Cholesterol Medicine May Help Children With Fragile X

Early Statin Treatment Could Yield Long-term Benefits

Researchers from the University of Edinburgh report that lovastatin (Mevacor, Altocor, etc.) corrected learning and memory problems in rats with a form of Fragile X Syndrome.

The rats received lovastatin for four weeks during infancy but the benefits continued for months afterward. The researchers suggest that learning problems in children with Fragile X could be prevented by a similar treatment during their early life.

Fragile X Syndrome is a common genetic cause of intellectual disability. It is often associated with autism and attention deficit and hyperactivity disorder, and also causes seizures in many patients. The condition is caused by the disruption of a particular gene, which leads to altered communication between brain cells. 

Children are typically diagnosed at around 3 years old, often because of their delayed speech. However, as genetic tests have enabled earlier diagnosis, treatment could potentially begin sooner. Patients with Fragile X require special education and most will need some type of lifelong support.

Although previous studies in mice and rats indicated that the gene disruption can be treated with drugs, it was not known for how long the treatment might remain effective.

The study included rats with a genetic alteration similar to the one in people with Fragile X Syndrome. Compared with typical rats, these rats have difficulty completing certain memory tasks.

After treatment with lovastatin between five and nine weeks of age, the rats’ normal development was restored. The animals were able to complete memory tasks more than three months after treatment ended, indicating that the drug’s effects were long-lasting.

The researchers plan to investigate whether there is a critical period during development when treatment is more effective. The study was published in Science Translational Medicine.

Source: Edinburgh University, May 30, 2019