New drug shows potential to revolutionize brain tumor treatment

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In the quest to combat brain tumors, particularly grade 2 IDH-mutant gliomas often diagnosed in younger individuals, a beacon of hope arises.

A notable expert from UVA Health, Dr. David Schiff, MD, has emphasized the transformative potential of a new drug named vorasidenib.

His insights, shared in the New England Journal of Medicine, hint at a groundbreaking advancement in the treatment of such brain tumors.

Unlocking New Potential with Vorasidenib

Vorasidenib, tested in the INDIGO clinical trial, has displayed promising results, substantially slowing down the tumor growth in patients suffering from grade 2 IDH-mutant gliomas.

In the trial, it extended the average time before the tumor started growing again, from 11.1 months to over 27 months.

Dr. Schiff regards these outcomes as “striking”, raising hopes that the drug could revolutionize the approach towards managing these tumors.

Gliomas and their Impact

About 2,500 Americans, primarily around the age of 40, are diagnosed with these gliomas annually.

These tumors severely affect cognitive functions, impacting daily life, employment, and ultimately become fatal as they grow resistant to existing treatments.

The current approach is often “watch and wait”, reserving treatment for when the tumor shows progression, due to limited available treatment options.

The INDIGO Trial’s Revelation

The INDIGO trial included over 300 patients, who were given either vorasidenib or a placebo, with neither the patients nor their doctors knowing which one they received.

Remarkably, those administered vorasidenib not only lived longer but also delayed the need for more intensive and toxic treatments like radiation and chemotherapy compared to those who received placebos.

A Step towards Approval and Beyond:

The exciting results from vorasidenib trials are now awaiting approval from the Federal Food and Drug Administration.

If approved, it will mark the inception of the first targeted therapy for low-grade gliomas, potentially putting an end to the prevailing “watch and wait” methodology.

However, as Dr. Schiff notes, there are still numerous unanswered questions on how to optimize the utilization of this medication post its approval, considering the adverse effects associated with the current standard therapies such as radiation and chemotherapy.

A New Horizon in Cancer Treatment

Dr. Schiff’s belief in vorasidenib’s potential to replace the current approaches illustrates the substantial progress in our understanding and management of gliomas.

Notably, gliomas with the IDH gene mutation show distinct biology, outcomes, and vulnerabilities that can be addressed by new therapies, enabling the development of more effective treatment strategies.


Vorasidenib’s promising results shine a light on the potential new paths in treating brain tumors, especially those typically affecting younger people.

This development could not only extend the lives of patients but also improve their quality of life by avoiding more toxic treatments, marking a substantial leap forward in cancer treatment.

It is a significant stride towards more humane and effective treatment options, allowing patients to retain a sense of normalcy in their lives while battling such a daunting condition.

The medical community eagerly anticipates its approval and subsequent integration into therapeutic regimens to turn the tide against grade 2 IDH-mutant gliomas.

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The research findings can be found in the New England Journal of Medicine.