Drugs Repurposed for Infant Leukemia

Scientists from the University of Edinburgh, led by Katrin Ottersbach, have identified three existing drugs - Acetazolamide, Tacrolimus, and LB-100 - as promising candidates for treating a severe form of infant leukemia (KMT2A::AFF1 positive B-cell precursor acute lymphoblastic leukaemia). In pre-clinical studies conducted in mice, these drugs demonstrated strong anti-leukaemic effects, significantly reducing disease burden. Acetazolamide, in particular, showed promising results by prolonging survival and improving the effectiveness of standard treatments with minimal toxicity, suggesting it could potentially replace or reduce reliance on the chemotherapy drug Cytarabine, known for its harsh side effects. The research, funded by organizations including the Kay Kendall Leukaemia Fund, Cancer Research UK, and the American Cancer Society, emphasizes the need for further clinical trials to confirm safety and effectiveness in human patients.