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NPM1 mutations in AML: What are they, and how do they influence the treatment of AML?
Harry Erba
July 18, 2025
Know AML was pleased to speak with Harry Erba, Professor of Medicine and Director of the Leukemia Program at Duke University in Durham, North Carolina. We asked, What are NMP1 mutations, and how do they influence the treatment of acute myeloid leukemia (AML)? Dr Erba explains that NPM1 mutations are a specific genetic change in the NPM1 gene, which causes over-production of stem cells in the bone marrow and blocks the normal process of stem cells developing into blood cells. This causes a specific subtype of AML, called NPM1-mutated AML.
Dr Erba discusses a new group of drugs called menin inhibitors, which block this abnormal process from taking place. He talks about the progress that has been made in clinical trials researching these drugs and explains that one of these menin inhibitors, ziftomenib, has been shown to work well and leads to high rates of remission when given to patients alongside chemotherapy. Dr Erba highlights that research is ongoing but that various drugs are being tested, which will hopefully have positive results for the treatment of people with NPM1-mutated AML.
This educational resource is independently supported by Kura Oncology. All content was developed by SES in collaboration with an expert ambassador group. Funders were allowed no influence on the content of this resource.
