Scientists are reporting a breakthrough discovery: A 50-cent-per-pill epilepsy drug may be used to “switch off” autism symptoms in mice, according to a new peer-reviewed study published this week in Molecular Psychiatry journal.
Autism spectrum disorder (ASD) is a complex developmental condition that impacts how an estimated one in 100 Australians perceive and socialise with others.
It is often accompanied by abnormalities such as epilepsy or hyperactivity, according to America’s Centres for Disease Control and Prevention data.
A team of experts at Germany’s Hector Institute for Translational Brain Research found that the medication lamotrigine — an anti-seizure drug approved for use in Australia — was able to curb behavioural and social problems linked to the disorder.
Now, their findings are being hyped as the closest thing yet to a potential cure for humans.
“Apparently, drug treatment in adulthood can alleviate brain cell dysfunction and thus counteract the behavioural abnormalities typical of autism,” lead researcher and cellular biologist Moritz Mall said in a statement.
“[This occurs] even after the absence of MYT1L has already impaired brain development during the developmental phase of the organism.”
Lamotrigine, which is sold under the brand name Lamictal, among others, is a medication used to treat epilepsy and stabilise mood in those who suffer from bipolar disorder.
The drug, which typically sells for about 50 cents per pill in Australia and $3 in the US, works by reversing changes to brain cells caused by a genetic mutation.
Scientists have spent years intensively searching for the molecular abnormalities that contribute to ASD and have identified MYT1L protein as one that plays a role in various neuronal diseases.
The protein is a so-called transcription factor produced by almost all the nerve cells in the body that decides which genes are or are not active in the cell. It also “protects the identity of nerve cells by suppressing other developmental pathways that program a cell towards muscle or connective tissue”.
Mutations of the protein have previously been linked to other neurological diseases and brain malformations.
To test impact of the protein on autism symptoms, researchers at HITBR genetically “switched off” MYT1L in mice and human nerve cells. They found that this led to electrophysiological hyperactivation in the mouse and human neurons impairing nerve function.
The mice lacking MYT1L suffered from brain abnormalities and showed several behavioural changes typical to ASD, such as social deficits or hyperactivity.
Researchers noted that the most “striking” reaction was the discovery that the MYT1L-deficient neurons produced extra sodium channels that are typically restricted to cells in the heart muscle.
These proteins are critical for electrical conductivity and cell function as they allow sodium ions to travel through the cell membrane. Nerve cells that overproduce these sodium channels can result in 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-associated behaviours such as hyperactivity,” the statement continued.
Clinical human trials studying lamotrigine’s impact on MYT1L are being planned — and while the research is currently limited to mice, the results are promising, researchers stressed.
This article was originally published by the New York Post and reproduced with permission
Story Credit: news.com.au