Leukemia Drug Development May Target Specific Protein

Existing treatments for acute myeloid leukemia (AML) are only effective in 20 percent to 40 percent of cases. Researchers at the University of Texas Health Science Center have found a protein that may exacerbate cases of AML, which could be the next focus of leukemia treatment.

Specifically, pediatric cases of AML are worsened when the protein WTAP is present. WTAP is said to contribute to abnormal cell behavior, which is how the leukemia begins and gradually worsens. When the cancerous cells begin to grow and spread they fail to develop functioning white blood cells. Sanjay Bansal, PhD, at the UT Health Science Center was the first to make the discovery.

A nationally renowned academic center, the University of Texas Health Science Center at San Antonio was previously named a National Cancer Institute Designated Cancer Center. It is a leader in developing new drugs for cancer through its participation in the development of cancer drugs that are approved by the Food and Drug Administration.

Dr. Bansal and his research team worked with leukemia cells in hopes to uncover the details of the role of WTAP. By "knocking down" WTAP expressions in leukemia cells, the researchers found that the spread of cells decreased and the differentiation of cells increased. But in order to arrive at the root of the problem, the researchers needed to find out how WTAP levels grew so high in the cells. They found this information in the behavior of the protein Hsp90.

Hsp90 helps stabilize more than 200 other proteins that are known as the protein's "clients." WTAP was found to be a member of this client list. Many of Hsp90s other client proteins are also targets in oncology.

"When we suppressed Hsp90, we reduced WTAP," Dr. Bansal said in this EurekAlert! article. "So we have discovered two things: WTAP's role in AML and the mechanism underlying its overexpression."

These findings are published in the journal Leukemia and are believed to provide further answers for treating pediatric acute myeloid leukemia, along with AML cases in newly diagnosed adults and previously diagnosed adults experiencing failing treatments.