Statin may be used to treat Fragile X Syndrome

Co-Director of the Patrick Wild Centre Professor Peter Kind and colleagues have recently published a study showing that a drug named lovastatin, which is commonly used to lower cholesterol, corrected learning and memory problems in rats with a form of Fragile X Syndrome. Rats were treated with the drug for four weeks during infancy, but the benefits persisted for months afterwards.

Fragile X Syndrome is one of the most common genetic causes of intellectual disability, and is often associated with autism and ADHD. The condition occurs when a particular gene is disrupted, leading to altered communication between brain cells. Although previous studies in mice and rats provide evidence that the disruption could be treated with drugs, it is not known how long treatment might be effective for.

Researchers say that children with an inherited form of intellectual disability and autism could be helped by a similar treatment, if used early in life.

Professor Peter Kind said: “Children with Fragile X Syndrome need special education and, although some will live semi-independently, most require some form of lifelong support. We have found that early intervention for a limited period during development can lead to persistent beneficial effects, long after treatment ends, in a rat model of Fragile X Syndrome. Our future experiments will focus on whether there is a critical time-window during development when treatment is more effective.”

Original publication in Science Translational Medicine (29 May 2019)