BioCentury
ARTICLE | Distillery Therapeutics

Therapeutics: Lysine-specific demethylase 1 (LSD1; KDM1A)

January 14, 2016 8:00 AM UTC

In vitro and mouse studies identified tranylcypromine-based inhibitors of LSD1 that could help treat leukemia. Chemical synthesis and in vitro testing of tranylcypromine analogs identified three compounds that inhibited LSD1 with IC50 values of 0.028-0.188 μM. In human and mouse leukemia cell lines, each compound decreased colony formation compared with vehicle. In a mouse model of leukemia, each compound increased survival compared with vehicle. Next steps could include optimizing and testing the compounds in xenograft models of leukemia.

Oryzon Genomics S.A. and Roche have the LSD1 inhibitor RG6016 (ORY-1001) in Phase I/II testing to treat acute myelogenous leukemia (AML)...