Anti-diarrhea drug repurposed to treat autism symptoms
Norwegian researchers at the University of Oslo have found possible treatment for the symptoms of autism spectrum disorder (ASD) in a common anti-diarrhea drug called loperamide. The drug may be dually beneficial in treating patients with ASD – it can both modulate problematic social behaviour as well as alleviate gastrointestinal symptoms.
“There are no medications currently approved for the treatment of social communication deficits, the main symptom in ASD,” said Dr. Elise Koch, from the University of Oslo. “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.”
The researchers experimented with novel computer modelling to stimulate complex protein interactions. Using this, a network modelling proteins associated with ASD was constructed; and the influence of pre-existing drugs was then modelled on the network, with curious results pointing to several molecules that could counteract the processes underlying ASD.
It led to the discovery of loperamide, a common anti-diarrheal drug that has been in use for over 50 years. Loperamide is an opiate that targets μ-opioid receptor proteins; loperamide only works on μ-opioid receptor activity in the gut, since it cannot cross the blood-brain barrier.
This significant mechanism of action offers intriguing clues to designing potential future ASD therapies.
In prior experiments, breeding μ-opioid-receptor-deficient mice led to the animals displaying behavioural deficits that resembled ASD.
“The identification of loperamide as a drug repurposing candidate for ASD is consistent with its potential to modulate social behaviour in ASD,” the researchers note. “In addition, it may have favorable gastrointestinal effects in individuals with ASD, as gastrointestinal symptoms are common in ASD.”
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