AI-10-49

Description: AI-10-49 is a potent and selective inhibitor of the binding of CBFbeta-SMMHC to RUNX1 with IC50 of 260 nM. AI-10-49 restores RUNX1 transcriptional activity, displays favorable pharmacokinetics, and delays leukemia progression in mice. Treatment of primary inv(16) AML patient with AI-10-49 triggers selective cell death. Direct inhibition of the oncogenic CBFbeta-SMMHC fusion protein may be an effective therapeutic approach for inv(16) AML, and they provide support for transcription factor targeted therapy in other cancers. The stability of RUNX1, CBFb, and CBFb-SMMHC was not affected by AI-10-49.

References: Science. 2015 Feb 13; 347(6223):779-84.