Biological Activity
CCI-007 is a small molecule with cytotoxic activity against infant leukemia with MLL rearrangements, with IC50 values of 2.5-6.2 μM in sensitive cells[1].
CCI-007 is a selective inhibitor of MLL-r, CALM-AF10 and SET-NUP214 leukemia[1].Following CCI-007 treatment for 24h, significant mitochondrial depolarization was observed in PER-485 cells as evidenced by a shift in JC-1 signal[1].Resistance to CCI-007 can occur by upregulation of MLL target gene expression[1].CCI-007 reverts the aberrant elevated transcription of disease-driving factors HOXA9, MEIS1, and CMYC downstream of the pathogenic MLL fusion protein, thereby inducing MLL-r leukemia cell death[2]. Cell Viability Assay[1] Cell Line: PER-485, MOLM-13, MV4;11 cells.
References
[1]. Klaartje Somers, et al. CCI-007, a novel small molecule with cytotoxic activity against infant leukemia with MLL rearrangements. Oncotarget. 2016 Jul 19;7(29):46067-46087.
[2]. Klaartje Somers, et al. A novel small molecule that kills a subset of MLL-rearranged leukemia cells by inducing mitochondrial dysfunction. Oncogene. 2019 May;38(20):3824-3842.
