Have Scientists Discovered a Pill That Cures Autism?

A new study found that lamotrigine, a medication used to treat epilepsy and symptoms of bipolar disorder, reversed autism-like behaviors in mice.

There is no known cure for autism spectrum disorder (ASD). However, medications can help manage some of the symptoms associated with the condition. These include antipsychotic drugs, such as risperidone and aripiprazole, antidepressants, anti-anxiety medications, and anticonvulsant drugs.

Still, none of these drugs cure autism, and not all autistic individuals benefit from medication.

However, in a study published on February 14 in the journal Molecular Psychiatry, scientists from the Hector Institute for Translational Brain Research (HITBR) discovered that lamotrigine (Lamictal) — an epilepsy drug that blocks sodium channels — reversed autistic-like behaviors in mice. According to the study authors, this discovery could lead to effective therapeutic options for people with ASD.

Based on previous knowledge that mutations in a protein in nerve cells called MYT1L play a role in other neurological disorders, the scientists wanted to investigate what role it might play in the development of autism.

When the team genetically turned off MYT1L in mice, the rodents showed behavior and gene expression changes similar to ASD. The mice also exhibited thinner cerebral cortexes.

Additionally, the scientists also observed impaired nerve function when they switched off MYT1L in human nerve cells.

What’s more, the MYT1L-deficient neurons produced a surplus of sodium channel blockers typically only found in heart muscle. When a nerve cell produces an excess of these channel blockers, it can result in electrophysiological hyperactivation — a symptom observed in autistic individuals.

However, when the team treated MYT1L-deficient human nerve cells and mice with lamotrigine, a sodium channel blocker, the hyper activation returned to normal in the cells and autism-like behaviors in mice ceased.

"Apparently, drug treatment in adulthood can alleviate brain cell dysfunction and thus counteract the behavioral abnormalities typical of autism — even after the absence of MYT1L has already impaired brain development during the developmental phase of the organism,” study author Moritz Mall explains in a news release.

Although the results in mice are promising, clinical trials using humans with ASD are only in the planning stages. Still, if human studies show similar results, the researchers suggest targeting MYT1L deficiency with lamotrigine could be a potential therapeutic option for autistic individuals.