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Despite a decade of intensive preclinical research, the translation of cancer nanomedicine to the clinic has been slow. Here, we discuss how recent lessons learned from the successes with immuno-oncology therapies could be applied to cancer nanomedicine and how this may help to overcome some of the key technical challenges in this field.
In the search for the first disease-modifying therapy for Parkinson disease, drug developers are advancing α-synuclein-targeted agents into proof-of-concept clinical trials.
This article presents the findings of a recent analysis of the costs of clinical trials, providing benchmark data for companies to assess their performance, as well as indicating cost drivers.
Genome editing has emerged as an attractive approach to therapeutically manipulate gene expression. Here, Anderson and colleagues provide an overview of genome-editing platforms, focusing on the methods and challenges of intracellular biomacromolecule delivery. Preclinical and clinical trials involving genome-editing technologies are also discussed.
Dysregulation of iron homeostasis occurs in haematological disorders and in other diseases such as cancer and neurodegeneration. Crielaard and colleagues discuss the progress made in interfering with iron metabolism as a therapeutic strategy, as well as in using iron metabolism to direct drugs to target tissues.
Inhibition of proteins other than the intended target by small molecules can lead to the incorrect assignment of biological functions to particular proteins and wasted drug development efforts. Potential inhibition of off-target kinases by kinase inhibitors is often investigated, but kinase inhibitors can also inhibit non-kinases. Munoz examines the growing number of examples of this issue and suggests a systematic strategy to verify which protein is responsible for the effects of a given small molecule.
