August 13, 2020

The Niche

Knoepfler lab stem cell blog

Why I am still very skeptical about stem cells for autism

When patient families reach out to me, one of the most common questions is whether stem cells for autism offer real hope.stem cells for autism

The short answer at this time remains “no”. Of course, it’s not as simple as just “no” because there isn’t enough data to be sure, but there is reason for major skepticism about the use of stem cells for autism.

I’ve covered this topic a number of times over the years because patients and families want to know and because it is so often hyped. Sure, there are real clinical trials now and then with FDA approval to test stem cells in autistic patients, and in theory some may ultimately prove to be safe and effective. However, of those recently and currently being tested as well as other “therapies” already being sold at dubious clinics, I doubt any will make it to the finish line as making a real difference.

Why am I so skeptical still? Many reasons.

  • There is no compelling, preclinical evidence that I know of that stem cells can help autism and that they would be definitely safe in kids.
  • The most common route of administration, IV into the blood stream, doesn’t make much sense to me. Do a meaningful number of cells even get into the brain that way?
  • If cells don’t get into the brain, they do something systemically that helps the autistic brain? Some folks believe that there is a definite autoimmune component to autism and that flooding the bloodstream with stem cells might reduce autoimmunity, but there’s little hard evidence for either of these ideas.
  • If yes they do get into the brain, what do the cells that get into the brain do that is helpful and where do they do it inside the brain? Which brings me to the next question.
  • What causes autism spectrum disorder? The field doesn’t really know so how can you treat it if you don’t understand it?
  • Under that umbrella “spectrum” term there are many manifestations and probably many causes so stem cells are unlikely to be an autism panacea.
  • Is there any evidence that once autism manifests that it is reversible?

And the list of concerns and questions goes on and on…

According to clinical trials.gov, there are now 13 studies across the globe and 4 registered stem cells for autism trials in the US (see map above). The one here in California at Sutter has ended and no data are posted or published that I know of at this point. The one listing in Arizona and two in Virginia as well as one of the two in Florida (really apparently four that are just one study together) are also completed. They were kind of different in that they was based on the idea that hyperbaric therapy would help boost stem cells in kids with autism. No data posted. The other one in Florida, with Ageless Regenerative Institute as sponsor, was withdrawn. There the notion was to use fat stem cells to treat autism. I have no idea how fat stem cells could by any stretch of the imagination help autism. Unfortunately quite a few stem cell clinics sell stem cells of autism, which is deeply concerning. It is experimenting on kids for profit with little chance of success.

My clinicaltrials.gov search did not find the specific stem cells for autism trial most in the news of late, the one at Duke by stem cell scholar Joanne Kurtzberg, because “stem cells” are not mentioned in the listing, just umbilical cord blood. Update: They’ve published their findings here, which supports safety but we can’t be sure about efficacy in this open-label, uncontrolled study (HT: commenter Matt). I’ve posted about Dr. Kurtzberg’s trial and did an interview with her (see pieces here) in the past. To specifically read my older two-part interview with Kurtzberg see here and here.

More broadly there are 24 research articles in PubMed with both “stem cells” and “autism” in the title. Some of this is very interesting research, but it is concerning to see that in this PubMed search as of today at least, the very first result on PubMed is a “trial” in India of infusing autistic children with apparently undifferentiated human embryonic stem cells, which in my opinion is extremely dangerous.

Overall, it’s just not adding up to me that this is a fruitful line of clinical research at this point. I hope I’m wrong. Some may say to me, “What’s the harm in trying?” and I’d say that there are risks of various kinds to the children including health-wise (although these may be relatively lower with cord blood products) and in raising parents’ hopes with little to back it up.

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