Search

International audience; Paediatric CNS tumours are the most common cause of childhood cancer-related morbidity and mortality, and improvements in their diagnosis and treatment are needed. New genetic and epigenetic information about paediatric CNS tumours is transforming the field dramatically. For most paediatric CNS tumour entities, subgroups with distinct biological characteristics have been identified, and these characteristics are increasingly used to facilitate accurate diagnoses and therapeutic recommendations. Future treatments will be further tailored to specific molecular subtypes of disease, specific tumour predisposition syndromes, and other biological criteria. Successful biomaterial collection is a key requirement for the application of contemporary methodologies for the validation of candidate prognostic factors, the discovery of new biomarkers, the establishment of appropriate preclinical research models for targeted agents, a quicker clinical implementation of precision medicine, and for other therapeutic uses (eg, for immunotherapies). However, deficits in organisational structures and interdisciplinary cooperation are impeding the collection of high-quality biomaterial from CNS tumours in most centres. Practical, legal, and ethical guidelines for consent, storage, material transfer, biobanking, data sharing, and funding should be established by research consortia and local institutions to allow optimal collection of primary and subsequent tumour tissue, body fluids, and normal tissue. Procedures for the collection and storage of biomaterials and related data should be implemented according to the individual and organisational structures of the local institutions.

Stroke presents a major global burden to patients, their relatives, and whole economies.1 Worldwide intense efforts are being undertaken to understand the pathobiology of stroke and to develop novel, effective treatments. This has led to the discovery of a plethora of preclinically promising treatment strategies,2 but intravenous thrombolysis remains the only specific pharmacological treatment with proven efficacy in acute ischemic stroke.3 Apparently, of several hundred controlled clinical trials4 aiming to protect the brain against ischemia (neuroprotection) with compounds previously found effective in animal experiments, not a single one has led to the regulatory approval of a drug for this indication. Many pharmaceutical companies have, therefore, stopped developing treatments for this devastating disease, and nihilism is spreading in the field. A roadblock seems to exist between bench and bedside, preventing the translation of highly effective preclinical neuroprotectants into the clinical realm.5 Despite the prevailing mantra of translational medicine,6 other fields report similar problems when making the preclinical-to-clinical transition. Much has been speculated about the reasons underlying the translational roadblock, and the phrase lost in translation has entered the titles of numerous biomedical publications. In this article, we propose that contrary to current opinion, preclinical stroke research can indeed predict clinical outcomes and phenotypes. Numerous neuroprotectants were reported as lost in translation (Figure 1A), but there is reason to argue that the clinical trials were based on rather weak preclinical evidence, and that many pathophysiological principles and therapies were actually discovered in translation (Figure 1B). We propose that there is little reason for nihilism. We present a concise list of potential improvements that may help to reduce the current attrition rate of bench-to-bedside translation, even in neuroprotection. Stroke may even serve as a model disease because our analysis may apply to other fields where development of …