UC San Diego Autism Research Described as “Game Changer”

Researchers say stem cells could lead them to the right drug to treat autism

Autistic stem cells are starting respond to experimental drugs in what San Diego researchers are calling a breakthrough in the disorder affecting one in 68 children.

UC San Diego scientists took stem cells from children with autism and reprogrammed them into brain cells.

“It was very surprising. For a long time we're thinking that those conditions such as autism that happens throughout development would be permanent but the fact that one can revert that or rescue that indicates that those conditions are potentially treatable," said Assistant Professor Alysson Muotri with the UC San Diego Stem Cell Program.

Muotri’s own son, Ivan, has autism and inspires him to keep searching for a cure.

"When I see my kid with autism I definitely want to wake up early in the morning and come to the lab to see if I can speed up the whole process," he said.

The lead researcher in this project, Muotri describes the discovery as a game changer.

Using blinking lights, researchers found non-autistic brain cells made lots of connections as if the cells were talking to each other. When examining brain cells from people living with autism, they found the cells showed much less activity.

"Because they're not blinking. It's like look silent," Muotri said.

But once researchers added experimental drugs to the autistic cells, the lights came on.

Researchers say stem cells could lead them to the right drug to treat the disorder.

Right now, the experimental drugs used in the research aren't safe for the human body. Muotri and his team are testing for the right candidate drug so they can one day find a cure.

"It gives me hope to the fact that perhaps one day we will be able to translate what happens inside the lab to the human brain the goal is to make him behave like someone that does not have autism become more independent and have like a normal life," he said.

Muotri recently received a $1.8 million grant for his research and hopes to have identified a drug to start clinical trials within next three years.

Contact Us