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Some experts say a “cure” for autism isn’t needed. Cavan Images/Getty Images
  • Researchers are reporting that a drug used to treat epilepsy seizures shows promise as a potential “cure” for autism.
  • They report that the drug, lamotrigine, reduced autism-like behaviors in mice that were genetically engineered with human neurons.
  • Other experts, however, say the search for an autism “cure” is misguided and a better approach is behavioral and societal modifications.

In a new study, researchers say they have identified a gene that could be a key to neurodevelopmental disorders such as autism as well as a potential therapy for such conditions.

“The development of the human brain is a very complex process,” said Bettina Weigel, a study co-author and a PhD student at the German Cancer Research Center in Heidelberg, in an article accompanying the study. “This complexity renders it sensitive to disruption, which can cause neurodevelopmental defects.”

For the study, researchers used mice that were genetically engineered with human neurons to more closely understand the interplay between these genes and neurodevelopmental disorders in people.

The scientists honed in on a specific “transcription factor,” which is a protein that determines which genes are active and inactive within a particular cell.

They reported that one transcription factor in particular, MYT1L, appears to help safeguard neuron development in the human brain.

When this factor was inhibited, the mice in the study displayed some functional changes and actions typical of autism and other neurodevelopment disorders, including hyperactivity and anxiety-like behavior.

“Losing this safeguarding function… seemed to result in the development of neurons with a ‘confused’ transcriptional identity… While similar observations have previously been described for other MYT1L mouse models, our study is the first to confirm these findings in human neurons,” Weigel wrote.

Some mice were then treated with a drug approved by the Food and Drug Administration (FDA) to treat epilepsy seizures and bipolar disorder called lamotrigine, which appeared to calm some of these behaviors.

The researchers said the findings could potentially point the way to future therapy in humans — although using such drugs as a treatment for autism or other neurological conditions is a long way off.

Overall, the findings represent a step forward in understanding the human brain and how certain gene functions affect neurodevelopment.

“The discovery of MYT1L as a life-long safeguard of neuronal identity and its crucial role in normal brain function raises new exciting questions,” Weigel wrote.

We lose MYT1L as we age, she noted, and reduction in this genetic component has also been suggested to be linked to Alzheimer’s disease and other neurodegenerative diseases.

While the science of this study is crucial for developing a deeper understanding of neurodevelopment, what it doesn’t do is promise a “cure” for autism — a framing that many find misguided in the first place.

“Autism spectrum disorder is classified as a disability because of the difficulties many — not all — autistic persons experience relating to communication, interpersonal relationships, and learning,” said Üma Kleppinger, communications director of IRL Social Skills, a social skills coaching and curriculum service for neurodivergent teens.

“A great deal of medical resources are directed to finding a ‘cure’ for these differences. But the ‘disease, not difference’ model is an ableist worldview and practice,” she told Healthline.

Daniel Marston, PhD, ABPP, a psychologist and specialist in cognitive behavior therapy, agreed.

“This study is limited in that it is only one step in a very, very long process of not just trying to understand the neurology of autism but also making practical use of whatever the research shows,” Marston told Healthline. “Autism represents a complicated and multi-faceted neurological condition where the person handles the social world differently than others. Like many other things in life, there is a wide variety of ways these differences manifest themselves and sometimes these differences have to be managed. Each case needs to be considered individually and it is wrong to just decide that the brains of everyone with autism need to be changed.”

“Researchers are still trying to understand the causes and origins of many forms of autism,” explained Genevieve Konopka, PhD, a neuroscientist who studies autism at UT Southwestern’s Peter O’Donnell Jr. Brain Institute in Dallas, Texas.

“Certainly, there are individuals diagnosed with autism for whom treatments could be beneficial to improve daily life activities like sleeping and communication,” she told Healthline. “There are other individuals who have autism who may prefer not to receive any forms of treatment, whether as drugs or behavioral therapies.”

Given this, while the science of understanding is essential, it might be more helpful to consider how society can better shape itself to accommodate people with neurodivergence, said Mara McLoughlin, founder of IRL Social Skills who has autism herself.

“The overwhelming majority of funding for autism research goes to the quest for a cure and to answer the question, ‘Why do you exist?'” McLoughlin told Healthline.

“A better question would be, ‘How can we better support autistic people and their families?'” she said.