RK-20449 is an experimental compound that inhibits haematopoietic cell kinase, an enzyme critical for the survival of AML–acute myelogenous leukaemia stem cells.
Post- RK-20449 treatment, AML cells in immune compromised mice with transplanted leukaemia become undetectable. RK-20449 is most effective in cells from patients with a mutation of FLT3-ITD, a gene associated with aggressive AML. As of 2016, RK-20449’s status in the drug development pipeline is uncertain.
Reference Sci Transl Med 2013; 5:181ra52