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Lay abstract (no permission to release technical abstract): Drugs clinically used or tested for alternate indications that have unrecognized ability to kill leukemia cells and leukemia stem cells but not normal blood cells be rapidly re-purpositoned for the treatment of leukemia given their prior safety and toxicity testing. By screening a large chemical library of old drugs, we identified Tigecycline a drug used for treatment of serious infections that has unrecognized anti-leukemia activity. Mechanistically, tigecycline acted as an anti-leukemic agent by inhibiting mitochondrial protein synthesis. Here, we propose to rapidly advance this drug into clinical trial for patients with relapsed/refractory leukemia and initiate a phase I clinical trial of tigecycline in patients with this disease. In the context of this trial, we will measure levels of tigecycline in the blood and leukemia cells. We will also conduct “proof-of-mechanism” studies to determine whether tigecycline is inhibiting mitochondrial protein synthesis in our patients. Thus, this drug will be a first-in-class therapy for patients with cancer.

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