In Science Translational Medicine this week, an international team of scientists reports the discovery of a potential new genetic target for treating urothelial bladder cancer. The researchers discovered that such cancers harboring common KDM6A mutations are dependent on the DNA-modifying protein EZH2 and that inhibiting this protein killed KDM6A mutant cancer cells in vitro. In mouse models of the disease, EZH2 inhibition reduced tumor volume by one third after just two weeks of treatment with no apparent toxicity. Notably, inactivating mutations in KDM6A have also been observed in several other cancers types.
Also in Science Translational Medicine, a group of German investigators present data indicating changes in infants' immune systems can predict which child will go on to develop type I diabetes. The researchers followed a cohort of children from birth who were at high familial risk for the disease but only half of whom developed type I diabetes. They discovered a divergent population of immature T cells with unique gene expression patterns that could be identified as early as six months of age and before the cells began attacking the pancreas. The investigators suggest that the events preceding type I diabetes are likely a combination of genetic and environmental processes that begin in infancy.