Hotspot point mutations in IDH1 occur in the vast majority of adult grade 2–3 gliomas. The understanding of their role in tumour biology continues to evolve. Therapeutic targeting of mutant IDH1 with vorasidenib demonstrated highly encouraging efficacy and minimal toxicity in a recent, randomized phase III trial involving patients with low-grade gliomas.
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Acknowledgements
The authors thank W. Kaelin (Dana–Farber Cancer Institute) for his input on this publication.
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D.A.R. has received financial compensation for advisory roles at Agios, AnHeart Therapeutics, Avita Biomedical, Blue Rock Therapeutics, Bristol–Myers Squibb, Boston Biomedical, CureVac AG, Del Mar Pharma, DNAtrix, Hoffman-LaRoche, Imvax, Janssen, Kiyatec, Medicenna Therapeutics, Neuvogen, Novartis, Novocure, Pyramid, Sumitomo Dainippon Pharma, Vivacitas Oncology and Y-mabs Therapeutics. D.P.C. has had consultant roles for Advise Connect Inspire, Boston Pharmaceuticals, Boston Scientific, German Accelerator, GlaxoSmithKline, Iconovir, Lilly, the Massachusetts Institute of Technology and Pyramid Biosciences.
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Reardon, D.A., Cahill, D.P. A new era for glioma therapy — targeting mutant IDH. Nat Rev Clin Oncol 20, 737–738 (2023). https://doi.org/10.1038/s41571-023-00804-8
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DOI: https://doi.org/10.1038/s41571-023-00804-8
