The role of vorasidenib in the treatment of isocitrate dehydrogenase-mutant glioma Journal Article


Authors: De La Fuente, M. I.; Touat, M.; Van Den Bent, M. J.; Preusser, M.; Peters, K. B.; Young, R. J.; Huang, R. Y.; Ellingson, B. M.; Capper, D.; Phillips, J. J.; Halasz, L. M.; Shih, H. A.; Rudà, R.; Lim-Fat, M. J.; Blumenthal, D. T.; Weller, M.; Arakawa, Y.; Whittle, J. R.; Ducray, F.; Reardon, D. A.; Bi, W. L.; Minniti, G.; Rahman, R.; Hervey-Jumper, S.; Chang, S. M.; Wen, P. Y.
Article Title: The role of vorasidenib in the treatment of isocitrate dehydrogenase-mutant glioma
Abstract: Isocitrate dehydrogenase (IDH)-mutant gliomas are the most common malignant primary brain tumors in young adults. This condition imposes a substantial burden on patients and their caregivers, marked by neurocognitive deficits and high mortality rates due to tumor progression, coupled with significant morbidity from current treatment modalities. Although surgery, radiation therapy, and chemotherapy improve survival, these treatments can adversely affect cognitive function, quality of life, finances, employment status, and overall independence. Consequently, there is an urgent need for innovative strategies that delay progression and the use of radiation therapy and chemotherapy. The recent Federal Drug Administration (FDA) approval of vorasidenib, a brain-penetrant small molecule targeting mutant IDH1/2 proteins, heralds a shift in the therapeutic landscape for IDH-mutant gliomas. In this review, we address the role of vorasidenib in the treatment of IDH-mutant gliomas, providing a roadmap for its incorporation into daily practice. We discuss ongoing clinical trials with vorasidenib and other IDH inhibitors, as single-agent or in combination with other therapies, as well as current challenges and future directions. © 2024 The Author(s). Published by Oxford University Press on behalf of the Society for Neuro-Oncology. All rights reserved.
Keywords: cancer radiotherapy; temozolomide; glioma; cancer immunotherapy; progression free survival; quality of life; drug synthesis; caregiver; cognitive defect; employment status; health care organization; isocitrate dehydrogenase; phase 3 clinical trial (topic); phase 1 clinical trial (topic); mortality rate; cancer prognosis; human; article; pembrolizumab; ivosidenib; isocitrate dehydrogenase inhibitor; vorasidenib; olutasidenib; isocitrate dehydrogenase (idh); idh-mutant; 1p-19q co-deleted oligodendroglioma; idh-mutant astrocytoma; safusidenib; federal drug administration; isocitrate dehydrogenase mutant glioma
Journal Title: Neuro-Oncology
Volume: 27
Issue: 5
ISSN: 1522-8517
Publisher: Oxford University Press  
Date Published: 2025-05-01
Start Page: 1135
End Page: 1148
Language: English
DOI: 10.1093/neuonc/noae259
PUBMED: 39723472
PROVIDER: scopus
PMCID: PMC12187374
DOI/URL:
Notes: Source: Scopus
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  1. Robert J Young
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