KDM6B-specific pharmacological inhibitor GSK-J4 had a significant anti-proliferative effect in AML cell lines and freshly isolated BM monocytes (MNCs) from AML patients, while H3K27me3 levels were also increasing. GSK-J4 also caused apoptosis and cell cycle arrest in vitro, and reduced tumor burden in vivo in AML xenograft mouse models.

It is worth noting that the injection of GSK-J4 attenuated disease progression in a human AML xenograft mouse model. Treatment with GSK-J4 mainly resulted in the downregulation of DNA replication and cell cycle-related pathways and prevents the expression of HOX, a key cancer gene. ChIP-qPCR verified the increased H3K27me3 enrichment in the HOX gene transcription initiation site [1].