Discovery of autism: a new drug may "refuse" an enzyme that diverts social behavior

Through a new discovery, a treatment for adults with autism can be considered.

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

Some advocates of autism are increasingly insisting that people in the spectrum should not be treated as "disordered" but simply different.

The FAU researchers also note that autism often accompanies physiological problems, especially for the intestines.

There is currently no treatment for autism in adults, but the team has discovered a drug that addresses behavioral "disturbances" in mice genetically modified to become so, which could be the case for people in the spectrum. .

In the United States, about 1.5 million people are afflicted with an Autism Spectrum Disorder (ASD).

The spectrum is wide, the symptoms appear very differently in men and women, children and adults, and this variance means that

Many live independently, have successful careers and romantic relationships.

But others struggle to engage socially, have repetitive behaviors, and easily reach sensory overload. Daily life can be excruciating and debilitating symptoms.

The causes of autism are not fully known, but experts suspect that it is partly genetic and partly due to environmental factors.

The only real treatments are early behavioral therapy and, in some cases, antipsychotic medications, or occasional antidepressants, but they do not treat the central symptoms of autism.

But these are largely aimed at children with ASD and there is no approved treatment for adults.

Through decades of research on the relationship between serotonin and autism, the FAU team may well be close to it.

First and foremost, they discovered 25 years ago that a genetic mutation was deregulating the regulation of serotonin, a key neurotransmitter of happiness, in people with ASD.

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

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

"To use the analogy of Spinal Tap, it goes to 11 and removes a lot more serotonin than it should," said Dr. Randy Blakely, author of the study, at the Daily Mail Online.

So he and his team went off in search of a drug that could "slow down this enzyme," he says.

And we found it.

In fact, they found it in another lab, at Northwestern University, where Dr. Martin Watterson had recently developed a new experimental compound called MW150.

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

It worked, a bit like an antidepressant.

"In a week, social behavior changes were suppressed, and we found a number of other features of their brain in terms of physiology that made them look more like mice that did not have the mutation, "said Dr. Blakely.

"This opens up a whole new class of drugs so we can consider thinking about the use in humans."

MW150 is still several steps – and years – to be available for people with autism. But Dr. Blakely said that even though the drug in question was not working, his team had learned to target an important target for the "fundamental symptoms" of autism.

And since they tested MW150 in adult mice, it seems that this drug could be given to adult humans because it is not necessary to make changes during development in utero.

"It's a foot in the door," says Dr. Blakely.

"We would not offer this drug to a person with mild autism who is perfectly capable of living in society and having a good quality of life … but there are children who are placed in an institution. . "

And even for fairly independent people, you wonder if they had a choice when they were a little younger [to take a medication]they might have an even better quality of life, "he added.