A Phase I Study of the Mitochondrial Metabolism Inhibitor CPI-613 for AML

Clinical Pearls

Timothy S. Pardee, MD, assistant professor, Hematology & Oncology, Wake Forest University Baptist Medical Center, discusses the results of a phase I study of the mitochondrial metabolism inhibitor CPI-613 for relapsed or refractory acute myeloid leukemia (AML).

• 24 patients with relapsed or refractory AML received the mitochondrial metabolism inhibitor CPI-613 in combination with high-dose cytarabine and mitoxantrone.
• The response rate was 54% (11 complete remissions + 2 complete remissions with incomplete count recovery).
• Patients with poor risk cytogenetics tend to respond poorly to chemotherapy.
• In this trial, the response rate of patients with poor risk cytogenetics was 53% (7/13).