In what could signal a turning point, researchers say they’ve identified a drug compound that may address two key facets of autism — repetitive behaviors and socialization.
The compound known as GRN-529 is showing significant promise when tested in mice, giving researchers hope that it could lead to a biomedical treatment for people with autism.
In a study published Wednesday in the journal Science Translational Medicine, researchers tested the compound on a strain of mice known to exhibit autism-like behaviors. They found that mice that received the treatment spent less time repeatedly grooming themselves and more time sniffing noses with other mice as compared to mice who did not receive the drug.
While treatments that are successful in animals don’t always have comparable results in people, scientists say they are particularly optimistic about the potential for this compound. That’s because it is similar to a drug that’s already in clinical trials for treatment of fragile X syndrome, which has many overlapping features with autism.
“Our findings suggest a strategy for developing a single treatment that could target multiple diagnostic symptoms,” said Jacqueline Crawley of the National Institute of Mental Health who worked on the study.
Perhaps even more significant, the findings could alter the way autism is perceived by offering evidence that core symptoms of the disorder may be treatable with medication, the researchers said.
“While autism has been often considered only as a disability in need of rehabilitation, we can now address autism as a disorder responding to biomedical treatments,” said Thomas Insel, director of the National Institute of Mental Health.
Currently the only drugs approved specifically for autism are Risperdal and Abilify, antipsychotics that are designed to address irritability associated with the disorder rather than its core issues.