BioCentury
ARTICLE | Targets & Mechanisms

AML takes LSD1

April 5, 2012 7:00 AM UTC

Two teams have independently provided preclinical proof of concept for using lysine-specific histone demethylase 1 inhibitors to treat acute myelogenous leukemia.1,2 One of the teams is now running a clinical trial of a generic monoamine oxidase inhibitor that counts the demethylase among its many targets, whereas at least four biotechs are advancing other more potent and selective inhibitors through preclinical development.

In 2004, researchers at Harvard Medical School and The Johns Hopkins University School of Medicine identified the monoamine oxidase lysine-specific histone demethylase 1 (KDM1A; LSD1) as being able to demethylate histones, thus constituting a key step in the regulation of chromatin structure and gene expression.3 Subsequent studies found LSD1 overexpression in several types of cancer, and small interfering RNA experiments showed LSD1 knockdown suppressed cancer cell growth.4,5...