miRNA expression signatures are undergoing clinical investigation in pediatric patients with central nervous system (CNS) tumors (NCT01595126, NCT01556178), CNS tumors along with leukemia and lymphoma (NCT01541800), acute myeloid leukemia (AML) (NCT01229124), and neurofibromatosis Type 1 (NF-1) (NCT01595139). This evidence concerns the gene NF1 and acute myeloid leukemia.