Nature Reviews Drug Discovery

Poor medication adherence in clinical trials: consequences and solutions ()
Poor adherence to medicines in clinical trials can undermine the value of the trials; for example, by compromising estimates of the benefits and risks of a medicine. In this article, we highlight such consequences and also discuss approaches to tackle this problem.
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Out-licensing role reversal ()
An innovative alliance structure is the latest advance in pharma's externalization push.
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Bracing for the biosimilar wave ()
The regulatory uncertainty around biosimilars in the United States is finally lifting, just months after the first biosimilar monoclonal antibody hit the US market.
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BACE inhibitor bust in Alzheimer trial ()
Merck & Co. has halted a pivotal trial of its β-secretase 1 (BACE1) inhibitor verubecestat in mild-to-moderate Alzheimer disease (AD) due to futility, providing a first critical failure for yet another class of amyloid-modulating drugs.Trials have already sunk other classes aimed at controlling amyloid
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New plaque psoriasis approval carries suicide warning ()
The FDA approved Valeant's brodalumab for the treatment of moderate-to-severe plaque psoriasis, but with the warning that the drug is associated with suicidal ideation and behaviour.The antibody targets the interleukin-17 (IL-17) pathway, fertile ground for drug developers. Although the drug follows on
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Biotech gender gap ()
Women hold only around 10% of the board positions in biotech firms, found a recent report on the biotech gender gap by Liftstream, a life sciences recruitment company.This finding is based on an analysis of the boards of 177 biotech firms that filed
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Regulatory watch: The target product profile as a tool for regulatory communication: advantageous but underused ()
A key objective in interactions between the pharmaceutical industry and regulatory authorities is to achieve clarity on the goals and expectations for the drug development process. In the United States, the target product profile (TPP) is a tool to facilitate communication between the pharmaceutical industry
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Trial watch: Clinical trial cycle times continue to increase despite industry efforts ()
One key issue facing pharmaceutical clinical development organizations has been increasing clinical trial cycle times. Despite substantial effort and attention from the industry on this issue, overall development timelines continue to increase, at both the programme and study levels. Indeed, cycle time continues to be
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Melinda Richter ()
When Melinda Richter started her career with the large telecommunications company Nortel, her focus was on, among other things, figuring out how to buy pop drinks from vending machines with cell phones. But after developing a severe case of meningitis while working in China — and realizing how much unmet medical need there was there — she turned her business acumen towards fostering the creation of life science start-ups that could develop new drugs. To this end, she joined up with Johnson & Johnson in 2012 to launch a network of incubators. For Richter, head of JLABS at J&J Innovation, this offered a clear win-win strategy. Start-ups benefit from no strings attached access to J&J expertise, while J&J gains revitalizing exposure to emerging science and entrepreneurial enthusiasm. Five years on, JLABS has just announced plans to launch its ninth incubator, in New York City. Richter spoke with Asher Mullard about the network's success to date and her plans for its future.
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Biopharma deal-making in 2016 ()
This article analyses merger and acquisition (M&A) activity and partnerships in the biopharma industry in 2016, highlighting trends and major deals.
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Antifungals: JNK inhibitors boost antifungal immunity ()
Invasive fungal infections, particularly with Candida albicans, kill approximately 1.5 million people worldwide a year. Current drugs are relatively toxic and fungal resistance is high, so there is an urgent need for new therapeutics. Now, reporting in Nature Medicine, Zhao, Lin and colleagues
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Ischaemic disorders: Safe path to thrombosis prevention ()
Existing oral antiplatelet medications effectively reduce atherothrombotic events in patients at high risk, but the use of such agents has been associated with a risk of life-threatening bleeding. Now, writing in Science Translational Medicine, Wong et al. report the identification of an antagonist
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Bone diseases: MST1R inhibitor prevents bone osteolysis ()
Bone osteolysis occurs in patients with metastatic cancer or osteoporosis and can lead to an increased risk of fracture as well as pain. Now, Andrade et al. have found that inhibiting macrophage-stimulating protein receptor (MST1R; also known as RON) blocks bone destruction in mice
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Anticancer agents: Allosteric histone methyltransferase modulators block tumour growth ()
Polycomb repressive complex 2 (PRC2), which regulates gene expression through its histone H3 lysine 27 trimethylation methyltransferase (H3K27me3) activity, is dysregulated in multiple human cancers and has attracted considerable interest as a therapeutic target. Two new studies, reported in Nature Chemical Biology, now present
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Cancer: CD99 marks malignant myeloid stem cells ()
Self-renewing cancer stem cells drive disease in acute myeloid leukaemia (AML) and myelodysplastic syndromes (MDS), but efforts to selectively target these cells have been hampered by a lack of specific markers. Now, Chung and colleagues have identified CD99 as a cell surface marker of disease
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Alzheimer disease: Antisense oligonucleotide reverses tau pathology ()
Cognitive decline in Alzheimer disease closely correlates with deposition of tau protein. DeVos et al. have designed antisense oligonucleotides (ASOs) that are specific for human tau. These ASOs inhibited loss of hippocampal volume and neurons, reversed tau seeding activity, prolonged survival and rescued nesting
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Ocular disorders: Collagen IV-derived peptide prevents angiogenesis ()
Vascular endothelial growth factor (VEGF)-neutralizing proteins can inhibit the pathological angiogenesis and vascular leakage that occur in many retinal and choroidal vascular diseases. However, these therapies require frequent intraocular injections, and outcomes are sometimes suboptimal. Here, Lima e Silva et al. report that a
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Anticancer agents: Engineered platelets deliver immunotherapy ()
Immunotherapeutic strategies, such as anti-programmed cell death protein (PD1) therapy, can prevent post-surgical cancer recurrence, but their use can be limited by off-target effects. Given that platelets migrate to surgical sites and can interact with circulating tumour cells (CTCs), Wang et al. intravenously administered
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Atherosclerosis: Haematopoietic mutation promotes atherogenesis ()
Somatic mutations in the gene encoding the epigenetic modifier enzyme ten-eleven translocation 2 (TET2) in haematopoietic cells promotes clonal haematopoiesis, which has been associated with atherosclerosis. Fuster et al. generated atherosclerosis-prone, low-density lipoprotein receptor-deficient mice in which bone marrow was reconstituted with Tet2-deficient
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Non-coding RNAs as drug targets ()
Most of the human genome encodes RNAs that do not code for proteins. These non-coding RNAs (ncRNAs) may affect normal gene expression and disease progression, making them a new class of targets for drug discovery. Because their mechanisms of action are often novel, developing drugs
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Aptamers as targeted therapeutics: current potential and challenges ()
Nucleic acid aptamers, often termed 'chemical antibodies', are functionally comparable to traditional antibodies, but offer several advantages, including their relatively small physical size, flexible structure, quick chemical production, versatile chemical modification, high stability and lack of immunogenicity. In addition, many aptamers are internalized upon binding
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MicroRNA therapeutics: towards a new era for the management of cancer and other diseases ()
In just over two decades since the discovery of the first microRNA (miRNA), the field of miRNA biology has expanded considerably. Insights into the roles of miRNAs in development and disease, particularly in cancer, have made miRNAs attractive tools and targets for novel therapeutic approaches.
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