As many parents already know, medications for managing autism spectrum disorder (ASD) can cause challenging side effects. For the common symptoms of irritability and agitation, only two drugs are FDA approved—risperidone and aripiprazole—and unfortunately both can cause intense food cravings that lead to weight gain.
The drugs are effective at reducing tantrums and aggression, so they are critical for treatment. However, children as young as five years old are now starting on the regimen, meaning some will face a decades-long struggle against obesity. Fortunately, it now appears a medication long used to treat Type 2 diabetes may provide a solution.
Testing a new treatment
In a recent study published in the Journal of the American Medical Association, my collaborators and I began a clinical trial for the drug metformin. It is the first metformin trial to look at potential benefits for children with autism. While no physician wants to use more medicine than absolutely necessary, the benefit of the added drug seems to counter excess weight gain, with limited risk for further side effects.
Metformin works by helping the body more efficiently use insulin to digest glucose, while dulling the body’s mechanisms for manufacturing or absorbing the sugar. For patients with Type 2 diabetes, the lower glucose levels help manage their disease while countering weight gain.
To see if metformin may yield a similar benefit for children with autism, we began a double-blind, placebo-controlled, randomized clinical trial at our four institutions: The Ohio State University Wexner Medical Center, the University of Toronto (the coordinating center), the University of Pittsburgh and Vanderbilt University.
We recruited participants from the Autism Speaks Autism Treatment Network and 60 have completed the first stage of the trial. With ages ranging from ages 6-years-old to 17-years-old, all of the participants had been showing signs of weight gain after starting on ASD medications.
Because children with ASD are not always able to communicate how they feel, our study also focused on careful evaluation of any adverse metformin effects. Through a series of periodic, structured observations and several well-established surveys for patients and parents alike, we were able to assess safety and tolerability with promising results.
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Our trial ran for 32 weeks; our findings from the first 16 weeks provided enough encouraging data to result in publication. In just the first 8 weeks, we observed that a significant number of patients taking metformin lost weight, with lower body mass index (BMI) scores. Over the full 16-week period, patients on the metformin lost an average of six pounds.
Managing benefits and risks
Autism is a complex condition, and any new treatment must be considered in relation to any new risks. There are no “silver bullets” for managing ASD.
While we did find that patients tolerated metformin well, our study was relatively small. The most common side effect we’ve observed is gastrointestinal discomfort, ranging from diarrhea to nausea–though the effects have tended to be mild and did not appear to interfere with eating habits.
We also had three patients discontinue treatment as a result of increased agitation possibly related to the medication, an observation we are monitoring closely. Such impacts appear to be minimal relative to the outcome: children taking a placebo continued to gain weight, while children on metformin tended to lose weight.
Further observation will reveal how that trend manifests over time, and we hope future studies will incorporate larger samples and additional weight-management tools, such as lifestyle and diet changes. However, we have been encouraged by what we are seeing—as with many other ASD challenges, we are hopeful that weight gain will soon become one that modern treatment can manage.
This article was featured in Issue 60 – Sensory Tools For The Future