Acute myeloid leukemia (AML) is a serious hematological malignancy whose treatment is often complicated by the development of drug resistance. Approximately one-third of patients harbor mutations in the protein kinase FLT3, which promote uncontrolled proliferation of leukemic cells. FLT3 is therefore an important target in the development of new anticancer drugs.
In a new study involving researchers from our department, scientists synthesized and evaluated 62 novel compounds derived from 7-azaindole. Their design was guided by structural information about the target protein, while systematic chemical modifications helped identify structural features important for biological activity. The most promising compound effectively inhibited the growth of leukemic cells, suppressed FLT3 signaling, and induced cell-cycle arrest and apoptosis. It also exhibited good metabolic stability and reduced leukemic blast counts in experimental studies using samples from AML patients. However, further analyses revealed that the compound also affects other protein kinases in addition to FLT3.
These findings represent another step toward the development of novel compounds with antileukemic activity. Further research will be needed to assess their potential therapeutic applications
J Transl Med. 2026
doi: 10.1186/s12967-026-09170-2