Children with autism and Fragile X could be helped by statins

Researchers' work aimed at helping children with Fragile X.
Researchers' work aimed at helping children with Fragile X.
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Children with an inherited form of intellectual disability and autism could be helped by a medicine commonly used to lower cholesterol, if used early in life, a study from the University of Edinburgh suggests.

The drug – lovastatin – corrected learning and memory problems in rats with a form of Fragile X Syndrome, tests revealed.

Rats were treated with lovastatin for four weeks during infancy but the benefits persisted for months afterwards.

Scientists say this suggests learning problems in children with Fragile X might be prevented by a similar treatment in early life.

Fragile X Syndrome is one of the most common genetic causes of intellectual disability. It is often associated with autism and attention deficit and hyperactivity disorder, or ADHD. Many individuals also have seizures.

Current medications help manage specific symptoms – such as hyperactivity and seizures – but there are not yet any treatments that tackle the underlying brain changes leading to Fragile X Syndrome.

Statins are widely prescribed to children and adults to control high blood cholesterol and reduce the risk of heart disease.

The study is published in Science Translational Medicine, was led by researchers at the university's Patrick Wild Centre and the Simons Initiative for the Developing Brain.

Professor Peter Kind, director of the university's Patrick Wild Centre and Simons Initiative for the Developing Brain at the University of Edinburgh, 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.”