Preclinical studies demonstrated that glioma cell lines harboring the H3 K27M mutation were more susceptible to ONC201 cytotoxicity than wild-type counterparts, and that this increased sensitivity was coincident with elevated DRD2 expression.21 Based on these clinical and preclinical findings, a series of clinical investigations were initiated to evaluate ONC201 in patients with H3 K27M-mutated disease. The gene discussed is DRD2; the disease is central nervous system cancer.