Poster Presentation 27th Lorne Cancer Conference 2015

The Bcl11a inhibitor simvastatin eliminates leukaemic stem cells (#151)

Philipp A Dietrich 1 , Murray D Norris 1 , Jenny Y Wang 1 2
  1. Children's Cancer Institute, Randwick, NSW, Australia
  2. School of Women's and Children's Health, Faculty of Medicine, Lowy Cancer Research Centre, University of New South Wales, Randwick, NSW, Australia

Persistence of cancer stem cells has been associated with disease relapse and drug resistance in a variety of malignancies, including acute myeloid leukaemia (AML) and breast, colon, and prostate cancers. One of the fundamental properties of cancer stem cells is their intrinsic capacity to self-renew. Previous studies have established beta-catenin as the central driver of self-renewal in cancer stem cells. More recent studies have shown that beta-catenin is required for the establishment of leukemic stem cells (LSC) in mixed lineage leukaemia (MLL)-AML. However, targeted inhibition of beta-catenin signalling has been hampered by the lack of pathway components amenable to pharmacological inhibition. Here we have identified a novel beta-catenin regulator, GPR84, a member of the G protein-coupled receptor family that represents a highly tractable class of drug targets. Our studies have shown that GPR84 simultaneously augments both β-catenin signalling and an oncogenic transcription program essential for the establishment of leukemia; and that aberrant activation of GPR84 is required for the maintenance of LSC in vivo. Microarray analysis of gene expression and subsequent quantitative real-time PCR also identified the proto-oncogene Bcl11a to be a new downstream target of GPR84. Significantly, treatment with simvastatin, a potent Bcl11a inhibitor, impaired LSC growth and survival, while GPR84 overexpression partially rescued this deficient phenotype in vitro (P=0.0094). Hence our results suggest that GPR84/β-catenin signalling is likely to represent an attractive therapeutic target in AML and simvastatin may be a promising candidate agent to eliminate AML LSC.