Abstract

Development of drugs for treating diseases of the central nervous system (CNS) has had some of the highest attrition rates in the pharmaceutical industry, and the high risk and cost of development has led to many companies withdrawing from the area, despite significant unmet medical needs remaining and, hence, large commercial potential. For many CNS diseases, particularly psychiatric diseases, our understanding of the fundamental neuroscience associated with the disease is limited, which has led to a lack of predictivity of animal models and, therefore, often a failure of translation from animal models into human patients. Fortunately, technologies such as genetics and functional imaging are rapidly advancing our understanding of the human brain. Historically, many drugs used to treat CNS disorders were discovered by serendipity, when activity was noticed in the clinic during use for another indication. Such clinical ‘repurposing’ of drugs is still a highly useful strategy, but requires the availability of selective and potent tool compounds, with an accompanying regulatory data package, that will allow their use in people. Many such compounds exist within pharmaceutical companies but are sitting on the shelf because their development has been terminated. The Medicines Chest initiative was set up, funded by the European College of Neuropsychopharmacology (ECNP), in order to gain access to such compounds, so they could be used by academics and small companies in experimental medicine studies. The purpose of this article is to provide an update on progress with this initiative.

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