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).

Twenty four patents with relapse or refractory AML received this agent in combination with high-dose cytarabine and mitoxantrone. The response rate was 54% (11 complete remissions and 2 complete remissions with incomplete count recovery).

Pardee says that, typically, patients with poor risk cytogenetics tend to respond poorly to chemotherapy. On this trial, the response rate of patients with poor risk cytogenetics was 53%.

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Timothy S. Pardee, MD, discusses the results of a phase I study of the mitochondrial metabolism inhibitor CPI-613 for relapsed or refractory acute myeloid leukemia (AML).

Dr. Pardee on CPI-613 for Patients With R/R AML

Timothy S. Pardee, MD, Assistant Professor, Hematology & Oncology, Wake Forest University Baptist Medical Center, describes a phase I study of the first-in-class pyruvate dehydrogenase complex inhibitor CPI-613 in patients with advanced hematologic malignancies.

CPI-613 attacks cancer cells in a different way, Pardee says, hitting the mitochondria and altering the ability of the cells to make the building blocks of the next cancer cell. In the trial, 40% of the 21 evaluable, heavily-pretreated patients responded. Researchers also observed several outstanding responses.

Researchers saw some evidence of acute renal failure as well as nausea and diarrhea associated with the trial, however, no myelosuppression was observed. There are a number of ongoing trials using this drug.

Timothy S. Pardee, MD, describes a phase I study of the first-in-class pyruvate dehydrogenase complex inhibitor CPI-613 in patients with advanced hematologic malignancies.

Dr. Pardee Provides an Update on CPI-613

Timothy S. Pardee, MD, Assistant Professor, Hematology & Oncology, Wake Forest University Baptist Medical Center, describes a phase I trial looking at the first-in-class mitochondrial metabolism inhibitor CPI-613 in patients with advanced hematologic malignancies.

This drug is a small molecular inhibitor of a mitochondrial enzyme called pyruvate dehydrogenase complex. While normal cells use mitochondria as a means to efficiently generate energy, cancer cells use mitochondria to generate building blocks for more cancer cells. Because of that difference, Pardee says, researchers hypothesized that there would be a therapeutic wind to target cancer cells more specifically.

Through a collaboration with Cornerstone Pharmaceuticals, researchers tested CPI-613 in a phase I trial. The trial was design to determine the safest dose, toxicities, and activity in patients with relapsed/refractory hematologic malignancies. To their delight, Pardee says, researchers observed several dramatic responses.

Timothy S. Pardee, MD, describes a phase I trial looking at the first-in-class mitochondrial metabolism inhibitor CPI-613 in patients with advanced hematologic malignancies.

Timothy S. Pardee, MD

Articles

Dr. Pardee on CPI-613 for Patients With R/R AML

Dr. Pardee Provides an Update on CPI-613

Dr. Pardee on CPI-613 in Hematologic Malignancies