Autistic boy meets his assistance dog 3:16
(CNN) -
In the beginning, Joann Fouquette's son, Ezra, reached all the goals.
It's what every new mom expects: a happy, healthy baby.
But around 17 months, things started to change.
He stopped talking.
He began to cover his ears and hit his head on the ground as if something was bothering him.
Fouquette recalls her mother telling her, "I think we need to test her. Something is definitely going on there."
Five months later, in 2012, Ezra was diagnosed with an autism spectrum disorder.
"It's devastating," he told CNN's chief medical correspondent Dr. Sanjay Gupta.
"I've heard people compare it to losing a child. You lose the idea of the child you were going to have, the life you were going to have, the life he was going to have."
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Ezra Fouquette was diagnosed with autism when he was 22 months old.
Autism is a neurodevelopmental disorder that affects 1 in 44 children in the United States, according to the US Centers for Disease Control and Prevention (CDC).
It begins early in life and the central symptoms are social and communication problems, as well as repetitive behaviors and rigidity.
"They can speak very well, but they can't carry on a conversation. So you have the opposite end of the spectrum, where you have children and adults who are completely non-verbal," said Dr. Doris Trauner, pediatric neurologist and distinguished professor of neuroscience and pediatrics at the University of California, San Diego.
"They have a certain routine. They like to do things over and over," he told Gupta.
"They have repetitive behaviors, the most typical are things like flapping with their hands or going around in circles."
But autism can also lead to more worrisome problems, such as serious disruptive behaviors and self-harm.
"Unfortunately, aggressive and self-injurious behaviors are very common, especially in children with severe autism," Trauner said.
"Anything from repeatedly banging your head against the wall to hitting your head with your hands, pinching yourself or biting your hands."
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Behavioral therapy, occupational therapy, and speech therapy can help, but there are no FDA-approved treatments for the core symptoms of autism.
There are two FDA-approved antipsychotic medications, used to treat bipolar disorder and schizophrenia, that are approved to treat children with autism, but only if they show severe aggression or self-harm.
"These medications are effective for these symptoms, but unfortunately they are associated with significant side effects," said Dr. Eric Hollander, director of the Autism and Obsessive Compulsive Spectrum Program at Montefiore Health System in New York.
"It can predispose them to developing things like diabetes or cardiovascular problems."
"There is a significant need to develop new treatments, both to treat the core symptoms of autism ... and to reduce the burden of side effects," he added.
Looking for answers
When it came to her son Ezra, Fouquette tried everything she could think of to help him.
"We tried gluten-free, casein-free, dairy-free. We have tried homeopathic remedies. We have tried all the therapies out there," he told Gupta.
But she didn't want to give him psychotropics when he started to get more aggressive.
"I didn't want to try any of those simply because there were so many side effects," he commented.
"But at one point, I had a lot of bruises because he was getting more violent."
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Fouquette was desperate for help.
That's when he saw a story on the local news about a clinical trial involving children with autism and CBD, the non-psychoactive part of the cannabis plant, at the Center for Medicinal Cannabis Research at UC-San Diego.
"He was 9 years old then," he said.
"What am I going to do in the future as he continues to grow if I am already having a difficult time with his aggression now?"
Fouquette did not hesitate to try medicinal cannabis for her son.
"I have seen the use of CBD in children with epilepsy," he said.
"I saw how much it helped other people and I thought, 'It's totally natural. It may not have any real side effects. Why not give it a try?'"
So he enrolled Ezra in the clinical trial.
Medicinal cannabis, autism and the brain
Trauner is the principal investigator of the UCSD trial, a double-blind, placebo-controlled crossover study, which means that the participants don't know when they are receiving the placebo or the drug, and neither do the doctors.
Researchers examine how CBD might affect the brains of children with autism.
"We know that in autism there are some differences in the chemistry of the brain. There are some changes in the neurotransmitter systems, both in the dopamine system and in the serotonin system, that can contribute to some of the symptoms," he said. he said to Gupta.
