Oral Drug for Brain Tumor Could Change Treatment Landscape

12 Jun 2023 • Patients with a certain type of brain tumor could soon be treated with an oral targeted drug instead of undergoing more toxic chemotherapy and radiation, say researchers reporting new results that could potentially change the treatment landscape. Results from the pivotal phase 3 INDIGO trial, involving 331 patients show that the drug was associated with a significant delay in time to disease progression when compared with placebo. The median progression-free survival (PFS) was 27.7 months for patients on vorasidenib, compared with 11.1 months for patients assigned to placebo.

The study was published online in The New England Journal of Medicine. In patients with grade 2 IDH-mutant glioma, vorasidenib significantly improved progression-free survival and delayed the time to the next intervention. Vorasidenib was also associated with significantly longer time to the next treatment, and patients generally tolerated the drug well, reported first author Dr. Ingo K. Mellinghoff, MD. The results show that "treatment with an oral precision medicine therapy can produce a reduction in the risk of tumor progression by 61%, so that is, we think, a significant sign of efficacy that has potential to change the landscape in this disease," he commented.

The results of this study suggest that in selected patients with IDH mutant low-grade gliomas we can potentially delay the use of these toxic chemotherapies and radiation, maybe for years if not many years, and as a result delay the long-term toxicities of those therapies in a group of patients who typically are experiencing long-term survival.

This study is the first clinical trial to examine a targeted therapy drug specifically designed to treat brain cancer, with Vorasidenib able to cross the blood-brain barrier.

Source: NEJM | Read full story

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