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    • has been a remarkable year of scientific achievements from a

    2018-10-23

    2014 has been a remarkable year of scientific achievements, from advances in our understanding of molecular pathways of human diseases to the development of new interventional tools to tackle them. Here, we highlight some promising initiatives and success stories from the past year with clear translational relevance. The list is by no means exhaustive but hopefully it provides a perspective on some of the achievements that are good examples of the types of research aims to foster.
    The discovery of insulin more than 90years ago transformed T1D from a fatal disease to a manageable condition. Improvements in insulin formulation and development of devices for patients to deliver insulin and measure blood sugar have combined to produce one of the most remarkable success stories in drug and device development in the 20th century. Life expectancy in T1D has increased dramatically and many patients, particularly those who are able to sustain the demanding and costly self-care regimen that good glucose control requires, live long and healthy lives. But T1D remains a very serious chronic illness – commonly beginning in childhood – a disease that imposes a huge burden on the patients themselves, as well as their ag1478 and on society as a whole. Furthermore, despite advances in insulin delivery and glucose monitoring, it is extremely challenging to achieve long-term glucose control, resulting in significant risks for acute and chronic complications. In addition, the incidence of T1D continues to increase, especially in the very young. In order to reduce the human suffering related to T1D as well as the large financial costs of T1D and its complications, efforts at curing T1D in those who have already been diagnosed, and preventing it for the future, are of paramount importance.
    Biotherapeutics have greatly advanced the world of medicine, and products such as monoclonal antibodies, fusion proteins and receptor antagonists have transformed the therapeutic armamentarium for rheumatic and musculoskeletal diseases (RMD) in the last 20years. Biological understanding of a disease can result in novel targeted therapeutics, whereas new molecular pathological insights can stem from observations when patients have responded differently to the same treatment. Rheumatoid arthritis (RA) provides an excellent example of the bidirectional nature of translational research involving clinicians and scientists working in concert with mutual understanding of both the clinical problem and the scientific solution. RA biotherapeutics are effective in many but not all patients, and adverse effects, namely increased infections, may limit their use. The current challenges for translational research and biomedicine in RMD/RA therefore focus on the following issues: (i) early diagnosis, (ii) personalised medicine, and (iii) assessment of outcome. The unifying theme to these challenges is the development of biomarkers, as will be evidenced in the next paragraphs. There have been significant advances in relation to early diagnosis for RMD over the past 10years. Clinical awareness and the importance of an early diagnosis were increased from 1991 with the growth of early arthritis clinics and the application of the 1987 diagnostic criteria for RA. These classification criteria were updated in 2010 with significant changes. A new biomarker for RA – anti-citrullinated antibody (anti-CCP) – was first suggested when citrulline was described as an essential antigenic constituent of RA-specific antibodies (). Anti-CCP antibodies only became widely available commercially as recently as 2007, and since this time their utility has been expanded to include both diagnostic and prognostic markers of RA, as they are recognised to be associated with poor prognosis or outcome as well as diagnosis (). Even more recently, the presence of anti-CCP and rheumatoid factor antibodies has been described in some subjects year before they develop the signs or symptoms of the disease (). This knowledge has led to a number of strategic studies exploring treatment options for very early RA, at a stage that subjects with joint symptoms and positive antibodies may receive therapy before signs of RA develop. The full implications of these studies are awaited ().