Scientists have learned to 'turn off' the symptoms of autism with an inexpensive drug

Scientists report groundbreaking discovery: A $3-a-pille epilepsy drug can be used to 'turn off' autism symptoms in mice, according to a new study. Writes about it New York Post.

Autism Spectrum Disorder is a complex developmental disorder that affects how approximately 5,4 million (2,2%) adults—and one in 44 children—in the United States perceive and interact with other people. According to the Centers for Disease Control and Prevention (CDC), this is often accompanied by conditions such as epilepsy or hyperactivity.

A team of experts from the German Hector Institute for Translational Brain Research found that lamotrigine, an anticonvulsant drug first approved for use in the US in 1994, is able to curb the behavioral and social problems associated with the disorder.

Now their discoveries are being touted as the closest thing to a potential cure for humans.

"It is clear that drug treatment in adulthood can alleviate brain cell dysfunction and thus counteract the behavioral abnormalities typical of autism," lead researcher and cell biologist Moritz Moll said in a statement. "This occurs even after the absence of MYT1L has already disrupted brain development during the organism's developmental stage."

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Lamotrigine, which is sold under the brand name Lamictal among other things, is a medication used to treat epilepsy and stabilize mood in people suffering from bipolar disorder.

The drug, which typically sells for just under $3 a pill, works by reversing changes in brain cells caused by a genetic mutation.

Scientists have spent years intensively searching for molecular abnormalities that contribute to autism spectrum disorders (ASD) and have identified the MYT1L protein as a protein that plays a role in various neuronal diseases.

The protein is a so-called transcription factor produced by almost all nerve cells in the body that determines which genes are active and which are not in the cell. It also "protects the identity of nerve cells by inhibiting other developmental pathways that program the cell into muscle or connective tissue."

Protein mutations have previously been associated with other neurological diseases and brain malformations.

To test the protein's effect on autism symptoms, researchers at HITBR genetically "turned off" MYT1L in mice and human nerve cells. They found that this led to electrophysiological hyperactivation of mouse and human neurons, impairing nerve function.

Mice lacking MYT1L suffered from brain abnormalities and exhibited several behavioral changes typical of ASD, such as social skill deficits or hyperactivity.

The researchers noted that the most "striking" response was the discovery that MYT1L-deficient neurons produce additional sodium channels that are normally restricted to heart muscle cells.

These proteins are critical for electrical conductivity and cell function as they allow sodium ions to pass through the cell membrane. Nerve cells that overproduce these sodium channels can lead to electrophysiological hyperactivation, a common symptom of autism.

“When MYT1L-deficient nerve cells were treated with lamotrigine, their electrophysiological activity returned to normal. In mice, the drug was even able to curb ASD-related behaviors such as hyperactivity,” the statement said.

These promising results come against the backdrop of a skyrocketing incidence of autism in urban areas of New York. Autism diagnoses have tripled in the New York-New Jersey metro area, from 1% of the population in 2000 to 3% in 2016.

Part of the dramatic increase in these diagnoses is believed to be due to the growing number of diagnoses in children without mental retardation, which are therefore less likely to be detected earlier.

But earlier, more accurate diagnoses did not fully explain the upward trend that was based on CDC estimates. Experts warn that the growing trend of women to give birth later in life may be partly responsible for this increase.

Meanwhile, human clinical trials investigating the effects of lamotrigine on MYT1L are being planned — and while research is currently limited to mice, the results are promising, the researchers stressed.