NIH Researchers Discover New Inflammatory Disease
NIH Researchers Discover New Inflammatory Disease Article taken from: http://www.dddmag.com/news/2016/08/nih-researchers-discover-new-inflammatory-disease The just-discovered otulipenia is a rare and sometimes lethal inflammatory disease that causes fever, skin rashes, diarrhea and joint pain in young children. Credit: Darryl Leja, NHGRI. Picture taken from: http://www.dddmag.com/news/2016/08/nih-researchers-discover-new-inflammatory-disease National Institutes of Health researchers have discovered a rare and sometimes lethal inflammatory disease – otulipenia – that primarily affects young children. They have also identified anti-inflammatory treatments that ease some of the patients’ symptoms: fever, skin rashes, diarrhea, joint pain and overall failure to grow or thrive. Otulipenia is caused by the malfunction of OTULIN, a single gene on chromosome 5. When functioning properly, OTULIN regulates the development of new blood vessels and mobilization of cells and proteins to fight infection. NIH researchers published their findings Aug. 22, 2016, in the early edition of the Proceedings of the National Academy of Sciences. Contributing to the work were researchers from the National Human Genome Research Institute (NHGRI), the National Institute of Allergy and Infectious Diseases, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Heart, Lung, and Blood Institute and the NIH Clinical Center, all part of NIH, along with their colleagues in Turkey and the United Kingdom. The results have been amazing and life changing for these children and their families,” said Daniel Kastner, M.D., Ph.D., co-author, NHGRI scientific director and head of NHGRI’s Inflammatory Disease Section. “We have achieved the important goal of helping these young patients and made progress in understanding the biological pathways and proteins that are important for the regulation of the immune system’s responses.” Cells use biological pathways to send and receive chemical cues in reaction to injury, infection or stress. Otulipenia is one of several inflammatory diseases that occur when the immune system attacks the host’s own tissues. Inflammation is the body’s natural response to invading bacteria or viruses. The body releases chemicals that cause blood vessels to leak and tissues to swell in order to isolate a foreign substance from further contact with the body’s tissues. Inflammatory diseases affecting the whole body are caused by mutations in genes like OTULIN that are part of a person’s innate immunity (the cells and proteins present at birth that fight infections). An international network of scientists studying inflammatory diseases identified four children from Pakistani and Turkish families with unexplained skin rashes and inflamed joints. NIH scientists then searched for disease-causing genes using next-generation DNA sequencing, technology that allows researchers to sequence DNA quickly and economically. Once they found that the OTULIN gene was abnormal in the sick children, they studied the immune pathway in order to understand the mechanisms of disease and to improve treatment of these patients. They discovered a problem in the processing of a small protein, ubiquitin, which is critical to the regulation of many other proteins in the body, including immune molecules. In the affected children, the inability to remove the ubiquitin proteins from various molecules resulted in an increased production of chemical messengers that lead to inflammation (inflammatory cytokines). The researchers determined that the children with otulipenia might respond to drugs that turned off tumor necrosis factor, a chemical messenger involved in systemic inflammation. Inflammation subsided in the children who had been treated with anti-tumor necrosis factor drugs (TNF inhibitors). TNF inhibitors are also used to treat chronic inflammatory diseases such as rheumatoid arthritis. “The malfunction in this protein has not been previously linked to clinical disorders of the human immune system,” said Ivona Aksentijevich, M.D., staff scientist in NHGRI’s Medical Genetics Branch and study co-author. “This discovery suggests a direction that can be explored for development of new therapies for patients with a wide range of inflammatory diseases.” This study together with NIH’s 2016 identification of haploinsufficiency of A20 (HA20), suggests a new category of human inflammatory diseases caused by impaired ubiquitination, according to the researchers. source: http://www.dddmag.com/news/2016/08/nih-researchers-discover-new-inflammatory-disease Date: 08/23/2016 Share this post:EmailPrintTwitterLinkedInFacebookGoogleLike this:Like Loading... Related
Nutrinia Raises $30M to Continue Development of Specialized Oral Insulin
