A novel targeted therapy drug, vorasidenib, has been shown to more than double the progression-free survival in patients with a subtype of glioma, according to an international study co-led by UCLA. By inhibiting mutant IDH1/2, the drug delays chemotherapy and radiation by almost 17 months, providing a breakthrough treatment for this deadly brain tumor.
In a study of 331 people with the disease, the drug was effective in lengthening the period of time before the patients’ cancer worsened, and with no observed adverse effects. The team found the drug vorasidenib more than doubled progression-free survival in people with recurrent grade 2 glioma with IDH1 and IDH2 mutations. Compared with people who received a placebo, those who took vorasidenib went for nearly 17 more months without their cancer worsening, delaying the time before they needed to begin chemotherapy and radiation.and presented on June 4, 2023, at the annual meeting of the American Society Clinical Oncology in Chicago.
Vorasidenib is classified as a dual inhibitor of mutant IDH1/2, meaning that it prevents the formation and accumulation of the onco-metabolite 2-Hydroxyglutarate, or 2-HG, that occurs when genetically altered versions of two enzymes, IDH1 and IDH2, are present in a tumor. 2-HG is thought to be responsible for the formation and maintenance of IDH-mutant gliomas.
While there has been great progress in using targeted therapies to treat many types of cancer, development of targeted therapies for brain tumors has been especially challenging because of the difficulty of getting through the blood-brain barrier. Vorasidenib is a brain-penetrant inhibitor, which means that it has the ability to cross the blood-brain barrier.
The disease progressed in just 28% of people receiving vorasidenib, compared to 54% of those receiving placebos. And as of September 2022, which was 30 months after the study began, 72% of patients who were in the vorasidenib group were still taking the drug and their disease had not progressed.
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