Functional Consequence and Therapeutic Targeting of Cryptic ALK Fusions
(ALK fus ) in Monosomy7 AML
Abstract
Acute Myeloid Leukemia (AML) patients have a wide array of cytogenetic
and molecular aberrations which can influence response to therapy.
Monosomy7 (Mono7) is a rare subset within pediatric AML (prevalence of
4-5%), that is highly associated with poor outcomes. Fusions involving
the ALK gene (14.3%) were exclusively identified within this
high-risk cohort while absent across all other AML. Given the dismal
outcomes of Mono7, we evaluated the use of crizotinib, an FDA-approved
tyrosine kinase inhibitor, used to treat patients with ALK fusions. Our
findings suggest that crizotinib may serve as a novel therapy for these
patients.