Investigational drugs for ischemic stroke: what's in the clinical development pipeline for acute phase and prevention?

Introduction Stroke is a leading cause of disability and mortality and its burden expected to increase. The only approved drug for acute ischemic stroke (IS) is the intravenous thrombolytic alteplase. The risk of bleeding complications is one of the reasons for the undertreatment of eligible patients. Numerous drugs are currently being developed to improve safety-efficacy. Areas covered We reviewed literature from 1 January 2000, to 15 January 2022 for the development and testing of novel drugs with the aim of targeting treatment at prevention of ischemic stroke: PubMed, MEDLINE, Google Scholar, and ClinicalTrial.gov. Expert opinion The pathophysiology of IS involves multiple pathways causing cerebral artery obstruction and brain tissue ischemia. Data suggest that tenecteplase is a promising fibrinolytic agent with a superior efficacy-safety profile, compared to alteplase. Current guidelines consider a short-term cycle of mannitol or hypertonic saline to be advisable in patients with space-occupying hemispheric infarction. Regarding primary and secondary prevention, research is primarily focused on identifying mechanisms to improve the safety-efficacy profile using a 'hemostasis-sparing' approach. Further evaluation on those agents that have shown promise for their risk/benefit profiles, would benefit greatly a neurologist's capacity to successfully prevent and treat IS patients.

Automated summary

This article discusses the development of new drugs for the treatment and prevention of stroke. Researchers have found that tenecteplase is a more effective and safer fibrinolytic agent compared to the currently approved alteplase. Additionally, the use of mannitol or hypertonic saline is recommended for patients with space-occupying hemispheric infarction. Future research should focus on improving the safety and efficacy of treatment and further evaluating promising drugs.