Spotlight on AML — Biology and Pathophysiology

Mark G. Frattini, MD, PhD

Room 308 will be abuzz three times over the next two days with discussion of the biology and pathophysiology of acute myeloid leukemia (AML). These oral sessions will occur today from 7:00 to 8:30 a.m. and 2:45 to 4:15 p.m. and on Tuesday from 7:30 to 9:00 a.m.

The first session today (abstracts 175-180) will focus on the genetics and genomics of AML and promises to have a lot of international flare with talks from investigators from France, Germany, Israel, Japan, and the United States. Highlights will include the emergence of MYH11 and NOTCH1 as possible recurrent mutations in normal cytogenetic AML, use of genome-wide analysis of alternative splicing as a method to identify potential new therapeutic targets, increased transcriptional activity secondary to NPM1 mutation, and results of a phosphatome and kinome RNAi screen performed in four separate leukemia cell lines.

This afternoon’s session (abstracts 499-504) will focus on how the immune system is affected by AML specifically by evading immune surveillance and suppressing NK cell function. In addition, there will be discussions on how microRNAs affect both the onset and cellular differentiation of AML and the emergence of the AXL/GAS6 pathway as a novel and specific method of targeting FLT3-ITD positive AML.

The final session on Tuesday morning (abstracts 871-876) will concentrate on the epidemiology and pathophysiology of acute promyelocytic leukemia (APL) and AML in Down syndrome patients. In terms of APL, there will be a discussion of the importance of risk-adapted therapy in children with newly diagnosed APL, and for adults, a population-based study suggesting a possible higher early death rate and lower overall survival than what is reported in clinical trials. The presentations on Down syndrome will entail a discussion of organ dysfunction, gene alterations, and perturbation of hematopoiesis in patients with Down syndrome and myeloid leukemia.

Bottom line: There is a lot going on concerning the biology and pathophysiology of AML — don’t miss it. 

Dr. Frattini indicated no relevant conflicts of interest.