Scientists ‘CURE autism’ in mice using $3 epilepsy drug — in potential breakthrough
- Researchers switched off MYT1L in mice and human nerve cells in the lab
- Mice without MYT1L had brain abnormalities such as a thinner cerebral cortex
- They also displayed autistic behavior including social deficits and hyperactivity
- The first clinical studies in humans are in the early planning phase
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, was able to curb behavioral and social problems linked to the disorder, which is becoming increasingly common in the US.
It is thought that the drug – which costs around $3 per pill (£2.50) – works by reversing changes to brain cells caused by a genetic mutation.
Previous studies have shown autism is more common in people who have mutations that ‘switch off’ a gene known as MYT1L.
Lead author of the study, Dr Moritz Mall from the Hector Institute for Translational Brain Research, said: ‘Apparently, drug treatment in adulthood can alleviate brain cell dysfunction and thus counteract the behavioral abnormalities typical of autism.
He added: ‘However, the results are still limited to studies in mice; clinical studies in patients with disorders from the ASD spectrum have not yet been conducted. The first clinical studies are in the early planning phase.’
Scientists are still not completely sure what the cause of autism is, though they understand it is likely a combination of genetic and nongenetic factors.
The severity of the disorder also varies greatly across the spectrum meaning there is likely no silver bullet treatment option.
MYT1L is a protein responsible for protecting the molecular identity of nerve cells, and decides which genes are active in the cells and which aren’t.
Earlier research indicated that factors influencing the molecular program of nerve cells may be involved in the development of autism.
For the latest study, researchers switched off MYT1L in mice and human nerve cells in the lab.
They found that mice lacking the protein had brain abnormalities, including a thinner cerebral cortex.
The mice also exhibited multiple signs of autism including social deficits and hyperactivity.
When used to treat epilepsy, lamotrigine works by blocking sodium channels in the body — preventing neurotransmitter release that would have otherwise caused 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, allowing just the right amount of sodium to past through.
The study’s results are only applicable to mice, meaning the drug will not necessarily work the same in humans for autism.