Inhibitor of 4-Hydroxyphenylpyruvate dioxygenase (HPPD, IC50 = 40 nM1 and 173 nM2) and is in clinical use for the treatment of hereditary tyrosinemia type 13. CD13+ cancer stem cells (CSCs) are dependent on aerobic metabolism of tyrosine - Nitisinone inhibition of tyrosine metabolism results in lowered availability of acetyl-CoA and fumarate for use in the citric acid cycle causing these CSCs to enter cell cycle, decreasing self-renewal, and making them more susceptible to chemotherapy.4 Nitisinone is a potential treatment option for cancers that rely on tyrosine metabolism.
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