Given the complexity of autism, researchers have struggled to detemine its causes and the best way to treat, but CIRM grantee Dr. Alysson Muotri of UC San Diego, is turning to cell therapy to treat autism, specifically stem cells— and help from the tooth fairy—to begin answering those questions.
Fairy Tooth Kit
He set out to compare the brain cells of autistic and unaffected individuals. Since brain biopsies are too risky and impractical, he used induced pluripotent stem cell (iPS) technology. His team took accessible cells, reprogrammed them into stem‑cell‑like cells, and then turned them into neurons.
To gather these samples, Muotri launched a “Fairy Tooth Kit Collection” social media campaign. Autistic and unaffected children donated baby teeth, which provided dental pulp cells. From there, his team generated iPS‑derived neurons, the cells that send electrical signals through the brain.
In explaining the condition, Muotri noted that autism affects language, social interaction, and behavior. Moreover, the condition spans a wide spectrum, with patients ranging from mild to severe. According to the CDC, 1% of the population has autism, with boys at higher risk.
Trapped inside his own brain
Jen, the mother of ten‑year‑old Milo, said his severe autism, tried to explain to what its like living with the condition.
“I look at my son and feel like he’s trapped inside his own brain,” she said. “His circuits are going crazy. He can’t figure it out. I can’t figure it out. He deserves a voice. He has never said ‘I love you.’ He has never said ‘Mom’ with meaning. I feel like he has been stolen from me.”
What does look like
At first, the autistic and unaffected neurons looked similar. However, closer study revealed that autistic neurons have fewer connection‑forming structures. Afterward, Muotri found a molecule that restored these connections. As a result, excessive activity in autistic neurons decreased.
In addition, the researchers discovered that growing autistic neurons alongside other brain cells from unaffected individuals could reverse autistic characteristics. For more context, the video explains these results in greater detail.
Does this mean we have a cure?
Not yet. Still, this human “model” of autism gives scientists a powerful tool to study the condition. In particular, it helps researchers see how drugs or environments might reverse autistic traits in a dish—an essential step toward future treatments.
For now, we can thank Dr. Muotri, his team, and the tooth fairy for opening a new window into autism research.
For more information about CIRM-funded autism research, visit our autism fact sheet page.
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Thank you so much for sharing such an informative blog post. Keep sharing such topic. Waiting for your more future posts regarding tooth.
Great video! Not a definite “cure” but very informative and helps us more understand about “cells”. I will definitely add this to our http://www.livingautismnow.com resources.
To whom may help me,
I am a mum of a 3 year old son who has been diagnosed with difficulties related to autism. Do you think that stem cells can help him out with this condition. I am really desperate to have a reply because I never felt so weak in my whole life and I am hoping that something will help him out of this.
Thanks so much for your help and best regards,
Michelle
Dear Michelle-Louise, thank you so much for your email and I’m sorry to hear about your son. I know how difficult it can be to get a diagnosis like this but there are many resources out there that can help you, your son, and your entire family.
I certainly hope that one day stem cells will be able to help your son and others like him, but for now that research is still at a relatively early stage. One exciting area that we are funding is the work of Dr. Alysson Muotri at the University of California, San Diego. He is comparing neurons, brain cells, made from stem cells of children with autism to the brain cells of children without autism. It’s helping him identify areas of difference, which is a key step in being able to develop a potential therapy.
Here is a link to a video of Dr. Muotri talking about his work: https://www.cirm.ca.gov/node/26011
I do hope that is of some use to you.
Yours truly
Kevin McCormack