ASCO 2023: New Research from Memorial Sloan Kettering Cancer Center Identifies Significant Treatment Advancement for Brain Tumors

Ingo Mellinghoff

Breakthrough data from researchers at Memorial Sloan Kettering Cancer Center (MSK) featured in the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting press program and simultaneously published in The New England Journal of Medicine (NEJM) today found that vorasidenib, an oral dual inhibitor of mutant IDH1/2 enzymes, significantly improved progression-free survival in patients with grade 2 diffuse gliomas, a type of malignant brain tumor. These findings represent a significant step forward in the treatment of grade 2 glioma with IDH mutations and were presented in the meeting’s press and plenary sessions on June 4, 2023, by Ingo K. Mellinghoff, MD, FACP.

The results of the Phase III INDIGO trial demonstrate significant improvement in progression-free survival, delay in the time to next treatment and present an opportunity to change the longstanding treatment paradigm in IDH-mutant glioma. Vorasidenib in IDH-mutant glioma, represents the first new treatment option, and first targeted therapy, in diffuse low-grade glioma in more than 20 years.


VIDEO | 02:08

New Experimental Drug Shows Promise for Brain Cancer

"This represents the first new treatment option in low-grade glioma in more than 20 years,” says Ingo Mellinghoff, MD, Chair of MSK’s Department of Neurology, who led a clinical trial showing the drug’s effectiveness.
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In patients with grade 2 gliomas with IDH mutations, vorasidenib led to a 61 percent reduction in the risk of tumor progression or death and significantly delayed the need for more toxic therapy when compared with placebo, as demonstrated by the results of the Phase III INDIGO study.

“Our study shows that targeting IDH mutations with vorasidenib significantly delays tumor growth and the need for more toxic therapies. This is clinically meaningful because patients diagnosed with grade 2 glioma with IDH mutations are typically young, otherwise healthy individuals. The results of the Phase III INDIGO trial offer a rare chance to change the treatment paradigm for this type of glioma and could bring the first new targeted therapy for diffuse low-grade glioma,” said lead author Dr. Mellinghoff, Chair of the Department of Neurology at MSK.  

Patients with IDH-mutant glioma are often diagnosed at a young age, on average in their fourth decade of life at diagnosis and have limited comorbidities early in the course of the disease. In the INDIGO study, a total of 331 total patients were randomized across ten countries, with a median age of 40 years old, and whose only previous treatment for glioma was surgery.

Foundational research for this study was supported by the MSK donor community, including Judith W. and Anthony B. Evnin and The AE Family Foundation, Cycle for Survival, the William Randolph Hearst Foundation, and the National Brain Tumor Society.

Dr. Ingo Mellinghoff has financial interests related to this research.  

John Connolly
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