Combating Blood Cancer

Over the past 50 years, survival rates for a group of blood cancers known as myelodysplastic syndrome (MDS) have not improved, with about 10,000 people in the U.S. dying from it annually. Now, researchers at The University of Texas at Austin, MD Anderson Cancer Center and the Montefiore Einstein Comprehensive Cancer Center have discovered a combination drug therapy that yielded promising results in a clinical trial.

“Cell metabolomics is a promising area of study in cancer research, particularly for hard-to-treat cancers like MDS,” said Stefano Tiziani, professor of nutritional sciences, oncology and pediatrics at UT Austin and a corresponding author on the study in the journal Nature Cancer. “It uncovers the metabolic dependencies that cancer cells rely on for survival. This approach opens new therapeutic avenues by attacking cancer’s altered metabolism, offering hope for more effective treatments.”

MDS cancer cells, as well as cells in certain other cancers, rely on metabolizing glutamine, the most abundant amino acid in the blood. The team realized that an investigational drug, CB-839, might weaken MDS cancer cells because it targets the enzyme glutaminase, which catalyzes the first step in glutamine metabolism.

In a clinical trial, the researchers treated 28 patients with advanced MDS with a combination of CB-839 and azacytidine, a drug that kills rapidly dividing cells and is typically used to treat MDS. In 70% of patients, tumors either shrank or disappeared. After analyzing blood samples following treatment, the researchers found that the combined therapy helped patients produce normal blood cells, a process often disrupted in MDS.

“This approach opens new therapeutic avenues by attacking cancer’s altered metabolism.”