Serotonin and dopamine are neurotransmitters that carry signals or information from one nerve cell to another.
Serotonin is believed to regulate mood and have a great effect on early brain development.
Dopamine can reinforce behavior when we receive a reward.
Several studies have shown that dopamine levels that are too low or too high in children with autism can cause certain areas of the brain to malfunction, resulting in high levels of repetitive behaviors and reduced levels of social interaction, Trauner said.
In animal models studying autism, brain serotonin levels may be lower than expected, and adding serotonin improves social functioning in mice, he said.
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"And CBD, among many other things, has effects on the serotonin system by increasing the availability of serotonin," he added.
"And that can help in terms of social interactions in particular."
A similar trial involving children and adolescents with autism and the cannabidivarin (CBDV) cannabinoid cannabidivarin (CBDV) is also being conducted nationwide in New York.
"We know that autism is a developmental disorder that begins early as the brain forms," said Hollander, the lead investigator for this double-blind, placebo-controlled study.
"I think CBD may play an important role in autism," he said.
"It can decrease excitation in neurons and increase inhibition."
When children with autism are overly excited or have insufficient inhibition, it can lead to explosive behaviors, tantrums, anger or self-harm, and they can display repetitive behaviors, Hollander said.
"So it changes the ratio of excitation to inhibition in different neurons," he said.
Ezra's mother, Joann Fouquette, said she believes her son's aggressive behavior was due to his failure to communicate his needs, as he was mostly not speaking.
While both studies are still ongoing and the blinds have not been broken, the initial feedback has been positive.
"Some of the patients have had a really substantial benefit," Hollander said.
"We have seen what we expected, which was a significant decrease in symptoms of irritability, tantrums or explosive episodes. We have also had patients who have improved in their repetitive behaviors."
In California, similar reports have come in from parents.
"We saw some pretty impressive changes," Trauner said.
"The kids whose aggressive behavior was everyday, it's gone. I mean, it's gone ... Kids whose self-injurious behavior is better, and they're to the point where the calluses on their wrists are starting to heal," he said.
"A lot of the kids are more sociable."
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But Trauner cautions that more research is needed.
"It is too early to get excited. I think there is some reason for hope, but it is not a good idea to rush out and buy it and try to use it on your own," he said.
"There are several reasons for that. One is that it can be toxic. It can cause liver dysfunction," he added.
"It is also unclear what the best dosage is if it works, and if what you are buying really has what you think it has, because it is not regulated."
"I'm getting my boy back"
During the trial, Fouquette's son Ezra received the placebo at one point and CBD at one point, but Fouquette and the doctors still don't know when he was receiving it.
Ezra was mostly silent when entering rehearsal, he said.
But in the first weeks something extraordinary happened.
"One day, I was at the grocery store and my husband sent me a video. And it's Ezra lying on the floor wrapped in a blanket, and he's singing," she said.
"He's never sung before ... and he's singing the whole song."
Fouquette told Gupta what was going through her mind at the time.
"I'm getting my baby back. I'm getting my son back," she said, fighting back tears.
"I can communicate with him. He is talking to me. He is happy. He is no longer aggressive. He is singing," he said.
"What more can I ask for?"
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Since Ezra finished the study a year ago, he has shown no assault and has continued to communicate.
"He has not had any regression," he said.
"It helped him ... whatever was going on in his brain, to make the connections that he needed to make. And once those connections were made, he never lost them."
It's unclear what role the treatment may have played in Ezra's progress.
The study has not been published and it was not analyzed whether cannabis or CBD could be neuroregenerative for the brain.
More research is required in this area.
"Some of the children who have shown an effect ... show it for several weeks after the study drug is withdrawn. And some seem to maintain some improvement," Trauner said.
"But I don't know why that would be."
Regardless of why it happened to Ezra, who is now 11, Fouquette is grateful.
"I don't think it's a cure. It gave him the ability to speak. It gave him the ability to communicate more. I think that's why the aggression went away," he said.
"It will just make things easier for him, easier for him to live, easier for him to be himself."
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