May 27, 2020

The Niche

Knoepfler lab stem cell blog

Stem cells for autism: are we there yet?

Autism affects millions of children in the U.S. alone.

It is a perplexing spectrum of disorders with “autism” being an umbrella term for a host of related neurological illnesses. The negative impact of autism on our society transcends economics, but has been estimated at 10s of billions of dollars.

There is currently no cure or even fully validated treatment for autism.

Part of the problem is that scientists do not really understand what causes autism. Without an understanding of the cause, it is difficult to treat a disease, huh?

Add in the fact that autism is really, as mentioned above, a host of related, yet distinct disorders, and treatment becomes even more challenging.

autism stem cells

Nonetheless, the idea of using stem cells to treat autism has gained some traction.

In fact, a first of its kind clinical trial for stem cell-based treatments of autism just started today right here in Sacramento run by Sutter Neuroscience Institute (I’m not affiliated with them as I work at UC Davis) run by Dr. Michael Chez.

The clinical trial, reported on the front page of today’s Sac Bee (see above) involves giving pediatric patients cord blood stem cells as a potential treatment for autism.

Some of the children in the study will be given placebo, while others will be given a treatment of their own cord blood stem cells that were banked when these kids were born. Thus, no immunosuppression should be needed.

The rationale behind the trial is that the stem cells will migrate to the brains of the kids and do something to repair some kind of underlying damage that is responsible for autism.

This is an exciting clinical trial and is an example of doing things right with stem cells in going through the FDA to do a legit clinical trial, rather than as so many clinics do treat patients for profit while dodging the FDA.

From the Sac Bee article:

Autism is thought to have multiple risk factors, including genetic, environmental and immunological components.

It is the immunological component that interests Chez most. Much of his research focuses on the relationship between a child’s immune system and the central nervous system. Evidence suggests that some children with autism have dysfunctional immune systems that may damage or delay development of the nervous system.

Cord blood stem cells may offer ways to modulate or repair the immune systems of these patients who have no obvious reason to become autistic,” Chez said.

I’m very excited about this trial, but at the same time frankly I believe there is probably not a super high chance of success with this study given all scientists and doctors still do not know about autism and also about the success rate of clinical trials more generally. However, even so, given the impact of autism on our world, we need trials like these even if some if not most do not work….and this one might work.
Indeed, I hope that Dr. Chez is right and that the kids show improvement. Sometimes in science you need to try things out to see if they work before you can be sure and doing so in an FDA-approved clinical trial is the way to go.
Cheers to Dr. Chez and the patients he is trying to help!

Look for more clinical trials of this kind in the future. A comprehensive list of stem cell-based clinical trials specifically for autism can be found here (and handy links for trials for other diseases can be found here on my clinical trial resource page).

 

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