Pediatric acute myeloid leukemia (AML) is a genetically heterogeneous disease. This has made it difficult to establish relationships between specific genetic alterations and treatment response or disease progression/relapse. Developing new therapies to treat pediatric and adolescent AML has also been hindered by this heterogeneity.

Under the direction of Richard Aplenc, MD, PhD, MSCE, researchers at the Center for Childhood Cancer Research are analyzing clinical samples and medical outcomes data from Children Oncology Group (COG) sponsored AML clinical trials to establish possible molecular links between specific AML germline variants and treatment response.

While preliminary results from these analyses look somewhat promising, additional genome and whole exome sequencing studies are necessary to confirm links between specific genetic alterations and clinical outcomes or treatment responses for pediatric AML. Also, these analyses may help to identify new molecular targets that can be used to develop novel targeted cancer therapies to treat children and young adults with AML.