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Rare disease drug development could benefit substantially from increased patient engagement and input to enhance understanding of the key aspects of disease impact, ways to measure these impacts and patients' perspectives on the benefit–risk profile of potential therapies.
Clinical trial failures of two drugs for hearing loss and tinnitus underscore pitfalls for a nascent area of drug development, but lessons learned may help in navigating the uncharted path to approval.
Although Agios and Celgene are set to file for approval for their first-in-class cancer metabolism drug, the field has started looking in new directions for the next batch of metabolic targets.
Deborah Zarin, director ofClinicalTrials.gov, discusses the new trial registration rules and the need for transparency in the clinical research enterprise.
Nonalcoholic steatohepatitis (NASH), an extreme form of nonalcholic fatty liver disease, is predicted to become the leading reason for liver transplantation by 2020. This analysis provides an overview of emerging therapies for NASH.
Traditional cell-based disease models often fail to adequately represent key disease characteristics, increasing the risk of subsequent attrition in clinical trials. This article presents a set of principles for disease-relevant assays, and discusses new opportunities for exploiting advances in cell-based assay technologies in drug discovery, including induced pluripotent stem cells as well as 3D co-culture and organ-on-a-chip systems, which are being complemented by progress with single-cell imaging and gene editing technologies.
KRAS is one of the most frequently activated proteins in cancer, yet the development of RAS inhibitors has proven to be extremely challenging. Here, Shokat and Ostrem discuss the latest insights into RAS structure and dynamics, consider potential mechanisms of action for effective RAS inhibitors, and examine recent reports of direct RAS inhibitors.
Although the liver has a key role in maintaining blood glucose homeostasis, few existing type 2 diabetes therapies directly target this organ. Here, Puigserveret al. provide an overview of the molecular mechanisms controlling hepatic gluconeogenesis and glycogen storage, focusing on emerging strategies to target hepatic glucose metabolism for the treatment of diabetes.
