Repurpose.

Pancreatic adenocarcinoma cells have uniquely fragmented mitochondria, suggesting a unique therapeutic target. This thought-provoking basic science work demonstrates that fusing these fragments back together reduces mitochondrial oxidative phosphorylation via mitophagy (yes, what it sounds like: autophagy of the mitochondria), which was directly associated with reduced tumor growth. In a brilliant move to bring their petri dishes closer to prime time, these scrappy authors determined that the already FDA-approved (for arthritis) drug leflunomide happens to double expression of a gene capable of—you guessed it—fusing fragmented mitochondria. When leflunomide was rebranded as an oncologic therapy (ka-ching!) and given to mice with pancreatic cancer, survival was dramatically improved. TBL: Coming to a clinical trial near you is the thoughtful repurposing of a proven-safe arthritis drug as a novel targeted therapy for pancreatic cancer. | Yu, JCI Insight 2019