Subtyping lessons in brain cancer

A team at St. Jude Children's Research Hospital believes a screening approach based on disease subtypes will be important for identifying therapeutics for brain cancer patients. As a proof of principle, the researchers conducted a small molecule screen in a neural stem cell model of one subtype of ependymoma, a rare brain cancer.¹

On the basis of the data, the investigators plan to start a Phase I trial of 5-fluorouracil (5-FU) in pediatric ependymoma this year. The Phase I trial will be open to all patients, with stratification for subsequent trials to be determined on the basis of the Phase I trial results and preclinical testing of 5-FU in other ependymoma subtypes as more models become available...