Nutrinia Raises $30M to Continue Development of Specialized Oral Insulin Article taken from: http://www.dddmag.com/news/2016/08/nutrinia-raises-30m-continue-development-specialized-oral-insulin Picture taken from: http://www.enerjouvence.com/bukos.jpg Nutrinia, a company developing a proprietary oral formulation of insulin for intestinal malabsorption in preterm newborns and Short Bowel Syndrome (SBS) in infants, today announced the closing of a $30 million Series D financing to fund two pivotal trials for registration. TPG Biotech, the life science venture investment arm of leading global alternative asset firm TPG, led the investment, joined by H.I.G. BioHealth Partners and WuXi Healthcare Ventures, as well as existing investors including OrbiMed, Pontifax and others. Nutrinia will use the proceeds to initiate two pivotal trials for registration in separate indications related to acceleration of gut maturation and adaptation: intestinal malabsorption in preterm newborns born between 26 and 32 weeks’ gestational age, and infants with SBS who are under 12 months old. “I’m happy to welcome TPG Biotech, H.I.G. BioHealth Partners and WuXi Healthcare Ventures into Nutrinia at this exciting time for the company,” said Miki Olshansky, Nutrinia’s Chief Executive Officer. “We are beginning two trials in the US and Europe that will provide robust data as to the safety and efficacy of our proprietary oral formulation of insulin, and if successful, provide a direct path to registration. These are orphan indications that affect fragile populations with high levels of unmet medical need.” “The biological rationale for Nutrinia’s product is strong and the company’s preclinical data and outcomes of multiple previous clinical trials are compelling,” said Eran Nadav, Ph.D., Partner and Managing Director at TPG Biotech. “We believe that neonatology, and specifically the NICU, are areas that deserve particular therapeutic development focus, and we are pleased to support Nutrinia’s efforts.” With this investment, Dr. Nadav will join Nutrinia’s Board of Directors, as will Aaron Davidson, Co-head and Managing Director of H.I.G. BioHealth Partners. Source: Article taken from: http://www.dddmag.com/news/2016/08/nutrinia-raises-30m-continue-development-specialized-oral-insulin
Music Found to Boost Quality of Life for Cancer Patients
Music Found to Boost Quality of Life for Cancer Patients poArticle taken from: http://www.biosciencetechnology.com/news/2016/08/music-found-boost-quality-life-cancer-patients Picture taken from: http://www.biosciencetechnology.com/news/2016/08/music-found-boost-quality-life-cancer-patients Hearing a favorite song can put a smile your face, but a new study demonstrates that music also help alleviate certain symptoms for cancer patients, such as anxiety, pain and fatigue. Researchers led by Joke Bradt, Ph.D., associate professor in Drexel University’s college of Nursing and Health Professions, conducted a systematic review of 52 trials that observed how two types of music interventions effect psychological and physical outcomes in cancer patients. There were 3,731 participants included in the studies. Of the two music interventions reviewed in the studies, 23 were classified music therapy, which is where a trained music therapist crafts a personalized music experience, and 29 were classified as music medicine, which is where a doctor or nurse provides the patient with pre-recorded music to listen to. The greatest benefit was reducing anxiety, where the researchers found that all kinds of music interventions resulted in a moderate-to-strong effect. They also found a large benefit in pain reduction, and a small-to-moderate treatment effect for fatigue. The study also tied music interventions to lowered blood pressure and small reductions in heart and respiratory rates. Only music therapy interventions were linked to a moderate increase in quality of life, but Bradt said it is still unclear which interventions might be best for certain outcomes. “The results of single studies suggest that music listening may reduce the need for anesthetics and analgesics, as well as decreased recovery time and duration of hospitalization, but more research is needed for these outcomes,” the authors wrote. The team hopes that the findings will help make music interventions more widely used in medical settings for people with cancer. The findings were published by the Cochrane Library. Source: http://www.biosciencetechnology.com/news/2016/08/music-found-boost-quality-life-cancer-patients
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Physicians, management teams, investigators, and clinical coordinators are fluent in English.
With over four million patients being seen by our hospitals each year and with relatively little to no clinical research exposure, our network is ideal for medical trials.