Cheap drug already on the market may improve autism symptoms for certain people

This news isn’t hard to swallow.Researchers at Yale University have identified a low-cost prescription drug already on the market that could help ease symptoms of autism spectrum disorder (ASD) in some people.And if you’re thinking it’s leucovorin — a vitamin B–derived drug hyped as a possible remedy — you’d be wrong.

In the study, researchers compiled a database of 774 FDA-approved drugs and tested how they affected the behavior of zebrafish genetically modified to exhibit autism-like traits.The choice of test subject may sound unusual, but these tiny tropical swimmers have become one of science’s most powerful research tools in recent years.Humans and zebrafish share about 70% of the same genes, and roughly 84% of genes linked to human disease have an equivalent in the animal, making it a powerful model for studying conditions ranging from muscular dystrophy and melanoma to epilepsy and Parkinson’s.Of the 774 FDA-approved drugs tested, the researchers narrowed the list down to 520 that were non-toxic and showed significant effects on zebrafish behavior.From that group, one drug in particular stood out as a potential candidate for treating ASD.

Sold under the brand name Carnitor, levocarnitine is currently used to treat low carnitine levels, a condition in which the body cannot properly convert fat from food into energy.But in the new analysis, the drug — which costs about 44 cents per pill — appeared to go beyond that.Researchers found that levocarnitine helped restore more normal function in zebrafish carrying mutations in two key genes tied to brain development and autism-related traits, SCN2A and DYRK1A.The drug appeared to “reverse” disruptive behaviors in the animals, while also promoting more balanced metabolism and more typical patterns of brain activity.But before you get too excited, there’s a bit of a catch.While mutations in SCN2A and DYRK1A are known risk factors for the neurodevelopmental condition, they appear to be rel...