Pyrvinium Selectively Targets Blast Phase Chronic Myeloid Leukaemia Through Inhibition Of Mitochondrial Respiration

The use of Bcr-Abl tyrosine kinase inhibitors (TKIs) has led to excellent clinical responses in patients with chronic phase chronic myeloid leukaemia (CML). However these TKIs have been less effective as single agents in blast phase (BP) CML and this represents an urgent unmet need. A number of novel agents are now being investigated but most have not been translated to the clinics yet. Pyrvinium, a FDA-approved anthelminthic drug, was reported to selectively inhibit growth of a number of tumour cell types including myeloma and erythroleukaemia. We investigated the effect and mechanism of action of pyrvinium in BP-CML.