Researchers publish breakthrough in reversing leukemia

Today in the journal Nature, a UGA research team led by Takahiro Ito published important new work that identifies 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:

By blocking a protein called BCAT1, the researchers were able to stop cancer cell 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 BCAAs, 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 to break them 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 the treatable phase," said Takahiro Ito, senior author on the paper and assistant professor in the Franklin College of Arts and Sciences department of biochemistry and molecular biology.

With the help of colleagues in Japan, Ito's team was able to test the drug and the genetic technique on human tissue. The fundamental idea behind this approach is genuinely new and counter to established thinking on the role of BCAT1 in this context. The team worked with GRA Eminent Scholar Arthur Edison in the NMR lab at the CCRC to prove their new theory and verify the work. Tremendous collaboration that will have far-reaching implications for patients around the world. Congratulations to Dr. Ito and the team on this important publication.

Image: assistant professor Takahiro Ito