Video: Reversing autism in the lab with help from stem cells and the tooth fairy

I look at my son a lot and feel like he’s trapped inside his own brain. Circuits are going crazy. He can’t figure it out. Of course, I can’t figure it out. And I think he deserves to have a voice…He’s never said I love you. He’s never said “Mom” with any meaning…I feel like he has been stolen from me.

Those sobering words from Jen, mother of 10 year-old Milo who has a severe form of autism, were shared during the Spotlight on Autism seminar at the March 13th CIRM governing Board meeting. You can watch a video of the Spotlight on our website (a short version with just Jen’s video clip is also available). CIRM grantee Dr. Alysson Muotri, the featured speaker who studies autism at UC-San Diego, explained that

people with autism have difficulty with language and social interaction and often get stuck in repetitive, focused behavior. It’s a spectrum, which means there are patients that are less severe and patients that are more severe…the CDC [Centers for Disease Control] estimates that we have 1% of the population with autism. If you are a boy, or male, you have a higher chance to be diagnosed.

Being such a complex, wide-ranging and very human behavioral disorder, how can researchers possibly learn the cause let alone develop treatments for autism? With the help of the tooth fairy, yes the tooth fairy, and stem cells, Dr. Muotri presented some startling research results that open a window into one day treating autism.

Dr. Muotri set out to compare the differences between the brain cells of autistic versus unaffected individuals. Since brain biopsies would be much too risky, impractical, and possibly unethical, he relied on the induced pluripotent stem cell (iPS) technique in which a cell sample from an accessible part of the body is grown in a petri dish, reprogrammed back into an embryonic stem cell-like state, and then transformed into brain cells.

To collect cell samples efficiently and non-invasively, Muotri devised a Fairy Tooth Kit Collection social media campaign in which autistic and unaffected kids could donate their baby teeth when they fall out. Muotri’s team then extracted the dental pulp cells from the teeth to ultimately generate iPS-derived neurons, the cells that send information throughout the brain and the body via electrical signals.

An initial glance between the autistic and unaffected neurons showed no differences. But on closer inspection the autistic neurons had fewer structures that are key to making connections with other neurons. But Muotri found after trial and error, that a particular molecule could restore the lost connections and lowered brain activity in the autistic neurons.

He also found that simply growing the autistic cells with other types of brain cells from unaffected individuals could also reverse the autistic characteristics. I encourage you to watch the video for more details of these fascinating results.

Does this mean we have a cure for autism? Not quite. But this human “model” of autism gives Muotri and other researchers a solid experimental platform for delving further into the changes that occur in the wide spectrum of autistic brain cells. And it helps them to understand more precisely how certain drugs or cellular environments reverse the autism in a petri dish. These studies are critical for one day propelling the work toward testing treatments in people.

And you can thank Dr. Muotri, his team, and the tooth fairy for that.

Read a blog, press release and brochure to learn about CIRM’s initiative to collect thousands of tissue samples in order to create and bank iPS cells from people with a wide range of disorders including autism. Also for more information about CIRM-funded autism research, visit our autism fact sheet page.

Todd Dubnicoff