Duke Launches Large, Debated Stem Cells for Autism Trial

Is there real promise of stem cells for autism?

At this point the jury remains out on that question, but a growing number of kids are nonetheless getting such “treatments” at for-profit dubious clinics. Academic clinical researchers are interested in this area as well.

Dawson Kurtzberg launching stem cells for autism trial
Dawson Kurtzberg are launching a new stem cells for autism trial.

Still, the potential use of stem cells to treat autism is a highly controversial area even in a clinical trial setting.

Today it was announced in a piece on the website of the Simons Foundation Autism Research Initiative that a dramatically larger study involving hundreds of autistic children and adults is now being run by a team at Duke led by Drs. Joanne Kurtzberg, M.D. and Geraldine Dawson, Ph.D. (pictured above in image from Duke PR).

2020 Update: Duke’s Phase 2 trial found no benefit of stem cells for autism, but the group plans to continue anyway.

A very surprising and intriguing aspect of the article on the Kurtzberg trial from the Simons Foundation is that it alternately quoted Kurtzberg and Arnold Kriegstein, Director of the Broad Center of Regenerative Medicine and Stem Cell Research in what feels like a debate format. Kriegstein is highly skeptical of the Duke trial, which he is quoted as calling “premature” and “more like a ‘Hail Mary pass’ than a rational therapy.”

The article quotes him as to why he is skeptical:

“These are not cells that can treat a laundry list of diseases,” he says. Because the stem cells are similar to those that normally give rise to blood cells, he says, it is unlikely that they can repair or replace neurons in the brain. Also, because autism results from errors during development, it is unlikely that the stem cells can reverse those effects.

It is unusual and valuable to quote researchers in this way with different opinions. I find it surprising how blunt Kriegstein is in his concerns about this trial. The trial was also announced on the Duke Medicine website here. According to the Duke PR, Dawson was “who was the founding director of the University of Washington Autism Center and then chief science officer at Autism Speaks before joining the Duke faculty in August 2013.” Dawson and Kurtzberg are two top-notch biomedical scientists.

The $40 million stem cell autism trial is funded in part by a $15 million gift from the Marcus Foundation in Atlanta. The official name of the trial is “Autologous Umbilical Cord Blood Infusion for Children With Autism Spectrum Disorder (ASD)”.

The primary outcome measure is safety, while secondary measures are potential efficacy as measured by cognitive and behavioral tests.

The trial will also use umbilical cord stem cells to treat other conditions such as stroke, but the team aims to treat 390 autistic people in that arm.

In fact, the trial apparently already started enrolling patients last month including 20 children ages 2-5 years old treated in an autologous fashion.

For more background on the use of umbilical cord stem cells for autism and other disorders see my two-part interview with Kurtzberg here and here.

Two of the difficult aspects of the idea of using stem cells to treat autism are (1) that autism spectrum disorder is a diverse umbrella group of disorders and (2) that it remains unknown what the various causes might be. Another challenge is that stem cells infused IV rarely if ever make it into the brain. At least in part for these reasons, I’m skeptical that stem cells used in the manners proposed can help autism.

autism stem cells

In 2012 a small trial right here in Sacramento by Sutter was launched using umbilical cord blood stem cells to treat autism. You can see the SacBee headline announcing it that I read at the breakfast table.

The Duke trial official clinicaltrials.gov listing can be read here for more details.

For example, the dose used with be 10-50 million nucleated cells/Kg of subject weight.

I wish we knew more about the pathogenesis of autism, which is a fancy word for saying what causes it, before we proceeded with these kinds of trials. On the other hand, I can see where such a trial could greatly advance knowledge and there is an urgent and seemingly growing need. Overall, I have very mixed feelings.

What are your thoughts on this trial? On the idea of using stem cells to treat autism? Perhaps we can have pro and con readers have a fruitful debate right here in the comments as well.

13 thoughts on “Duke Launches Large, Debated Stem Cells for Autism Trial”

  1. There are potential dangers of Stem Cell treatment for developmental disorders like ASDs. Uncertainties shadow the possibilities of success.

    First of all, we don’t know when the alterations in the connections and wiring takes place in the brain. Second, we don’t know where exactly in the brain does it go wrong. Is it the cingulate gyrus, precuneus, frontal cortex, OFC, DLPFC or hippocampus or amydala. Thirdly, the SVZ and the sub Granular zones of the brain have already been implicated in neurogenesis. In 2-5 year old children, the neurons are actively growing and making connections. And by implanting new cells (which we are even unsure if they’ll ever reach the proper destination), I believe we are putting the lives of these children into grave danger.

    Also, experts in the field would also not deny the fact that stem cells have tremendous notoriety for developing into massive teratomas, which would even worsen the situation. The stem cells can definitely be used to study the pathogenesis of the disorder, but the clinical trials, I believe, it still can take some more time.

  2. I know nothing about medicine, research or stem cells however, I do know that Duke is using the child’s own stem cells on the Autistic children in the study so I fail to see why they should NOT study this as a potential way to help these children. What if just one of the 20 kids improves? Wouldn’t that be wonderful? I hope they are successful in finding a way to improve, even just a little, a cild or children afflicted with this terrible disorder. Shame on all of you that are judging without knowing for sure that it will not help.

