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 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. By blocking the BCAT1 gene, the research team was able to make the disease less aggressive and slow growing, similar to the treatable chronic phase.
The findings suggest that BCAT1 may be an ideal therapeutic target that does little harm on normal blood production. Furthermore, results indicated that BCAT1 is also a key player in acute myeloid leukemia, which is more prevalent than the blast crisis chronic myeloid leukemia. Patients with AML with high BCAT1 tend to survive less than those with low BCAT1. Blocking the BCAT1 activity also proved effective on human acute myeloid leukemia cells.
The researchers worked with colleagues at the Complex Carbohydrate Research Center Nuclear Magnetic Resonance facility, the UGA Cancer Center, the College of Veterinary Medicine, and the University of Tokyo in Japan.
An online version of the full study is available here.