A groundbreaking study co-led by UCLA has shown that the targeted therapy drug vorasidenib significantly improves outcomes for patients with recurrent grade 2 glioma, a slow-growing but deadly brain cancer. This international trial, the first to focus on a targeted therapy for brain tumors, involved 331 patients and demonstrated that vorasidenib more than doubled the progression-free survival time compared to a placebo—extending it to an average of 27.7 months versus 11.1 months. Patients taking vorasidenib experienced almost 17 months before requiring chemotherapy or radiation, providing a critical delay in treatment that could help preserve neurological function. The dual inhibitor targets mutant IDH1/2 enzymes, reducing the accumulation of an onco-metabolite believed responsible for glioma development. The study found limited adverse effects, with only 28% of patients on vorasidenib experiencing disease progression, a stark contrast to 54% in the placebo group. Given the challenges of existing treatment options—which often lead to neurological deficits—vorasidenib presents a promising new avenue for this patient population. Although the drug is not yet FDA-approved, the findings, published in the New England Journal of Medicine, mark a significant advancement in glioma treatment.
