Advances in AML treatment target leukemia stem cells and transplant decisions

Researchers are developing new methods to combat acute myeloid leukemia (AML), a fast-growing blood and bone marrow cancer that often relapses due to surviving leukemia stem cells.

At Thomas Jefferson University, scientists discovered that a combination of three drugs—inobrodib, venetoclax, and gilteritinib—was significantly more effective at killing leukemia cells in laboratory and mouse studies than using the drugs individually or in pairs. While inobrodib alone slowed cancer growth, the three-drug combination successfully targeted the resilient stem cells responsible for relapse.

In a separate development, researchers from institutions including Chongqing Medical University and Sun Yat-Sen University have developed a machine-learning model called HSCT-64. This transcriptomic framework uses RNA-sequencing data to help clinicians decide which pediatric AML patients would most benefit from allogeneic hematopoietic stem cell transplantation. By analyzing gene expression profiles, the model aims to provide an objective assessment of disease severity, helping to ensure timely transplants for high-risk patients while avoiding unnecessary procedures for others.