Treatment Approach Using IDH-Inhibitors

UCSF neuro-oncologist Jennie Taylor, MD, MPH, explains that the mutated form of IDH produces an harmful byproduct, or oncometabolite, called 2-hydroxyglutarate (2-HG). IDH inhibitors like vorasidenib block the mutant IDH protein, thereby reducing the amount of 2-HG in the tumor. 

Taylor also outlines the clinical data that formed the basis for the U.S. Food and Drug Administration's approval of vorasidenib as a new brain tumor treatment. A phase 3 clinical trial showed that vorasidenib reduced the risk of disease progression in patients with grade 2 IDH-mutant gliomas who were within one to five years of having had surgery to remove their tumor. The study participants who received vorasidenib also had a lower risk of needing another treatment invention compared to those who received the placebo.