Researchers Are Testing a New Medicine That Could Treat Autism Symptoms

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A new drug that strives to treat the symptoms of autism is currently in the process of being studied. It’s called NitroSynapsin and it’s an aminoadamantane nitrate compound that aims to “restore an electrical signaling imbalance in the brain found in virtually all forms of autism spectrum disorder.”

Research of this drug is being led by Dr. Stuart Lipton, a clinical neurologist and professor at The Scripps Research Institute. He is also working with researchers from various other universities, including the University of California, Scintillon Institute, and San Diego School of Medicine.

Though research on this medicine was newly published, the history behind it goes back all the way to 1993, when Lipton, then a researcher at Harvard Medical School, found that a gene called MEF2C played a role in brain development. He and his colleagues found that disrupting this particular gene in mice fetuses resulted in them developing autism-like traits.

Adobe Stock/Ermolaev Alexandr

Adobe Stock/Ermolaev Alexandr

This kind of phenomenon isn’t unique among mice; other researchers have found that it occurs in humans. When one of the two copies of the gene (one from mom and the other from dad) gets mutated, it results in a condition called MEF2C Haploinsufficiency Syndrome, or MHS for short. The characteristics of this disorder look very similar to those of the mice with the disrupted MEF2C gene.

In mice and humans, MHS is characterized by traits like repetitive behaviors, struggles with spatial memory, and high anxiety. In mice models with the condition, it was also found that their brains had an imbalance of excitatory and inhibitory signals—in other words, neurons were being directed to fire away too much, and the signals that were supposed to suppress these neurons were reduced.

Adobe Stock/Kirill Kurashov

Adobe Stock/Kirill Kurashov

When these genetically modified mice were given NitroSynapsin for three months, however, their symptoms markedly improved—some of them even had their “performance [restored] essentially to normal.”

It should be noted that only a small number of autism cases are believed to involve MHS. However, researchers are confident that it will translate onto a variety of ASD cases. “Because MEF2C is important in driving so many autism-linked genes, we’re hopeful that a treatment that works for this MEF2C-haploinsufficiency syndrome will also be effective against other forms of autism,” said Lipton.

But before you get too excited about this, remember that the medication still needs to go to human clinical trials. So if it even works on people, it will be a while before it’s available. Still, it’s one of the many medications for autism being tested in recent years. Hopefully this one finds success!

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A. Stout received a Bachelor of Arts in Writing through Grand Valley State University, graduating Magna Cum Laude in 2015. In addition to being a passionate autism advocate, she is a member of various fandoms, a study abroad alumna, and an animal lover. She dreams of publishing novels and traveling all over the world someday.