A study published in Science Translational Medicine says that researchers at the RIKEN Center for Integrative Medical Sciences in Japan have identified a compound that can possibly prevent a relapse in acute myeloid leukemia patients. AML is the most common acute leukemia found in adults and starts in the blood-forming cells in the bone marrow.
Many patients suffering from AML fight off the disease with conventional chemotherapy. But majority of these patients often relapse later on. The research uncovered this compound that reduced the risk of a relapse in a mouse model of the disease. Bone marrow cells called leukemia stem cells (LSC) are said to cause the relapse when not properly eliminated during chemotherapy. So what the researchers did was to transplant the LSC from AML patients into immune-deficient newborn mice in order to study what causes the relapse. They were then able to discover a protein (HCK) that can be used as a target for healing agents against AML LSCs.
They then screened tens of thousands of molecules that could become therapeutic agents until they were able to isolate one small molecule that became active against the AML LSCs. When applied to the mouse model, it significantly reduced the AML cells in the blood and bone marrow of the mice. But the researchers say there is still a lot of work to be done before the molecule can actually be given to human patients. According to Dr. Ishikawa, the leader of the researchers, the next steps are to go more in-depth, to find out if it is safe and then finally develop a drug from this small molecule so that AML patients will benefit from it.