  3. I find it surprising that anyone is still pushing the “repair or replace neurons” argument – it has been clear for many years that most stem cell effects are indirect – anti-inflammatory, anti-apoptotic, immunomodulatory, release of neurotrophic factors etc. Almost none of the preclinical studies show replacement of neurons, yet they almost all show efficacy. In the case of CP (and possibly autism) the mechanism of action is unknown, but is likely to include increased dendrite formation by existing neurons, driven by neurotrophic factors produced by cord blood cells (not even necessarily the stem cells – monocytes and T-cells seem to be important as well).
    It is an interesting commentary on the way peoples minds work that if they cant fit something into their pre-existing paradigm, they assume its wrong. I have heard this sort of argument from so many scientists who know a little, but not enough, about the current state of the research, yet feel free to pass judgement.
    Perhaps researchers are partly to blame. I still see numerous presentations that start off talking about differentiation capacity of stem cells, then demonstrate that the effects they are showing are nothing to do with direct differentiation and are all about the indirect effects.
    But all of this demonstrates that we are going to have to find out if it works first, then sort out the exact mechanism of action, rather than assume it wont work because we dont know the mechanism of action.

  4. Stem cells are like mini drug factories, releasing the correct proscription upon diagnosis of cellular and tissue problems once they arrive in the local vicinity. This doctor should know this, seems like he’s got some preconceived notions of what he’d like them to do or where he’d like them to go. Typical of physicians . They think they understand but they don’t have a clue in most cases. If they weren’t told something by one of their peers, they wouldn’t think it was true. so here comes antidotal proof and it doesn’t fit their paradigm so they shoot holes into it. again typical. but when things start to show signs of improvement in a new therapy they are the first to say I knew there was promise there, aren’t i just soooo smart?pa lease let this trial go forward it’s autologous, safe, and we’ll all see it effects soon. We’ve already seen some amazing responses. everyone keep track of this doctor so we can all drop him an email when the result are published.

  5. Dear Frances;
    Clearly you are in the know with respect to preliminary data which the scientific community is not. Generally, I believe that foundations are bigger risk takers (for good reason, as I was a former scientific adviser for CAN), but that hope-driven philosophy can also lead to big mistakes. In this case, for example, except for the “dose” of “therapeutic” cells, I suspect that there is nothing that is uniquely circulating in cord blood that is not already present in the newborn. So, skepticism is warranted.

    1. I wish I was as “in the know” as you think! 😉 I spent hours studying every release on the grant and trials so I could write up the newsletter article… There is definitely some wishful thinking with every grant from an advocacy organization, I hope this one pays off.

  6. There are a lot of inaccuracies in the description of Duke’s cord blood trial for autism given by SFARI.org. I would urge skeptics to read the trial rationale on ClinicalTrials.gov or (dare I say it) read the July newsletter from my Foundation that I released this morning: parentsguidecordblood.org/newsletters/

    The rationale for trying cord blood stem cells on Autism Spectrum Disorder (ASD) is based on previous results at Duke, with 100s of kids over a decade, using cord blood therapy for acquired neurological disorders.

    The exact mechanism by which cord blood stem cells benefits kids with hypoxic ischemic encephalopathy, for example, is not understood. No one is claiming that cord blood stem cells completely regenerate brain neurons. Probably the paracrine effect plays an important role.

    To give some context from history: No one understood how penicillin worked when it was first discovered either.

    Duke has just received $15M, not $40M, and the first trial is a safety study of autologous cord blood for 20 kids with autism. The additional trials for hundreds of children and adults have not been approved yet, they are proposed in the grant application but may be redesigned based on initial trial outcomes.

    In my opinion, the plan to launch a US trial of allogeneic cord blood for cerebral palsy is a bigger piece of news than the autism safety study, but since the autism trial has left the gate it makes better press.

  7. I side with Arnold Kriegstein on this issue, and am glad that he is willing to be so straightforward. The implications from the description of the study are that: 1. autism has a clear pathological cause (like spinal cord injury or Parkinson’s disease), and 2. that umbilical cord stem cells can turn into neurons, or at least some kind of neuronal support cell that will fix the pathology. Both of these implications are wrong. No subtleties- they are wrong.

    I’m afraid that studies like these are used as rationalization for charlatans. I spoke just recently with a woman who sent her child to Ukraine to have a “stem cell treatment for autism”. Twice, because, gosh, it didn’t work the first time!

    Brain transplants of umbilical cord stem cells are a decent idea for treatment of lysosomal storage diseases, like Tay-Sachs disease and Pompe disease (the subject of a book by Geeta Anand- “The Cure”, and a movie- “Extraordinary Measures”). In those diseases, the cord stem cells just supply an enzyme that is missing.


    1. Thanks for the comment, Jeanne. I agree with your take on this. I find it notable and admire that Kriegstein was so willing to take a public stand on this issue. I get emails and calls at least once a month about stem cells for autism.

    2. Geneticist from the East

      I agree with Jeanne too. But I do know that there is a small subset of autism cases that are caused by non-functioning or weakened enzymes. So the stem cell therapy might work in those cases. Still, the PIs should at least genotype the autism patients and find out who are more likely to be beneficial to such therapy,

  8. It is surprising that funding to the tune of 40 million dollars was able to be secured. Increasingly, the best bet for garnering PI (and institutional)-acclaim and notoriety seems to be less what rationale or knowledge base they bring to the table for innovation, but rather how great they are at sales(wo)manship and PR.

    1. But those are the ones that fall apart every single time. Go back to the time when Korean scientists claimed that they’d found a way to clone human embryos, and remember how much press they got. Look at the Dainippon Sumitomo stem cell drug failure, and how much coverage they had when things looked promising. Look at the monumental press that STAP had in the beginning. Look at the cardiac ASC trials, another scandal in the making, IMHO. They’ve had lots and lots of glowing press.

      The loudest advertising always seems to be leading to the biggest failures. Funders may be fooled by PR for awhile, but results will ultimately determine who really succeeds. Eventually, people will start to figure out that news pieces and publicity stunts aren’t getting them anywhere when there is nothing substantial to back up the claims. I could really name some names of incipient failures that I think are coming down the pike shortly, but for now, let’s leave it at that!

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