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The Mechanism and Utility of CPI-613 for the Treatment of Hematologic Malignancies

Timothy S. Pardee, MD
Published Online:4:31 PM, Thu February 6, 2014
Timothy S. Pardee, MD, assistant professor, Hematology & Oncology, Wake Forest University Baptist Medical Center, discusses the mechanism and utility of the pyruvate dehydrogenase complex inhibitor CPI-613 for the treatment of patients with advanced hematologic malignancies.

Clinical Pearls

  • A phase I study is analyzing the first-in-class pyruvate dehydrogenase complex inhibitor CPI-613 in patients with advanced hematologic malignancies
  • CPI-613 attacks cancer cells by hitting the mitochondria, which alters the ability of the cells to make the building blocks of the next cancer cell
  • In the trial, 40% of the 21 evaluable and heavily-pretreated patients responded
  • There was some evidence of acute renal failure, nausea, and diarrhea associated with the trial, though no myelosuppression was observed
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