Autism breakthrough: New drug may ‘turn down’ social behavior problems

Autism breakthrough: New drug may ‘turn down’ enzyme that hijacks social behavior in people on the spectrum

  • About 1.5 million people are on the autism spectrum 
  • Some people with ASD are independent, but others struggle to interact socially, develop repetitive behaviors and cannot take care of themselves 
  • There are not drugs that target the core symptoms of autism
  • Scientists at Florida Atlantic University found genetic mutations that change serotonin levels in people on the autism spectrum 
  • Serotonin is key to social behaviors  
  • A certain enzyme sweeps up too much serotonin in the brains of people with ASD
  • But a drug developed by Northwestern University ‘turned down’ that enzyme when the Florida researchers tried it in mice 

A treatment for adults on the autism spectrum may be in sight, thanks to a new discovery.

After decades of research, scientists at Florida Atlantic University (FA) have discovered a new pathway in the brain linked to behavioral symptoms of autism – and a drug that may quell them. 

Some autism advocates have increasingly been pushing for people on the spectrum to not be treated as ‘disordered,’ but rather as simply different. 

On the other hand, the FAU researchers note that autism also frequently comes with physiological problems – particularly for the gut. 

There are currently no treatments for autism in adults, but the team discovered a drug treated the behavioral ‘disruptions’ in mice genetically engineered to have autism, suggesting it might do the same for people on the spectrum. 

An experimental drug may treat the core symptoms of autism, helping people on the spectrum to have better social interactions and stop repetitive behaviors, a new study in mice suggests

About 1.5 million people in the US have autism spectrum disorder (ASD). 

The spectrum is a broad one, with symptoms appearing very differently in men and women, children and adults, and this variance means that  

Many live independently, enjoy successful careers and loving relationships. 

But others struggle to engage socially, are racked with repetitive behaviors and easily reach sensory overload. Day-to-day living can be excruciating, and the symptoms debilitating. 

Autism’s causes are not entirely known, but experts suspect it is in part genetic, and in part due to environmental factors. 

The only real treatments are early behavioral therapy, and in some cases antipsychotic drugs, or the occasional antidepressant, but these don’t treat the central symptoms of autism. 

But these are largely intended for children with ASD, and there are not any approved adult treatments.  

Thanks to decades of research on the relationship between serotonin and autism, however, the team at FAU may be on the verge of one. 

First, they discovered 25 years ago that a genetic mutation was throwing off the regulation of serotonin, a key neurotransmitter to the feeling of happiness, in people with ASD. 

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Serotonin also plays an important role in social functioning, which made the team suspect its involvement in autism.  

Then they found that a particular enzyme had a dramatic effect on the reabsorption of serotonin. 

‘To use the Spinal Tap analogy, it turns it up to 11 and takes away way more serotonin than it should,’ lead study author Dr Randy Blakely told Daily Mail Online. 

So he and his team went looking for a drug that could ‘slow that enzyme down,’ he says. 

‘And we found it.’ 

They actually found it in another lab, at Northwestern University, where Dr Martin Watterson had only recently developed a new, experimental compound called MW150. 

So Dr Blakely and his team decided to test MW150 in mice that they had genetically engineered to have the genetic mutation and behaviors similar to those of autistic humans. 

It worked, much like an antidepressant might. 

‘In a week, it had removed those social behavior changes, and we saw a number of other features of their brains in terms of physiology that made them look more like the mice that didn’t have the mutation,’ Dr Blakely said. 

‘This opens up a whole new class of drugs for us to consider thinking about using in humans.’ 

MW150 is still several stages – and years – off from being available to autistic people. But Dr Blakely says that, even if the specific drug doesn’t work, his team has learned an important target for the ‘core symptoms’ of autism. 

And because they tested MW150 in adult mice, it seems it could be given to adult humans, since the changes it affects don’t have to be made during in utero development. 

‘It’s a foot in the door,’ Dr Blakely says. 

‘We wouldn’t be suggesting this medication to someone who has a mild form of autism and is perfectly capable of having a good quality of life and functioning in society … but there are children who become institutionalized.’ 

And even for people who are quite independent, ‘you do wonder, if they had the option when they were a little younger [to take a medication], maybe they would have an even higher quality of life,’ he added. 


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