Dr. Pardee on CPI-613 in Hematologic Malignancies

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.