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Metabolism Harnessed

Published: 2017-05-17 |

Source: University of Georgia

University of Georgia researchers, with colleagues from the University of Tokyo, have identified a new drug target for the two most common types of myeloid leukemia, including a way to turn back the most aggressive form of the disease. They published their findings today in the journal Nature.

By blocking a protein called BCAT1, the researchers were able to stop cancer growth in mice and human blood samples from leukemia patients. The BCAT1 protein activates the metabolism of a group of amino acids known as branched-chain amino acids, or BCAA's, that are essential building blocks of proteins in all cells and thus necessary for aggressive leukemia cells to grow. The same enzymes are also responsible for the development of brain and lung tumors.

Earlier research indicated that BCAT functions to break down the BCAAs in most healthy tissues. The new paper shows for the first time that, rather than break down, leukemia cells use the BCAT1 pathway to produce BCAAs. By blocking the protein, researchers can reverse the disease's aggressiveness.

"We wanted to understand what is driving aggressiveness in acute leukemia, and then examine whether targeting such a pathway would reverse the disease back to a treatable phase," said Takahiro Ito, senior author on the paper and assistant professor in the Franklin Collage of Arts and Sciences department of biochemistry and molecular biology.

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