Epilepsy Drug Could Help Kids With Autism, Study Shows

The new study marks an important breakthrough in autism research.

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A new study out of the German Cancer Research Center suggests an epilepsy drug may help alleviate behaviors observed in people with autism. Autism Spectrum Disorder (ASD) is a developmental disability that can cause social, behavioral and communication challenges. According to the Centers for Disease Control (CDC), about 1 in 44 children fall on the autism spectrum. Currently, there are no medications to treat ASD.

The new study was conducted on mice with a MYT1L gene defect. It focused on the long-used epilepsy medicine Lamotrigine, which is used to stop seizures. It can also be used to treat bipolar disorder. In the mice, electrical activity in the brain returned to normal, and behavioral challenges associated with autism, like hyperactivity, were alleviated. Though the study has not yet progressed to human trials, it represents a potential breakthrough in autism treatment.

The Connection Between An Epilepsy Drug And Autism

In a press release from the German Cancer Research Center, scientists noted a specific gene transcription, MYT1L, protects how certain nerve cells operate. (Gene transcription is defined as how genes get written in the body.) The researchers’ assertion is the MYT1L transcription factor works less effectively in people with autism than in the rest of the population.

In people with autism, because the MYT1L factor does not operate properly, it causes a variety of behavioral challenges. In the study, mice with MYT1L insufficiencies were more hyperactive and did not interact with other mice as often as neurotypical mice. They also appeared to be more anxious than their counterparts without the MYT1L insufficiency.

However, the mice treated with Lamotrigine did not demonstrate these behavioral challenges, despite the MYT1L mutation. This suggests the medication might be useful in treating autism in people.

What Is Autism?

Autism is a developmental disorder that impacts a person's entire life. Social interaction, communication skills, and executive function are often impacted, explains Tiffanie Moore, Senior Vice President of Clinical Services at BlueSprig Pediatrics, which focuses on treating autism. "Individuals with this diagnosis may have restricted interests and forms of repetitive behavior. It is important to know that the behaviors that characterize this diagnosis present differently across individuals," Moore says.

Because autism is a lifelong condition that causes varying challenges, parents may wonder about what "cures" may exist. But Moore explains autism should not be viewed as an illness, but rather as a unique way of thinking, learning, and interacting.

Current Treatments For Autism

Currently, the American Academy of Pediatrics (AAP) recommends something called "complementary and alternative therapies" to help those with autism. Some examples of this include yoga, specific diets, or acupuncture. Michael Cummings, M.D., a child and adolescent psychiatrist at BlueSprig Pediatrics, says treatments are limited to early detection, which relies on parents and caregivers to report delays or concerns to pediatricians. Early speech and language interventions, occupational and physical therapy, and behavioral interventions are all also options.

At this point, there are no pharmaceutical interventions. "It is important to realize that, to date, there are no medications that specifically target the core symptoms or behaviors associated with autism," says Dr. Cummings. This is why the breakthrough in the new MYT1L study is so promising. "Essentially all funded autism research has been geared at identifying genetic causes of the condition."

Limitations of Epilepsy Drug Study

While this new MYT1L study is promising, it's important to remember there are more trials that must be completed before the epilepsy medication is recommended to children and adults with autism, says Dr. Cummings. Autism, like other behavioral disorders, exists on a spectrum—not just in behavior, but also in genetic presentation. While this particular MYT1L gene expression may be representative of some of the population with autism, there's a chance autistic people without this mutation exist.

"We must remember that autism is not one neurodevelopmental condition, but potentially hundreds or thousands of conditions with similar appearances or 'phenotypes,'" says Dr. Cummings. "As such, even if this agent is found to be useful in reversing symptoms of autism, it is likely that it would only be effective on children with that specific genetic variant, while not necessarily being helpful in others."

Additionally, because autism is a condition rather than an illness, Moore explains that parents should not view individuals with it as broken or otherwise deficient. "When considering supports needed, the focus is to reduce perceived barriers for the individual—not to change them or 'cure' them of differences," Moore says.

Related: Yes, Kids With Autism Can Be Creative! (But You Knew That!)

Psychiatric medications may be used to treat "behaviors" associated with autism, but medication can often be overused, he continues. Because the MYT1L drug research is so new and in its early stages, Dr. Cummings cautions that researchers do not yet know how applicable this medication will ultimately be.

However, this could be a significant breakthrough in autism genetics, and much can be learned from the study's research.

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  1. Centers for Disease Control and Prevention. 2022. Autism Spectrum Disorder (ASD).

  2. Prevalence and Characteristics of Autism Spectrum Disorder Among Children Aged 8 Years. Morbidity and Mortality Weekly Report - CDC. 2021.

  3. Molecular Psychology. 2023. MYT1L haploinsufficiency in human neurons and mice causes autism-associated phenotypes that can be reversed by genetic and pharmacologic intervention.

  4. American Academy of Pediatrics (AAP). 2022. Complementary & Alternative Therapies for Autism.

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