Loperamide, a common anti-diarrhea drug, may treat the social symptoms of autism. The corresponding study was published in Frontiers in Pharmacology.
Autism spectrum disorder (ASD) is a neurological and developmental disorder that affects key aspects of behavior, communication, and learning. While it can be diagnosed at any age, it is considered a 'developmental disorder' as symptoms typically become apparent within the first two years of life. Multiple factors are thought to contribute to the condition, including genetics and environmental factors.
“There are no medications currently approved for the treatment of social communication deficits, the main symptom in ASD,” said Dr. Elise Koch of the University of Oslo, lead author of the study. “However, most adults and about half of children and adolescents with ASD are treated with antipsychotic drugs, which have serious side effects or lack efficacy in ASD.”
In the current study, the researchers searched a computer-based protein interaction network to identify drug candidates that may counteract the biological processes underlying ASD. In the end, they identified four potential drugs with the potential to treat the condition.
The most promising of these drugs is known as Loperamide, and is a common anti-diarrheal medication that binds to and activates the μ-opioid receptor. Normally affected by opioid drugs such as morphine, the drug is known both to induce pain relief and affect social behavior.
The researchers noted that previous research found that mice genetically engineered to lack the μ-opioid receptor had similar social deficits to those with ASD, and that drugs that activated the receptor restored social behaviors.
The researchers wrote that while their study identified potential new drugs for autism, further research is needed to test them. They concluded: “Based on our bioinformatics analyses of ASD genetics, we shortlist potential drug repurposing candidates that warrant clinical translation to treat ASD-specific symptoms.”