Scientists ‘CURE autism’ in mice with $3 epilepsy drug – potential breakthrough
- Researchers switched off MYT1L in mice and human nerve cells in the laboratory
- Mice lacking MYT1L had brain abnormalities such as a thinner cerebral cortex
- They also displayed autistic behaviors, including social deficits and hyperactivity
- The first human clinical trials are in the early planning stages
A cheap epilepsy drug has cured autism-like symptoms in mice for the first time – in what could be a breakthrough.
Lamotrigine, brand name Lamictal, has been shown to curb behavioral and social problems associated with the disease, which is becoming more prevalent in the United States.
The drug – which costs about $3 a pill (£2.50) – is believed to work by reversing changes in brain cells caused by a genetic mutation.
Previous studies have shown that autism is more common in people who have mutations that “turn off” a gene known as MYT1L.
The lead author of the study, Dr. Moritz Mall of the Hector Institute for Translational Brain Research, said: “Apparently, drug treatment in adulthood can alleviate dysfunction of the brain cells and thus counteract the behavioral problems typical of autism.
He added: “However, the results are still limited to studies in mice; clinical trials in patients with ASD spectrum disorders have not yet been conducted. The first clinical studies are in the early planning phase.’
Scientists are still not entirely sure what causes autism, although they understand it’s likely a combination of genetic and non-genetic factors.
The severity of the disorder also varies widely across the spectrum, meaning there is likely no miracle cure treatment option.
MYT1L is a protein responsible for protecting the molecular identity of nerve cells and deciding which genes are active in the cells and which are not.
Previous research has suggested that factors affecting the molecular programming of neurons may be involved in the development of autism.
For the latest study, researchers turned off MYT1L in mice and human neurons in the lab.
They found that mice lacking the protein had brain abnormalities, including a thinner cerebral cortex.
The mice also displayed several signs of autism, including social deficits and hyperactivity.
In the treatment of epilepsy, lamotrigine works by blocking the sodium channels in the body, preventing the release of neurotransmitters that would otherwise cause seizures. Almost two million people in the US were prescribed the drug in 2020.
In autism, the drug is thought to partially block sodium channels, letting just the right amount of sodium through.
The results of the study only apply to mice, meaning the drug might not work the same way in humans for autism.