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Children with Fragile X Syndrome could benefit from drugs used to treat high cholesterol

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Posted May 30, 2019

Fragile X Syndrome is a debilitating condition, which occurs when a particular gene is disrupted causing disturbed communication between brain cells. This is why is is one of the most common genetic reasons for mental disability, including such conditions as autism, and attention deficit and hyperactivity disorder (ADHD). Now scientists that children with Fragile X syndrome could benefit from early statin therapy.

Fragile X Syndrome is one of the most common genetic causes for learning disabilities. Image credit: Peter Saxon via Wikimedia (CC BY-SA 4.0)

The statin in question, called lovastatin, is typically used to lower the level of cholesterol. Scientists performed experiments with mice models and found that lovastatin corrected learning and memory problems. Interestingly, rats were only treated with statin for four weeks during infancy, but the benefits were long-lasting. Although, obviously, more tests with human subjects will have to be completed, now scientists are saying that children with the Fragile X Syndrome could experience the same benefits from using lovastatin early – it could prevent many learning problems.

Previous studies have shown that the disruption of the communication between brain cells in Fragile X Syndrome could be treated using drugs. However, scientists were not sure how it could be done, what kind of medicine could be used and for how long the effects would last. Previous studies were also conducted with animal models (mice and rats), but the results provided hope that learning problems as well as seizures could be prevented using pharmaceutical methods. Current medication for the Fragile X Syndrome deal with symptoms, but none of them address the underlying issues.

Scientists treated rats with lovastatin between five and nine weeks of age. However, animals were able to complete the memory tasks more than three months after treatment ended – something that was not previously possible due to the condition. This indicates that the effects of treatment are long-lasting. Professor Peter Kind, one of the authors of the study, said: “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”

Fragile X Syndrome is not easy to diagnose. Children are typically diagnosed at the age of three because they are late learning to speak. Researchers say that determining the correct time for the treatment is extremely important, but for now it looks like an early start of statin therapy could bring significant benefits.

 

Source: University of Edinburgh

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