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Sánchez-Serrano discusses the story of the innovative anticancer drug bortezomib to dissect the key public-sector-private sector interactions that made the development of this drug successful despite many barriers, and considers the implications for improving translational research in general.
Although the prospects of gene therapy have not been fully realised, there is hope that nanotechnologies will have great impact on this field. Crommelin and colleagues describe such approaches to gene therapy. Nucleic acid delivery systems are being developed that incorporate virus-like functions in a single nanoparticle.
Pegaptanib, an RNA aptamer that binds to vascular endothelial growth factor, was approved for the treatment of age-related macular degeneration in December 2004, becoming both the first approved aptamer therapeutic and the first anti-angiogenic agent for treating ocular disease.
Schizophrenia trials are subject to multiple endpoint comparison problems, the risk of false-positives, patient non-compliance, high drop-out rates and missing data. Stroup and colleagues discuss current trial limitations and how these might be addressed by innovations in trial design and statistics.
Targeting drugs specifically to tumour cells to improve efficacy and reduce toxicity is paramount to the future development of anti-cancer agents. Schrama and colleagues critically review current approaches to achieving this and the status of targeted treatments in development.
Excessive glutamate receptor activity, principally of theN-methyl-D-aspartate (NMDA) subtype, contributes to neuronal damage in a large number of neurologic disorders, including dementia. Until recently, however, NMDA receptor antagonists had all failed in clinical trials. Stuart Lipton reviews the mechanism of action that led to the clinical approval of the first NMDA receptor antagonist, memantine, which has become the newest and one of the best-selling drugs for Alzheimer's disease.
