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A recent landmark analysis compared genome-wide mutations and associated 'omics' features for 3,281 tumours across 12 human cancer types. There is a clear need for objective target assessment and prioritization by the drug discovery community to make the best use of such vast amounts of data.
The expedited US regulatory pathway for 'breakthrough therapies' has already yielded its first two approvals and more than 26 designations, for 30 candidates in 22 indications.
Japan's biggest pharmaceutical companies, the Bill and Melinda Gates Foundation, and the Japanese government have awarded their first round of global health research grants.
Charles Hugh-Jones, Chief Medical Officer in North America at Sanofi, discusses Project Data Sphere, a new collaborative platform for sharing cancer clinical trial data.
This analysis of factors that affect the likelihood of success in drug research and development (R&D) indicates that scientific acumen and good judgment — particularly with regard to the early termination of less viable drug candidates — are crucial.
Regulatory agencies have been criticized both for being overly tolerant of risks or being excessively risk-averse, but the potential for adverse effects on public health owing to the absence of new drugs because of regulatory risk-aversion is less apparent. Here, Eichler and colleagues discuss the consequences of regulatory risk-aversion and suggest what might be done to best align acceptance of risk and uncertainty by regulators with the interests of public health.
Specific patterns of post-translational modifications in chromatin structure are important factors in the regulation of gene expression, and when deregulated they can contribute to diseases including cancer and neurological disorders. Here, Helin and colleagues focus on the role of one class of chromatin-modifier enzymes — the histone lysine demethylases — highlighting their links to cancer and their potential to be therapeutically targeted.
Cancer cells have high levels of reactive oxygen species (ROS) owing to metabolic and genetic alterations. The role of ROS in cancer cells is controversial as they can have both pro-tumorigenic and antitumorigenic properties. In this Review, Mak and colleagues discuss recent findings that cancer cells upregulate antioxidant pathways to counteract ROS, and explore the potential of anticancer strategies that target the antioxidant capacity of tumour cells.
The 'quality' of small-molecule drug candidates — encompassing aspects including their potency, selectivity and pharmacokinetic characteristics — is a key factor influencing the chances of success in clinical trials. Cumming and colleagues discuss the application of computational methods, particularly quantitative structure–activity relationships, in guiding the selection of higher-quality drug candidates, as well as cultural factors that may have affected their impact.
