UK, Europe and other countries. Multicentre trials of intensive treatments using conventional drugs have extended the range of drugs available, established the effectiveness of early intensive treatment, and shown that early combination therapies are safe.
Research in Leeds led by Professor Paul Emery pioneered early diagnosis and treatment for patients with rheumatoid arthritis (RA), with the aim of disease remission rather than reduction of symptoms. This approach has transformed management of RA and is now standard practice for patients worldwide. It has led to greatly improved disease control, increased quality of life and reduced disability as well as direct productivity gains of an estimated £4 million per year to the UK economy.
The research described below has made a major contribution to the clinical and preclinical development of endothelin receptor antagonists (ERAs) for the treatment of systemic sclerosis (SSc). As a result, ERAs are now standard management for pulmonary arterial hypertension related to connective tissue disease and specifically complicating SSc. This work has also led to the licensing of bosentan (one of the ERAs) for digital ulcer disease, a major non-lethal complication of SSc that impacts on quality of life, employment status and the major economic cost of SSc management. By 2012, more than 16,000 patients with SSc had been treated worldwide with these therapies, with numbers increasing every year.
Rheumatoid arthritis (RA) is a costly and debilitating autoimmune disorder that is characterized by joint pain, stiffness, and impaired functionality. Work at Imperial College identified tumour necrosis factor (TNF) as a key therapeutic target in the abnormal joint lining in RA. This discovery revolutionised the treatment of Rheumatoid Arthritis and other inflammatory conditions. Since 2008 the anti-TNF inhibitor infliximab (Remicade®) has been used to treat more than 1.3 million patients worldwide who have inflammatory conditions such as plaque psoriasis, rheumatoid arthritis, psoriatic arthritis, adult and paediatric Crohn's disease, ulcerative colitis, and ankylosing spondylitis. The work has had ongoing impact across the globe for the treatment of inflammatory diseases. It established the concept of biological therapy demonstrating the use of an antibody to block a cytokine and treat chronic inflammatory disease. In 2012 Remicade® was the 4th best-selling worldwide drug with total global sales of $7.67 Billion.
The UCL Centre for Amyloidosis and Acute Phase Proteins has identified the cause and treatment for the prototypical cryopryin associated periodic syndrome (CAPS), and subsequently for a range of other hereditary and acquired autoinflammatory disorders. As a result of the research, canakinumab was licensed for this condition. In recognition, NHS Specialised Services commissioned the UK CAPS Treatment Service in 2010 to deliver life-changing IL-1 blocking therapy to the national caseload of CAPS patients at UCL.
Rheumatoid arthritis (RA) is a common destructive joint disease, causing pain and swelling, affecting 1 in 100 people. Work conducted by the University of Birmingham's Rheumatology Research Group has shown that early diagnosis is important, as the first few months represent a critical therapeutic window during which treatment can significantly improve health outcomes, increasing the chances of achieving disease remission and reducing the rate of progressive joint damage. The group have demonstrated that there are significant delays in patients making initial contact with their GP, which leads to delays in referral to a Rheumatologist and starting treatment; this situation has been shown to be worse in patients of South Asian origin. The outcome of the work has been incorporated into national policy documents and clinical guidance material and has underpinned a patient focused campaign to raise awareness of the disease and the need for early diagnosis.
Chronic granulomatous disease is a rare but very serious inherited disorder of the immune system that leaves sufferers vulnerable to potentially fatal bacterial and fungal infections. Researchers at Newcastle University demonstrated very high survival and cure rates following bone marrow transplantation for the disease and good quality of life for successfully transplanted patients. This led to a change in national clinical policy, and doctors at both specialist disease centres in the UK now recommend transplantation to families where previously they would not have done so. In the five years prior to 2008 there were only 11 transplants for chronic granulomatous disease in the UK and in the following five years, 36 transplants. 32 children are alive and cured of the disease.
Research conducted by Professor Tim Goodship and co-workers at Newcastle has had a profound effect on the prognosis for patients with atypical haemolytic uraemic syndrome (aHUS). By engaging in research on the genetic factors underlying the disease they developed an understanding of the molecular mechanisms responsible. Identifying that the majority of patients with aHUS have either acquired or inherited abnormalities of the regulation of complement (part of the immune system) led to the establishment of a UK national service for genetic screening and treatment with the complement inhibitor eculizumab. As eculizumab is now available to patients in England, the progression to end-stage renal failure can be prevented and patients already on dialysis will soon be successfully transplanted.