A new drug for treatment of a type of brain cancer, called IDH-mutant low-grade glioma, was approved August 6 by the U.S. Food and Drug Administration (FDA). The promising new drug stems from a 2008 genetic discovery made at the Johns Hopkins Kimmel Cancer Center.
The drug, called vorasidenib, is a targeted cancer therapy that works by inhibiting the activity of a mutated gene called IDH, slowing the growth of the cancer. The gene was identified by Bert Vogelstein, M.D., and team at the Johns Hopkins Kimmel Cancer Center’s Ludwig Center in 2008 when they mapped the genetic blueprint for brain cancer.
The researchers found that the IDH gene—which had never been suspected to be involved in any tumor type—was frequently mutated in a subset of brain cancers.
In June 2023, findings from a multicenter, phase 3 clinical trial of vorasidenib in 331 patients with IDH-mutant low grade glioma was published in the New England Journal of Medicine and concluded that patients with grade 2 IDH-mutant glioma who received the drug had significantly improved progression-free survival and that the therapy delayed the time to the next intervention compared to patients who received a placebo. The trial was sponsored by the international pharmaceutical company Servier, who is bringing vorasidenib to market.
In addition to this newly FDA-approved drug, the IDH gene discovery led to a new classification of gliomas, differentiating cancers with an IDH mutation that have overall better outcome and response to treatment from very aggressive gliomas without an IDH mutation, including glioblastoma, the most common primary brain cancer in adults. It has also paved the way for additional studies in other types of brain cancer.
