Antidiarrheal medications can help treat the underlying symptoms of autism

Currently, there are no effective treatments for the underlying symptoms of Autism Spectrum Disorder (ASD), such as difficulties in socializing and communicating. A new study uses a computerized network of protein interactions to determine if existing drugs can provide a new approach to treatment. Scientists have discovered that a common anti-diarrheal drug may have the potential to treat the social difficulties associated with ASD.

Can you teach the old drug new tricks? Although medications for the underlying symptoms of Autism Spectrum Disorder (ASD) are not currently available, could an existing drug provide a new treatment, even if it was not previously related to ASD? This question was asked by a new study in the journal Limits in pharmacology. Researchers used a computer model involving the proteins involved in ASD and how they interact.

By looking at how different drugs affect proteins in the system, they identified potential candidates for their treatment. The widely used anti-diarrheal drug called loperamide was the most promising candidate, and scientists have an interesting hypothesis about how it might work to treat ASD symptoms. Some of the most common symptoms of ASD include difficulties with social interaction and communication.

“There are currently no drugs approved to treat social communication deficits, a major symptom of 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 that have serious side effects or are not effective in ASD.”

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Changing the use of drugs for new treatment methods

In an attempt to find a new treatment for ASD, scientists turned to drug repurposing, which includes investigating existing drugs as potential treatments for another disease. This approach has many advantages as there is often extensive knowledge of existing drugs in terms of their safety, side effects, and the biological molecules with which they interact in the body.

To identify new treatments for ASD, scientists used a computer-based protein interaction network. Such networks involve proteins and the complex interactions between them. It is important to consider this complexity when studying biological systems, as interacting with one protein can often have a knock-on effect elsewhere.

Scientists constructed a protein interaction network that included ASD-related proteins. By studying existing drugs and their interactions with proteins in the network, the team identified several candidates that oppose the biological process underlying ASD.

The most promising drug is loperamide, which is commonly used to treat diarrhea. While it may seem strange that an anti-diarrheal drug can treat the underlying symptoms of ASD, scientists have developed a hypothesis about how it might work.

From digestive upset to ASD

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 the effects normally expected from an opioid drug, such as pain relief, the μ-opioid receptor also influences 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 behavior.

These results in mice highlight the tempting possibility that loperamide or other drugs targeting the μ-opioid receptor may be a novel treatment for the social symptoms found in ASD, but more work is needed to test this hypothesis. In any case, the current study shows the power of assuming that old drugs can actually learn new tricks.

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