Two researchers from the Icahn School of Medicine at Mount Sinai recently published their critical evaluation of a new brain tumor medicine in the journal Nature Reviews Clinical Oncology . In the September 2024 issue, Stanislav Lazarev, MD, and Kunal Sindhu, MD, from Mount Sinai's Department of Radiation Oncology, offer a thorough critique of vorasidenib, a new drug recently approved by the Food and Drug Administration (FDA) for treating brain tumors called IDH-mutant low-grade gliomas (LGGs). Their research raises significant concerns about the approval process and the drug's actual benefits for patients.
LGGs are a type of brain tumor that has mutations in the isocitrate dehydrogenase (IDH) genes, which are important in the development and progression of these cancers. LGGs are typically slow-growing tumors that can affect brain function depending on their location. Vorasidenib is aimed at patients with these specific genetic mutations, offering a targeted treatment approach.
Drs. Lazarev and Sindhu scrutinize the INDIGO trial, the clinical study that supported vorasidenib's approval. One major issue they highlight is the trial's comparison methodology.
Instead of comparing vorasidenib directly with the current standard treatment, chemoradiotherapy, it was tested against a placebo. This approach has raised ethical concerns , as it involved withholding an established and effective treatment from some participants. Another critical point made is the lack of evidence showing .