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Peer-reviewedCC BY-NC 4.0Journal of Korean Neurosurgical Society

Targeting Isocitrate Dehydrogenase-Mutant Glioma with Vorasidenib : A New Era in Precision Neuro-Oncology

11 affiliations
  1. 1Korean Brain Tumor Society, Seoul, Korea
  2. 2Department of Neurosurgery, Inha University Hospital, Inha University College of Medicine, Incheon, Korea
  3. 3Department of Neurosurgery, Asan Medical Center, Ulsan University College of Medicine, Seoul, Korea
  4. 4Department of Neurosurgery, School of Medicine, Kyungpook National University, Daegu, Korea
  5. 5Department of Neurosurgery, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea
  6. 6Department of Neurosurgery, Seoul National University Bundang Hospital, Seoul National University College of Medicine, Seongnam, Korea
  7. 7Neuro‑Oncology Clinic, National Cancer Center, Goyang, Korea
  8. 8Department of Neurosurgery, Dong-A University College of Medicine, Busan, Korea
  9. 9Department of Neurosurgery, Severance Hospital, Yonsei University College of Medicine, Seoul, Korea
  10. 10Department of Neurosurgery, Kyungpook National University Chilgok Hospital, School of Medicine, Kyungpook National University, Daegu, Korea
  11. 11Department of Neurosurgery, Korea University Anam Hospital, Seoul, Korea

Abstract

Advancement of molecular targeted therapies has revolutionized the treatment of several challenging and previously refractory tumors. Since the introduction of rituximab in 1997, the field of molecular targeted agents have continued to evolve. Notably, vorasidenib, a brainpenetrant dual inhibitor of mutant isocitrate dehydrogenase (IDH) 1 and 2, has recently demonstrated promising results in the 2023 INDIGO trials with improved progression-free survival and delayed need for subsequent interventions in patients with IDH-mutant low-grade gliomas. Although therapeutic options for glioma have long remained largely limited to surgery, radiation, and conventional chemotherapy despite extensive and ongoing research, the advent of vorasidenib opens a new chapter in glioma treatment. This review summarizes the key clinical studies of vorasidenib and discusses its potential role, limitations, and future directions in the evolving treatment paradigm of IDH-mutant gliomas.

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BibTeX
@article{kim2026targeting,
  title = {{Targeting Isocitrate Dehydrogenase-Mutant Glioma with Vorasidenib : A New Era in Precision Neuro-Oncology}},
  author = {Kyung-Min Kim and Yukyeng Byeon and Chaejin Lee and Won‑Jae Lee and Kihwan Hwang and Yun-Sik Dho and Kyoung Su Sung and Tae Hoon Roh and Kyuha Chong and Ki-Su Park and Shin-Hyuk Kang and Eui Hyun Kim},
  journal = {Journal of Korean Neurosurgical Society},
  year = {2026},
  month = jun,
  doi = {10.3340/jkns.2026.0113},
  url = {https://doi.org/10.3340/jkns.2026.0113},
  note = {CC BY-NC 4.0},
}
RIS
TY  - JOUR
TI  - Targeting Isocitrate Dehydrogenase-Mutant Glioma with Vorasidenib : A New Era in Precision Neuro-Oncology
AU  - Kyung-Min Kim
AU  - Yukyeng Byeon
AU  - Chaejin Lee
AU  - Won‑Jae Lee
AU  - Kihwan Hwang
AU  - Yun-Sik Dho
AU  - Kyoung Su Sung
AU  - Tae Hoon Roh
AU  - Kyuha Chong
AU  - Ki-Su Park
AU  - Shin-Hyuk Kang
AU  - Eui Hyun Kim
PY  - 2026
DA  - 2026/06/24
JO  - Journal of Korean Neurosurgical Society
DO  - 10.3340/jkns.2026.0113
UR  - https://doi.org/10.3340/jkns.2026.0113
AB  - Advancement of molecular targeted therapies has revolutionized the treatment of several challenging and previously refractory tumors. Since the introduction of rituximab in 1997, the field of molecular targeted agents have continued to evolve. Notably, vorasidenib, a brainpenetrant dual inhibitor of mutant isocitrate dehydrogenase (IDH) 1 and 2, has recently demonstrated promising results in the 2023 INDIGO trials with improved progression-free survival and delayed need for subsequent interventions in patients with IDH-mutant low-grade gliomas. Although therapeutic options for glioma have long remained largely limited to surgery, radiation, and conventional chemotherapy despite extensive and ongoing research, the advent of vorasidenib opens a new chapter in glioma treatment. This review summarizes the key clinical studies of vorasidenib and discusses its potential role, limitations, and future directions in the evolving treatment paradigm of IDH-mutant gliomas.
SN  - 2005-3711
SN  - 1598-7876
ER  -