Cellular and Molecular Targeting of Recurrence in Acute Myeloid Leukemia

While disease recurrence remains the outstanding clinical challenge in acute myeloid leukemia (AML), the basis of relapse remains poorly characterized and thereby preventing effective therapeutic targeting. We performed gene expression analysis of human AML patient samples in addition to in vitro and in vivo assays of leukemic cell survival and self-renewal using xenograft modeling. These molecular and functional analyses afforded the identification of unique target genes that support recurrence. Preclinical modeling using these novel targets provided proof-of-principle for combination therapies towards more effective and durable suppression of AML regrowth.

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Boyd AL, Aslostovar L, Reid J, Ye W, Tanasijevic B, Porras DP, Shapovalova Z, Almakadi M, Foley R, Leber B, Xenocostas A, Bhatia M