A new drug helps treat autism

Children with autism symptoms whose cause is fragile X chromosome syndrome have been given good news by the results of two new studies. British researchers have found a drug that repairs diseased cells in lab rats with fragile X chromosome syndrome, while U.S. research shows the drug does improve socialization in human patients. Fragile X chromosome syndrome is a genetic disorder. Patients have a change in a gene on the X chromosome that causes a protein called FMRP to not be produced properly in brain cells, commonly manifesting as autism and mental retardation. Researchers at the University of Edinburgh in the United Kingdom and other institutions report in a new issue of the U.S. journal Science Translational Medicine that lab rats with fragile X chromosome syndrome had some of their physical abnormalities in their cells corrected after taking a drug code-named STX209, and they also experienced fewer external symptoms such as spasticity. A U.S. study published in the same issue of Science Translational Medicine reported that some people with fragile X chromosome syndrome who took the drug improved their scores on a social status scale and showed no major adverse effects. Professor Peter Kind, of the University of Edinburgh, said that some physical lesions in brain cells are thought to be the cause of fragile X chromosome syndrome, and the study showed that the diseased cells could be repaired with the drug and the external symptoms could be improved. This is good news for people with fragile X chromosome syndrome, whose lives are expected to change as a result of the drug.