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This article analyses the characteristics of the pipeline for experimental drugs for central nervous system disorders and how they have fared in the clinical phases prior to FDA approval over the past two decades.
Technological advances coupled with novel collaborative strategies for compound sourcing and management are poised to transform the utility of high-throughput screening.
A new model for translational research and drug repositioning has recently been established based on three-way partnerships between public funders, the pharmaceutical industry and academic investigators. This article discusses the progress with two pioneering initiatives — one involving the UK Medical Research Council and one involving the US National Center for Advancing Translational Sciences — and the unique requirements and challenges for this model.
As critical regulators of glucose and lipid metabolism, as well as vitamin D and phosphate homeostasis, the endocrine fibroblast growth factors (FGFs) have therapeutic potential in various chronic human diseases. Here, Moschetta and colleagues discuss the physiological roles of FGF19, FGF21 and FGF23, focusing on recent advances and the associated challenges in their therapeutic exploitation.
Several Phase I trials evaluating systemically administered siRNA-based therapeutics for cancer have recently been completed. Here, Zuckerman and Davis critically assess these studies and discuss key lessons learnt and implications for the future development of siRNA-based therapeutics and clinical trial design.
Dysfunction of voltage-gated calcium channels is associated with a variety of neurological and psychiatric conditions. Several drugs that block these channels are already in clinical use, with new agents currently in development. Here, Zamponi highlights the functions of voltage-gated calcium channels and assesses their potential as therapeutic targets for nervous system disorders.