Towards Improved Translational Stroke Research: Progress and Perspectives of the Recent National Institute of Neurological Disorders and Stroke Consensus Group Meeting

Preclinical stroke research faces a substantial transition. Past classical rodent stroke models and study designs revealed numerous potential targets for novel stroke therapies; yet, subsequent clinical stroke trials failed to confirm promising preclinical findings. Pharmacological and mechanical recanalization therapies, representing the only strategies that have substantially improved acute ischemic stroke outcomes, were largely developed omitting conventional preclinical methods.1–3 In this issue of Stroke, a National Institute of Neurological Disorders and Stroke consensus group comprising leading academic, industry, and Food and Drug Administration (FDA) experts working at the forefront of stroke research has recently published guidelines for improved translational studies.4

Distinguishing between explorative (basic) versus confirmative (translational) studies has been suggested. Exploratory research uses simpler rodent models but a broad spectrum of basic science methods to gain comprehensive information on a putative treatment target. Subsequent confirmative research adopts study designs similar to those used in clinical stroke trials and puts more emphasis on predictive stroke models and study end points using larger and adequate sample sizes for necessary statistical power.5 The group’s recommendations reflect latest developments and concepts in the field, aiming to ultimately enhance the predictive value of preclinical stroke research.