Autism symptoms can now be eliminated with a single injection

Autism is one of the most mysterious and difficult-to-understand brain conditions. According to the WHO, it affects one in every 100 children worldwide, and in the US, approximately one in 30. Despite decades of research, the causal mechanisms are not fully understood, and there is still no effective medication capable of eliminating the symptoms. However, new research by scientists at Stanford School of Medicine has given hope — they have shown that just one injection of an experimental drug can completely eliminate the manifestations of autism. This result could be a breakthrough in understanding how the brains of people with autism spectrum disorders work and bring medicine closer to developing effective treatments.

For the first time, scientists have been able to completely eliminate the symptoms of autism.

How the new drug works

It has long been known that autism is a mental disorder associated with brain development abnormalities. However, it was not fully understood how these abnormalities manifested themselves. In a recent study, scientists focused on a specific structure, namely the reticular-thalamic nucleus, in a region of the brain known as the thalamus. This structure is responsible for processing sensory information. It was in this region of the brain that excessive neuronal activity was observed in mice with autism. The researchers used the drug Z944 (ulexacaltamide), which was originally developed to treat epilepsy. It blocks calcium channels in neurons and reduces their excitability.

The effect was remarkable—after just one injection, the animals’ key symptoms of autism disappeared. The mice stopped overreacting to light and sound, reduced the number of repetitive movements and stereotypes, and became more social. The tendency to have seizures, which often accompanies autism, also decreased.

The development of autism and epilepsy is based on common mechanisms

The discovery was particularly significant because autism and epilepsy often go hand in hand. It is known that up to a third of people with autism suffer from epilepsy, and their risk of developing it is dozens of times higher than that of ordinary people. This impairs cognitive function and can lead to regression in speech and social skills.

One in every hundred children worldwide is diagnosed with autism.

The results of the study confirm that the two conditions do indeed have a common biological basis. Pathological activity in the same brain circuits can explain both sensory overload and seizures. Therefore, Z944 has the potential to become a universal treatment for both disorders.

The experiment used mice with a mutation in the CNTNAP2 gene, one of the key genes associated with autism in humans. Animals with this mutation exhibited typical behavioral signs — increased anxiety, avoidance of social contact, and repetitive actions. As mentioned above, electroencephalograms showed that their thalamic nucleus neurons were overly active and their calcium channels were working too intensely.

When scientists suppressed this activity with Z944, the symptoms disappeared. But as soon as this area of the brain was stimulated again, the autistic behavior returned. This proves that it is the reticular-thalamic nucleus that plays a key role in the development of autism.

After the injection, the mouse’s behavior changed and no longer showed signs of autism.

However, despite the encouraging results, these are still only laboratory experiments. Z944 has not yet passed all clinical trials even as an antiepileptic drug, let alone for use in autism. Scientists emphasize that it is unknown how successful the effect will be in humans.

Nevertheless, the discovery opens up new horizons for science. It shows that autism can be viewed not as an incurable developmental disorder, but as the result of specific dysfunctions in the brain’s neural circuits. In the future, this could lead to the creation of targeted therapies that act on specific areas and channels of the brain.

The study shows that epilepsy and autism may have common roots, which means that approaches to their treatment may overlap. The very possibility of “turning off” symptoms with just one injection gives hope to millions of families around the world.