Breakthrough Hope: A Revolutionary Advance in Glioma Treatment After 20 Years

Written by Dr. Kaique Filardi

After more than twenty years without considerable advancements, a recent discovery has sparked a new future for patients diagnosed with grade 2 glioma. This achievement comes from studies that have delved into a better understanding of how genetic mutations affect the cancer environment and, most importantly, how these new genes can drive new treatments.

A drug called Vorasidenib was recently approved by the Food and Drug Administration (FDA) for use in adults and children ≥12 years of age with an isocitrate dehydrogenase (IDH) mutant, grade 2 glioma. 

SurvivorNet Connect consulted Dr. Henry Friedman, a deputy director at the Preston Robert Tisch Brain Tumor Center at Duke University to understand which patients are eligible for this promising new treatments and what physicians need to know. 

Diagnosing & Grading Gliomas

This primary brain tumor can manifest with a variety of symptoms that will depend on the size and localization of the tumor within the skull.  Generally, the first symptom observed is a seizure episode, but headaches, movement and sensibility deficits, and loss of consciousness (passing out) may all indicate glioma as well. Upon suspicion of a glioma or a brain tumor diagnosis, it is essential to promptly refer the patient to a neurology specialist for further evaluation and management. 

“The way [patients] present is based on the location of the tumor. So different locations will give you motor deficits, sensory deficits, speech deficits, and seizures. Different kinds of problems can happen, but the common pathway is that ultimately, an MRI is done, a tumor is visualized and they either take the first steps in the community” setting, or a cancer center,” Dr. Friedman explains.

He recommends patients be transferred to larger, academic centers where possible.

They are “much better off if [testing is] done at an academic center because the tissue is handled in a certain way and things that are done at a [major] center go far beyond what’s done in the local communities,” he adds.

The tumor’s grade, among other factors, will determine if the patient is eligible for an innovative new treatment. 

Finding the Treatment Path: What’s New?

Grading not only defines the patient’s stage of the disease but also supports treatment decisions. Surgery, radiation therapy, and chemotherapy may all be part of the treatment path. Low-grade glioma patients can benefit from surgery and systemic therapy with medication. In this set of patients, a recent group of researchers discovered a new drug that can prolong survival. 

The discovery of a mutation in the gene encoding the protein called isocitrate dehydrogenase (IDH) allowed scientists to realize that the IDH mutation is closely associated with the development and progression of glioma. Consequently, it has led to numerous studies across the oncology community and pharmaceutics companies.

“Grading a tumor is very important, but knowing if there is a mutation or if there are mutations, such as the IDH1 mutation and IDH2 mutation, becomes very helpful from a therapeutic standpoint as well as a diagnostic standpoint. When you’ve got that mutation, you do better,” Dr. Friedman explains.

A new drug called Vorasidenib was approved by the FDA in August 2024 for use in adults and children ≥12 years of age with an IDH-mutant, grade 2 glioma. 

This medication is a dual inhibitor of the IDH mutation (IDH1 and IDH2), which is administered orally once daily. In the study, patients who had taken this medication were shown to significantly improve progression-free survival. The period of time to have new interventions also increased (time to next intervention). 

Despite significant advances in the use of targeted therapies for various cancers, developing such treatments for brain tumors remains particularly challenging due to the difficulty of some drugs penetrating the blood-brain barrier. Vorasidenib, however, is a brain-penetrant inhibitor, meaning it possesses the capability to cross this barrier effectively.

“Patients with grade 2 glioma who had IDH1 mutation treated with Vorasidenib doubled in terms of survival — and it’s a remarkable story. It’s splashed all over the media, and it’s a game changer for these patients. So now we have a different strategy,” Dr. Friedman explains.