Statins have been shown to reverse the learning deficits caused by a mutation linked to a common genetic cause of learning disabilities.
In a new study published in the journal Nature Neuroscience, researchers at the University of California-Los Angeles report that mice genetically engineered to develop the disease, called Noonan syndrome, were effectively cured of the condition after being treated with cholesterol-lowering statins.
The disorder can disrupt a child's development in many ways, often causing unusual facial features, short stature, heart defects, and developmental delays,
Medical Xpress reports.
"Noonan syndrome affects one in 2,000 people, and up to half of these patients struggle with learning disabilities," explained lead researcher Alcino Silva, professor of neurobiology, psychiatry and psychology at the David Geffen School of Medicine at UCLA.
"Our approach identified the mechanism causing the disease, as well as a treatment that reversed its effects in adult mice. We are excited about these findings, because they suggest that the treatment we developed may help the millions of Noonan patients with intellectual disabilities."
The UCLA research centered on a single gene that causes about half of all Noonan syndrome. The gene is involved in regulating a protein called Ras, which controls how brain cells talk to each other, enabling learning to take place.
When the UCLA team treated the mice with the statin lovastatin, they found that it blocked the creation of certain fat molecules that Ras requires to function — in turn, allowing brain cells to communicate properly, so that normal learning to take place.
"Noonan syndrome interferes with the changes in brain cells needed for learning, which results in learning deficits," Silva said. "Statins act on the root of the problem and reverse these deficits. This enables the process of learning to physically change the brain and create memory."
According to Silva, UCLA's approach could help the estimated 35 million Americans who struggle with learning disabilities. The next step will be to bring the drug therapy into clinical studies for Noonan.
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