For decades, glioma treatment relied mainly on surgery, radiation, and chemotherapy. A newer category — targeted therapy — attacks the specific molecular features driving a tumor, and it's beginning to change the landscape, especially for tumors defined by particular mutations.
Vorasidenib: a landmark for IDH-mutant glioma In August 2024, the FDA approved vorasidenib (Voranigo) — the first targeted therapy specifically for IDH-mutant grade 2 astrocytoma or oligodendroglioma, in adults and children 12 and older, after surgery. It's an oral, once-daily pill that crosses into the brain and blocks the mutant IDH enzyme that helps drive these tumors. In a large randomized trial (INDIGO), vorasidenib significantly delayed tumor growth and the time until patients needed their next treatment, compared with placebo — a meaningful advance for a group of patients who previously faced a choice between radiation/chemotherapy and watchful waiting.
Why is this important? IDH-mutant low-grade gliomas often affect younger adults and can be managed over years, so a well-tolerated pill that delays progression — and potentially delays the need for radiation, with its long-term cognitive effects — is genuinely significant.
Other targeted therapies in glioma
- BRAF-targeted drugs. Many pediatric low-grade gliomas carry BRAF gene alterations. Dabrafenib plus trametinib is used for tumors with a BRAF V600E mutation, and tovorafenib (Ojemda) for certain BRAF fusions and rearrangements (covered in the pediatric articles).
- Dordaviprone for H3 K27M diffuse midline glioma (covered in the DIPG article).
- Other targets (such as NTRK fusions) may be relevant in rare cases.
An honest frame Targeted therapy depends entirely on having the right molecular target — which is why molecular testing matters so much. Not every glioma has a targetable mutation, and these drugs have their own side effects and limits. But this is one of the most hopeful and fast-moving areas of neuro-oncology, and it's worth asking whether your tumor has any targetable features.