CPI-613 is primarily used in cancer research and experimental therapy, focusing on targeting the altered energy metabolism of cancer cells. It selectively disrupts mitochondrial enzymes involved in energy production, particularly pyruvate dehydrogenase and α-ketoglutarate dehydrogenase, which are critical for tumor cell survival. This disruption leads to metabolic stress and cell death in malignant cells, especially in cancers with high metabolic demands such as pancreatic cancer, leukemia, and lymphoma. It has been investigated in clinical trials, often in combination with other chemotherapeutic agents, to enhance their effectiveness. For example, when used with modified FOLFIRINOX in patients with advanced pancreatic cancer, CPI-613 has shown potential to improve response rates and survival outcomes. Its ability to sensitize resistant cancer cells to conventional treatments makes it a valuable candidate in developing combination therapies for aggressive and hard-to-treat cancers.