Bromodomain brake on AML
A Cold Spring Harbor Laboratory-led team has shown that a bromodomain inhibitor can help treat acute myeloid leukemia in mice, greatly expanding the therapeutic potential forthis emerging drug class.1 Newly founded Tensha Therapeutics Inc. haslicensed the findings and hopes to bring a compound into Phase I trials within two years.
Bromodomain containing proteins regulate gene expression by binding to acetylated histones and altering chromatin structure. They were widely believed to be undruggable until late last year, when two groups reported small molecules that selectively inhibited the BET family of bromodomain containing proteins, specifically bromodomain containing 2 (BRD2), BRD3 and BRD4.2,3
Translocation of BRD4 has been linked to NUT midline carcinoma, a rare form of aggressive cancer diagnosed in 20-40 people per year in the U.S. In vivo proof of conceptforBET inhibition was seen in a xenograft mouse model of