Scientists discover new drug that REVERSES autism symptoms with just one dose
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Drugs used to treat seizures could reverse common signs of autism, a new study suggests.

A team of researchers from Stanford Medicine examined autism spectrum disorder (ASD) using mouse models, as this condition, now affecting one in 31 children in the US, has experienced a significant increase since the early 2000s.

The study highlighted the reticular thalamic nucleus (RT), a component of the brain’s thalamus that plays a key role in how sensory information is processed. 

Giving the mice Z944, also known as ulixacaltamide, suppressed this area of the brain, stopping the autistic symptoms.

Z944 is a drug being studied as a potential treatment for the seizure disorder epilepsy.

The researchers discovered that administering the drug to mice a single time could reverse autism-related symptoms, such as heightened sensitivity to light and sound, stimming, repetitive actions, social difficulties, and an elevated risk of seizures.

This research implies that the brain mechanisms governing both epilepsy and autism might be interconnected, potentially clarifying why individuals with autism are more prone to experiencing seizures.

It is believed that those with autism are up to 30 times likelier to develop epilepsy compared to the average person, potentially leading to a decline in cognitive abilities and contributing to speech and social difficulties over time.

Researchers at Stanford University found a drug being developed for epilepsy may quell certain signs of autism (stock image)

Researchers at Stanford University found a drug being developed for epilepsy may quell certain signs of autism (stock image)

The findings come as autism is on the rise in the US, up to one in 31 kids compared to one in 150 in the early 2000s. 

Many experts attribute the rise in autism cases to improved diagnostic procedures and increased recognition of the condition in groups that were previously underrepresented, such as girls and adults.

However, health secretary Robert F Kennedy Jr has launched a series of studies to pin down a definitive cause, suggesting pesticides, ultra-processed food and toxic metals could be to blame. 

About 3million Americans, or one in 100, are diagnosed with epilepsy, a neurological disorder that causes frequent, unprovoked seizures due to abnormal electrical activity in the brain.

While one percent of Americans have epilepsy, nearly one in three autistic people have the condition. 

It’s thought that the two conditions could be related due to shared genetic mutations and similar abnormal brain activity.

In the new study, published Wednesday in the journal Science Advances, mice underwent EEG scans, which measure electrical activity in the brain, and behavioral tests.

They were genetically modified with mutations in their CNTNAP2 gene, which has been strongly linked to autism. 

Mice with these mutations had overactive neurons in their reticular thalamic nucleus, thought to be caused by strong currents in T-type calcium channels inside the neurons.

Z944 is a T-type calcium channel antagonist, meaning it blocks these currents.

Mice with genetic mutations showed less interest in social interactions, more repetitive grooming and hyperactivity. They were also more prone to seizures.

However, mice who were given Z944 just once had decreases in repetitive behaviors and social isolation. They were also less hyperactive. 

But when researchers modified the mice to have more activity in the reticular thalamic nucleus, the autistic behaviors returned. The findings suggest Z944 quiets this region by blocking T-type calcium channel activity.

The researchers noted Z944 could be used to treat both autism and epilepsy. However, the drug is not yet available for general use and is still undergoing clinical trials for epilepsy.

It’s also unclear how these findings would translate to humans.

The researchers wrote: ‘Future research should aim to elucidate how RT-mediated circuit dynamics throughout the brain influence the broader neurobehavioral landscape of ASD, paving the way for circuit-specific, precision interventions.’ 

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