minocycline

Researchers from UC Davis School of Medicine and Shriners Hospitals for Children – Northern California have identified a group of cells in the brain that they say plays an important role in the abnormal neuron development in Down syndrome. After developing a new model for studying the syndrome using patient-derived stem cells, the scientists also found that applying an inexpensive antibiotic to the cells appears to correct many abnormalities in the interaction between the cells and developing neurons. “We have developed a human cellular model for studying brain development in Down syndrome that allows us to carry out detailed physiological studies and screen possible new therapies,” said Wenbin Deng, associate professor of biochemistry and molecular medicine and principal investigator of the study. “This model is more realistic than traditional animal models because it is derived from a patient’s own cells.”

This preliminary clinical trial shows small but significant benefits of minocycline in children with FXS. Other treatments for FXS are being investigated, including a new class of drugs called mGluR5 inhibitors. However, minocycline is the only targeted FXS treatment that is currently available by prescription. Because of its long history of use, the side effects and safety characteristics of minocycline are well known.