New leukemia treatment approach targets cancer genes

Targeting two proteins that influence the development of leukemia may render cancer genes powerless, a study by an international research team suggests.

The research was a collaboration between scientists from the Mainz University Medical Center, the Cancer Center in New York, and Harvard University in Boston. Scientists involved with the study demonstrated that a targeted drug-based inactivation of two chromatin regulators can interrupt the development of leukemia in blood cells. Their findings were published in the journal Cancer Discovery.

Acute myeloid leukemia is a highly aggressive form of cancer that almost always results in death. The disease is typically treated using chemotherapy, but the Mainz-led research team sought to pursue a less toxic method.

During the experiment, researchers assessed the activation of homeobox stem cell genes, which scientists believe play a strong role in blood cells developing into leukemia cells. By examining a targeted manipulation of leukemia cell DNA, they were able to observe how two specific proteins are responsible for influencing cancerous development.

The research team then targeted the two proteins, called the mixed lineage leukemia protein and the disruptor of telemeric silencing 1-like, with specialized chemical agents that caused the leukemia cells to begin reverting back into normal blood cells.

The approach marked the first molecularly targeted treatment of NPM1mut leukemia by reversing a key mechanism of its development. Researchers are confident their findings will be helpful for creating new drugs to combat the disease.


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