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Fergus Sweeney, Head of Compliance and Inspection Sector at the European Medicines Agency, discusses the strengths and weaknesses of the new EU Clinical Trials Register.
Regulation on orphan medicinal products was adopted in the European Union in 2000, with the aim of benefiting patients who suffer from serious, rare conditions for which there is currently no satisfactory treatment. This article highlights the outcomes of this regulation, such as the approval of more than 60 orphan drugs, reflects on the experience gained so far and discusses key issues for the next 10 years.
Synthetic lethality screening involves searching for genetic interactions of two mutations in which the presence of either mutation alone has no effect on cell viability, but the combination of the two mutations results in cell death. The presence of one of these mutations in cancer cells but not in normal cells could allow cancer cells to be selectively killed by mimicking the effect of the second of the two mutations with a targeted drug. Chan and Giaccia review strategies to identify synthetic lethal interactions and present case studies of anticancer agents that act by inducing synthetic lethality.
Current atherosclerosis therapies largely act by lowering lipid levels and although they are effective, patients are still at risk for major adverse cardiovascular events (MACE). It has recently emerged that inflammation has a major role in the development of MACE and this is not addressed by existing agents. Here, Charo and Taub discuss key anti-inflammatory targets and associated therapeutics that are now being developed to treat atherosclerosis.
Neuroprotective and/or disease-modifying treatments are urgently needed for Parkinson's disease. This Review describes how an increased understanding of genetic mutations that lead to Parkinson's disease, better translation between preclinical animal models and clinical research, and improved design of future clinical trials are priorities for overcoming the limitations of current therapies.
