Predictive Biomarkers and Targeted Therapies in Adult Brain Cancers

Abstract

Diffuse glial neoplasms in adults remain incurable, with high morbidity and mortality, despite significant advances in our understanding of their molecular characteristics and the underlying alterations in specific signaling pathways. With rare exceptions, and unlike the successes in treatment of other solid tumors, successful application of targeted therapies for glial neoplasms has remained elusive. Mutations in the IDH1/2 genes are factors in the neoplastic transformation of a large proportion of intermediate and high-grade gliomas, and a variety of treatment approaches including the development of vaccines and molecular inhibitors are currently being evaluated. Epigenetic factors such as DNA methylation also play an important role in the response to specific therapies. The accurate screening of predictive biomarkers in the diagnosis of gliomas and other tumors of the nervous system is now needed to allow selection of the most appropriate therapeutic approaches based on the molecular characteristics of the tumor.