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).

 

7 Comments


  1. “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.”

    That’s it? They got approval to inject cells into human subjects on the possibility that they will ‘do something’?

    I don’t get it. If those cells were going to ‘do something’ wouldn’t they have already done it and allowed the child to develop without autism? What makes them think these cells are going to prevent autism now when they didn’t when the kid was in utero?

    I’m all for stem cell research and I applaud the researchers working on getting cells to differentiate and target particular diseases, but this? Unless I am really missing something here, it seems they are just putting these cells in there and hoping for a magical response.

    Why would these magical cells be any more inclined to migrate to the brain to ‘do stuff’ than to say, a diabetic pancreas, a defective heart, hell even the bones underlying a hare lip?

    Is there more information on why they think this is going to work?


    • You ask some good questions. Tough ones, which I like.
      I was perhaps too easy on the folks doing this clinical trial in my blog piece.
      The fundamental weakness of this Sutter clinical trial is that it is targeting a disease, autism, for which we do not know a cause….as a result it is high risk in the sense that many assumptions have to be true for it to work. I don’t think it’s high risk in the sense of endangering the children.
      Stem cells are thought to home in on areas of damage in the body so in this clinical trial one big assumption is that the umbilical cord stem cells will go to the brain because in autistic kids there is something that the stem cells will sense as damage. How likely is this? I don’t know, but it seems a stretch to me.
      Another big question is can the cells actually get to the brain in the first place from the bloodstream, not a trivial matter. Most stem cells injected into the bloodstream are filtered out in the lung in fact.
      Even assuming the stem cells can (A) get into the brain and (B) migrate to the still unknown regions that are responsible for autism…the folks running the clinical trial have to hope that (C) the cells can fix whatever is causing autism. You can see the hurdles start piling up for this thing to work.
      Alternatively, the cells may not need to get to the brain to work, but might somehow change the function of immune system cells in the blood so they no longer cause autism….of course we do not know in the first place if immune cells play any role whatsoever in autism.
      Take altogether, these are the reasons along with the fact that most clinical trials more generally fail is why I think this trial is unlikely to work, but I guess one should not give up hope.


  2. How much research is there for immuno involvement and autism? Perhaps they are hoping to truncate inflammatory cascades as they may see this as systemic and they could be focusing on the immediate properties of the injected cells rather than expecting them to reproduce. I have had people come to me in relationship to cogniitive components following non FDA treatment. Treatment with cells did not show improvement over baseline but did not test autism specific individuals.


  3. The down side of this study is that it is being funded by CBR cord blood bank and will only allow children that have cord blood stored with CBR to participate. As opposed to what Dr. Kurtzberg is doing at Duke with autologus cord blood and cerebral palsy where the subjects are not limited by where their cord blood was stored. This is an example of what happens when private industry controls research funding to use as a marketing tool, creating a world of haves and have nots.

    Another key point, CBR also has a study going on CP at the Medical College of Georgia that is having difficulty reaching the enrollment quota. This was a undisguised initiative by CBR to put out a paper first and try to beat Dr. Kurtzberg to the “punch” if you will.

    Considering the fact that the press release went out on the autism study but enrollment was not open on clinical trials.gov tells me that CBR already had their subject quota pre-filled before the press release ever went out. I personally have spoken with several parents of autistic children who are CBR clients that have pushed for years for this kind of study only to now find out that their child has aged out of the trial and have been refused by Sutter to participate in any compassionate care, Unlike the option Duke offers when children who have autologus cord blood and who are just outside the subject criteria for the CP study, for example TBI or near drowning ect.. are allowed compassionate care. Am I happy there is at least a autism study going? Yes. Am I happy about the way it is happening? Absolutely not.


  4. The recent article in the New York Times provides a possible reason for certain stem cells to possibly affect autism http://www.nytimes.com/2012/08/26/opinion/sunday/immune-disorders-and-autism.html?pagewanted=all.

    The effect would be through the immune suppressive activities that appear to be associated with transplant of mesenchymal stem cells. I still don’t really understand the immune effects of MSCs myself, but enough has been published to make me think there is something there.

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