Breakthrough Hope: A Revolutionary Advance in Treating Brain Cancer Called Glioma — The Drug Vorasidenib Represents The First Progress in 20 Years

Glioma is a primary brain tumor that originates from the glial cells present in the central nervous system. The glial cells have the primary function of giving structural support to the neurons (nervous cells), which are responsible for your thoughts, movements, sensations, equilibrium, and coordination.

Glioma, a 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) can also be symptoms.

These symptoms are not specific to glioma and can be caused by other neurologic illnesses. A clinician must be consulted to explore the clinical history and perform a physical examination carefully. 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.

How is Glioma Diagnosed?

Dr. Friedman tells SurvivorNet that patients may present [with symptoms] differently based on where their tumor is located.

“So different locations will give you motor deficits, sensory deficits, speech deficits, and seizures,” he explains, adding that an MRI, or magnetic resonance imaging, will then be done to diagnose the tumor.

Dr. Friedman stresses that after a tumor is found, patients are better off being referred to an academic center to determine the best form of treatment.

“When a patient is diagnosed with a brain tumor, typically that’s in an outside hospital or smaller community location, and then they’re referred to a quaternary center such as us,” he says.

Making Treatment Decisions

After evaluating symptoms and interpreting imaging exams, the treatment team will grade the tumor. Glioma ranges from grade one to four. The more advanced the disease is, the higher the tumor grade.

“Grade one is a very indolent, benign tumor that basically can be cured with surgery alone,” Dr. Freidman explains. “Grade two is not cured with surgery alone. There’s always going to be microscopic disease left behind even after what appears to be a gross total resection … Grade threes are more aggressive, and grade fours are the most aggressive tumor.”

This step not only defines the patient’s stage of the disease but also helps drive treatment decisions, including whether to give surgery, radiation therapy, and/or chemotherapy. Low-grade patients can benefit from surgery and systemic therapy with medication. For this set of patients, recent researchers indicates Vorasidenib can significantly prolong survival.

A Pioneering New Treatment Option: Vorasidenib

After more than twenty years without considerable advancements, the recent data on Vorasidenib has sparked a new future for patients diagnosed with grade two 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.

From this perspective, 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 pharmaceutical companies.

“So 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 groundbreaking development occurred in August 2024 when the Food and Drug Administration (FDA) approved Vorasidenib for treating both adults and children aged 12 and older with IDH-mutant, grade two 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, which means stable disease for a more extended period than the group that did not receive the drug. With that, 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 (a natural protection to the brain). Vorasidenib, however, is a brain-penetrant inhibitor, meaning it possesses the capability to cross this barrier effectively.

“Patients with grade two 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 adds.

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