Mutant IDH1 inhibitor treatment is currently in clinical trials for glioma patients. Therefore, in vivo biomarkers are needed to assess early therapeutic responses. Here we treated mutant IDH1-expressing cells and animals with patient-derived mutant IDH1 brain tumors with the emerging inhibitor BAY-1436032. Using 1H MRS we found a significant decrease in 2HG that was accompanied by an increase in glutamate and phosphocholine, and using 13C MRS we detected a significant decrease in 2HG and a significant increase in glutamate produced from hyperpolarized [1-13C] α-ketoglutarate in cell and animal models.
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