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2HG, elevated in patients with IDH mutations, can also be measured in the serum and urine of affected patients, where it can serve as a biomarker of disease activity and therapeutic response. These advances triggered development of a new class of small molecule inhibitors of mutant IDH enzymes. After demonstrating promising anti-leukemic activity in animal models, phase 1 and 2 clinical trials of IDH inhibitors followed. Trials of the IDH1 inhibitor ivosidenib and the IDH2 inhibitor enasidenib demonstrated single agent activity, response rate, transfusion independence, and long-term outcomes.
Video Overview: https://youtu.be/WTX_WqOz5q8
Amir Fathi, MD
MGH/Harvard Medical School
STO gratefully acknowledges educational grants in partial support of this activity from:
Novartis Pharmaceuticals Corp.