Scientists Discover Promising New Target for AML Treatment
Researchers at Hadassah Medical Center have identified a gene that could become a promising target for treating acute myeloid leukemia (AML), an aggressive form of blood cancer that often returns after treatment.
The study found that switching off a gene called IPO11 stopped leukemia cells from growing in laboratory tests and greatly reduced their ability to cause the disease in mice. Similar results were seen in leukemia cells taken from patients, including those whose cancer had returned after treatment. Although the findings are encouraging, researchers say the work is still in its early stages, and no drug targeting IPO11 has yet been developed or tested in humans.
AML is the most common type of acute leukemia in adults. It develops when immature blood cells in the bone marrow grow uncontrollably instead of maturing into healthy blood cells. This reduces the body’s ability to produce normal red blood cells, white blood cells, and platelets, leading to fatigue, infections, bleeding, and bruising. While treatments such as chemotherapy, targeted drugs, and stem cell transplants can put the disease into remission, many patients experience a relapse. Scientists believe this happens because a small group of leukemic stem cells survive treatment and later restart the disease.
To better understand these cells, researchers used CRISPR gene-editing technology to examine thousands of genes in AML cells. They identified IPO11 as one of the most important genes for the survival of both leukemia cells and leukemic stem cells.
IPO11 produces a protein called Importin-11, which helps transport other proteins into the cell’s nucleus, where they control important processes such as cell growth and division. When researchers switched off IPO11, leukemia cells stopped growing. Tests on patient samples showed the same result, including cells from patients with treatment-resistant AML.
The team also tested the cells in mice. Leukemia cells with an active IPO11 gene successfully grew and caused disease, while cells with the gene switched off struggled to survive and failed to produce leukemia in the same way. “We showed that silencing IPO11 dramatically impairs the ability of leukemic stem cells to function,” said Dr. Boaz Nachmias, senior physician in Hadassah’s hematology unit and director of its leukemia service.
The researchers are now working to develop a treatment that can block IPO11. However, they caution that more studies are needed to ensure such a treatment would be safe and effective, since the gene also plays important roles in healthy cells.
Although the research does not yet provide a cure for AML, it identifies a new weakness in leukemia cells that could eventually lead to more effective treatments and reduce the risk of the disease returning.
Source: The Voice of Science Website
Author Joyce Owusu
