Macarena I de la Fuente, Mehdi Touat, Martin J van den Bent, Matthias Preusser, Katherine B Peters, Robert J Young, Raymond Y Huang, Benjamin M Ellingson, David Capper, Joanna J Phillips, Lia M Halasz, Helen A Shih, Roberta Rudà, Mary Jane Lim-Fat, Deborah T Blumenthal, Michael Weller, Yoshiki Arakawa, James R Whittle, François Ducray, David A Reardon, Wenya Linda Bi, Giuseppe Minniti, Rifaquat Rahman, Shawn Hervey-Jumper, Susan M Chang, Patrick Y Wen
{"title":"沃拉西尼在异柠檬酸脱氢酶突变型胶质瘤治疗中的作用。","authors":"Macarena I de la Fuente, Mehdi Touat, Martin J van den Bent, Matthias Preusser, Katherine B Peters, Robert J Young, Raymond Y Huang, Benjamin M Ellingson, David Capper, Joanna J Phillips, Lia M Halasz, Helen A Shih, Roberta Rudà, Mary Jane Lim-Fat, Deborah T Blumenthal, Michael Weller, Yoshiki Arakawa, James R Whittle, François Ducray, David A Reardon, Wenya Linda Bi, Giuseppe Minniti, Rifaquat Rahman, Shawn Hervey-Jumper, Susan M Chang, Patrick Y Wen","doi":"10.1093/neuonc/noae259","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":19377,"journal":{"name":"Neuro-oncology","volume":" ","pages":"1135-1148"},"PeriodicalIF":16.4000,"publicationDate":"2025-06-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12187374/pdf/","citationCount":"0","resultStr":"{\"title\":\"The role of vorasidenib in the treatment of isocitrate dehydrogenase-mutant glioma.\",\"authors\":\"Macarena I de la Fuente, Mehdi Touat, Martin J van den Bent, Matthias Preusser, Katherine B Peters, Robert J Young, Raymond Y Huang, Benjamin M Ellingson, David Capper, Joanna J Phillips, Lia M Halasz, Helen A Shih, Roberta Rudà, Mary Jane Lim-Fat, Deborah T Blumenthal, Michael Weller, Yoshiki Arakawa, James R Whittle, François Ducray, David A Reardon, Wenya Linda Bi, Giuseppe Minniti, Rifaquat Rahman, Shawn Hervey-Jumper, Susan M Chang, Patrick Y Wen\",\"doi\":\"10.1093/neuonc/noae259\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Isocitrate dehydrogenase (IDH)-mutant gliomas are the most common malignant primary brain tumors in young adults. 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The role of vorasidenib in the treatment of isocitrate dehydrogenase-mutant glioma.
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.
期刊介绍:
Neuro-Oncology, the official journal of the Society for Neuro-Oncology, has been published monthly since January 2010. Affiliated with the Japan Society for Neuro-Oncology and the European Association of Neuro-Oncology, it is a global leader in the field.
The journal is committed to swiftly disseminating high-quality information across all areas of neuro-oncology. It features peer-reviewed articles, reviews, symposia on various topics, abstracts from annual meetings, and updates from neuro-oncology societies worldwide.