Prediction of Venetoclax Activity in Precursor B-ALL by Functional Assessment of Apoptosis Signaling

Small molecule inhibitors of key apoptosis regulators have been developed that bind to anti-apoptotic molecules like BCL-2, leading to release of pro-apoptotic proteins and cell death induction. This study shows that functional analysis of the interplay of apoptosis regulators in precursor B-ALL predicts preclinical in vivo activity of the BCL-2 inhibitor venetoclax and provides a robust assay for upfront identification of leukemias eligible for therapy. Gene expression was measured using Affymetrix platform in 27 precursor B-ALL xenograft samples