Antidiarrheals may help treat autism

Autism spectrum disorder (ASD) is a heterogeneous group of neurodevelopmental phenotypes characterized by social and communication deficits along with restrictive behaviors accompanied, most of the time, by psychiatric comorbidities such as sleep problems, anxiety, depression, ADHD, or aggressiveness and irritability. 

While there are currently no approved medications for the treatment of social communication deficits, the main core symptom in ASD, most adults and approximately half of children and adolescents are treated with psychotropic drugs to reduce non-core symptoms. such as irritability, hyperactivity, and self-injurious behavior. 

A study from the University of Oslo, Norway, used a protein interaction network to identify whether existing drugs could provide a new treatment approach. The researchers found that a common antidiarrheal drug may have the potential for treating the social difficulties associated with the disorder: loperamide. 

Loperamide binds to and activates a protein called the μ-opioid receptor, which is usually affected by opioid drugs, such as morphine. In addition to its normally expected effects, such as pain relief, the μ-opioid receptor also affects social behavior.

In previous studies, genetically modified mice lacking the μ-opioid receptor showed social deficits similar to those seen in ASD. Interestingly, drugs that activate the μ-opioid receptor helped restore social behaviors.

These results in mice highlight the tantalizing possibility that loperamide, or other drugs targeting the μ-opioid receptor, may represent a new way to treat the social symptoms present in ASD, but more work is needed to test this hypothesis.

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