2016-08-12There is a growing consensus that drug discovery thrives in an open environment. Here, we describe how the malaria community has embraced four levels of open data — open science, open innovation, open access and open source — to catalyse the development of new medicines,
2016-09-29A wave of companies are developing personalized vaccines, built from tumour-specific neo-antigens, providing a timely boost for a failure-ridden area of cancer immunotherapy.
2016-09-29A recent FDA workshop brought new diabetes outcome measures to the table, focusing on patient-oriented end points.
2016-09-29Merck & Co. has discontinued development of its cathepsin K inhibitor odanacatib, citing an increased risk of cardiovascular events for the osteoporosis drug. The decision was an expensive setback for the company: the drug spent 12 years in clinical development and its pivotal trial enrolled
2016-09-29Between 1990 and 2012, the proportion of new cardiovascular drugs entering into clinical trials declined across all stages of clinical drug development, shows a recent pipeline analysis (JACC Basic Transl Sci.1, 301–308; 2016).The analysis, by academic researchers from Harvard University,
2016-09-29A new global vaccine development fund called the Coalition for Epidemic Preparedness Innovations (CEPI) aims to stimulate, facilitate and finance the development of vaccines against infectious disease epidemics.The CEPI public–private partnership is currently backed by the Bill & Melinda Gates Foundation, the Wellcome Trust,
2016-09-29One of the primary mechanisms used by the FDA to protect human subjects participating in clinical trials is the requirement for mandatory safety reporting. Under the Code of Federal Regulations (21 CFR §312.32), sponsors of clinical trials conducted under an investigational new drug application (IND)
2016-09-29Potential important market catalysts in the fourth quarter of 2016 include an FDA approval decision for Heplisav (developed by Dynavax and Pfizer) for the immunization of adults against hepatitis B infection. Additionally, an FDA approval decision for telotristat etiprate (developed by Lexicon Pharmaceuticals) for neuroendocrine
2016-09-29Although biopharma start-up companies have to be laser-focused on translational science, this single-mindedness sometimes comes at the expense of clinical development expertise. The venture capital group Flagship Ventures has now named Michael Rosenblatt as Chief Medical Officer to help its portfolio of companies fill that experience gap. Rosenblatt was Chief Medical Officer at Merck & Co. for 7 years prior to this role, and Dean of Tufts University School of Medicine before that. He talks with Asher Mullard about his new job, and his novel proposal for how to handle the reproducibility crisis.
2016-09-12Recent investments in nascent public biopharmaceutical companies indicate that companies focused on small-molecule approaches attracted more funding than might have been expected based on rates of return on investment compared with biologic approaches.
2016-09-29Although there are currently no major outbreaks of Ebola, new ones are likely to emerge in the coming years. Ideally, treatments will be strain-adaptable. A new paper in Nature Microbiology demonstrates that a Sudan ebolavirus (SUDV)-targeting small interfering RNA (siRNA) can prevent death
2016-09-29Chagas disease, leishmaniasis and sleeping sickness are caused by infection with kinetoplastid parasites and are prevalent among the world's poorest populations. Although treatment options for these diseases exist, their use is limited by several factors, including toxicity, poor efficacy, difficult administration and cost. Now, writing
2016-09-29Immunotherapeutic anticancer strategies that facilitate the activation of immune effector cells (such as immune checkpoint inhibitors) or that inhibit immunosuppressive cells have achieved impressive results, but can also induce potentially fatal autoimmune side effects. Reporting in Science Translational Medicine, Kobayashi and colleagues now demonstrate
2016-09-29Activation of the μ-opioid receptor by morphine and related opioids provides valuable analgesia but can also lead to addiction and potentially lethal side effects such as respiratory depression. Now, an article in Nature reports a structure-based approach to the discovery of novel 'biased' ligands
2016-09-29Inflammation of the skin can be triggered by lipids such as urushiol, which is found in various plants, including poison ivy. Now, the surface molecule CD1a has been found to have a role in inflammation by presenting urushiol and other lipid antigens to T cells.
2016-09-29The Hippo signalling pathway has a pivotal role in regulating stem cell renewal, tissue regeneration and organ size control. Fan et al. now describe the use of an enzyme-linked immunosorbent assay (ELISA)-based high-throughput screen of a kinase-directed compound library, to identify the benzenesulfonamide-based small
2016-09-29The recombinant variant of activated protein C (APC), 3K3A-APC (in which three Lys residues (KKK191–193) are replaced with alanine), exhibits reduced anticoagulant activity and is being clinically investigated for the treatment of ischaemic stroke. Recent in vitro studies have shown that 3K3A-APC also stimulates
2016-09-29Mechanical ventilation of critically ill patients can result in ventilation-induced diaphragm dysfunction. Here, using a rat intensive care unit model of mechanical ventilation, Salah et al. show that such negative effects on diaphragm muscle are caused by impaired myosin function, which is related to
2016-09-29Decreased expression of CCAAT/enhancer binding protein-α (C/EBPα) frequently occurs in non-small-cell lung cancer (NSCLC) and has been implicated as a key tumourigenic event. Here, Yong et al. analysed 261 primary lung cancer patient specimens and noted that 80% of those with low or no
2016-08-12Over the past 25 years, biophysical technologies such as X-ray crystallography, nuclear magnetic resonance spectroscopy, surface plasmon resonance spectroscopy and isothermal titration calorimetry have become key components of drug discovery platforms in many pharmaceutical companies and academic laboratories. There have been great improvements in the
2016-07-22Castration-resistant prostate cancer (CRPC) is associated with a poor prognosis and poses considerable therapeutic challenges. Recent genetic and technological advances have provided insights into prostate cancer biology and have enabled the identification of novel drug targets and potent molecularly targeted therapeutics for this disease. In
2016-09-12Exercise reduces the risk of a multitude of disorders, from metabolic disease to cancer, but the molecular mechanisms mediating the protective effects of exercise are not completely understood. The realization that skeletal muscle is an endocrine organ capable of secreting proteins termed 'myokines', which participate