Drug Discovery Research for Hydrocephalus

In 2013, clinical and basic science research has rolled the dice further to extend our understanding to hydrocephalus. A group of research team at Boston Children’s Hospital and Harvard Medical School, led by neurosurgeon, Joseph Madsen, has published the laboratory investigation results on vascular endothelial growth factor, VEGF, and showed its potential effect on the development of experimental ventriculomegaly in an animal model [1]. Ventriculomegaly, the dilatation of the lateral ventricles is one of the diagnostic criteria for hydrocephalus. Hydrocephalus, water in the head problem, is a neurological condition characterized by accumulation of fluid in the cerebral ventricles. The current treatment is either ventriculostomy or shunt surgery to drain excessive fluid. However, shunt surgery often requires additional intervention due to functional failure. Concern over pediatric patients with hydrocephalus managed successfully by shunt, say, for 10 years without revision, is the fact that anatomy surrounding the shunt changes with growth, while shunt is not designed so. Adaptation to altered anatomy can affect physiology and shunt function. The risk for young patients is the shunt malfunction and resulting complications. For this reason, a nonsurgical approach that may lessen this difficulty in the future is necessary. Pharmacological approach alone, however, can also potentiate an adverse effect. How we manage this condition with currently available surgical and potential non-surgical approach might be, therefore, the key to success. In doing so, drug discovery and testing candidate molecules in animal models for achieving this purpose can enhance the quality of clinical decision-making at the end.