Emerging Molecular Targets and Personalized Therapeutics in CNS Tumors

Abstract

This is a review of the current preclinical data and early-phase clinical trials evaluating the use of personalized on-treatment molecular imaging in central nervous system tumors (CNS). Epigenetic modulators targeting IDH1-mutated, histone H3-mutated, or MGMT-methylated gliomas and “hot spot” mutations could increase the effectiveness of ICIs in glioma patients. Theranostic radioligands targeting somatostatin and prostate-specific membrane antigen could be of benefit in neuroendocrine CNS tumors and primary brain malignancies. In the presence of specific molecular features, these radioligands could be used for theranostic purposes. Recognizing IDH1-R132H mutation on brain cytopathological specimens allows for a tailored patient care approach by providing real-time diagnostics even if standard mutational analysis is uninformative. Including immunohistochemical testing in the routine intraoperative examination of benign oncological procedures for lesion excision would prevent multiple repeat surgeries in glioma patients and improve patient outcomes.