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Part 1 of Hope, plus hype: The rise and fall of “stem cells for autism” at Duke University
Information from my upcoming book “The Children Do Not Consent”
This is the first in a series about Duke University’s experiments on autistic children, in which young children, including those with IQs below 70, were injected with stem cells to see if it would stop them from being autistic. This series looks at the 9 years from first FDA approval in 2014, to the addition of Expanded Access (EAP) “pay to play” clinical trials, to pushback from experts and finally the cancellation of the EAP in 2023.
Let’s start this story at the end of it. In late 2022, Duke University abruptly ended its pay-to-play stem cell experiments on autistic children, a program that had been operating since 2017. The experiments, inflicted on developmentally disabled children as young as two years of age, had initially been approved as an Expanded Access Program by the FDA, which enabled a team at Duke, led by Dr. Joanne Kurtzberg, to charge parents $10,000 to $15,000 per visit to bring their child to Duke for stem cell infusions, with the hope that this unproven and risky treatment could cure them of autism.
The cancellation was a dramatic turnaround for the team who, since they began in 2014 with a series of clinical trials, had been building hype around the promise of stem cells as a potential autism treatment for families (and a potential licensable product for its partners). In the immediate aftermath, former project associates seemed to scuttle away into dark corners, with the Duke team itself not releasing any public statement at the time about the closure. There appeared to be a media blackout of sorts by the team, which included Duke as well as Cryo-Cell.
Duke team and Cryo-Cell had an interesting relationship. Cryo-Cell is a stem cell company that had been lined up to have an exclusive patent rights to the procedure if researchers could provide proof of concept. The agreement would have granted the company the “rights to proprietary processes and regulatory data related to cord blood and cord tissue developed at Duke, [making] Cryo-Cell the only cord blood bank with the right to offer treatments to [autistic] children at its own infusion clinic under Duke’s FDA-approved Investigational New Drug Application.”
Kurtzberg, the project lead, was actually the Medical Director for Cryo-Cell International and Director of the Carolinas Cord Blood Bank, which dealt in stem cells. Prior to the project’s cancellation, Cryo-Cell was breaking ground on clinics that promised to deliver stem cell “therapy” to autistic children, for a hefty fee. Cryo-Cell had initially announced in 2021 that it would open its first dedicated clinic in 2022 and, in its pitch deck to investors, projected annual revenue per clinic to be $24 million.
Word got out about the program’s ending before any press release from the team. I first read about it in posts by parents in a closed Facebook groups for waitlisted families, many months before anything was made public. Parents had begun receiving phone calls about the cancellation in the fall of 2022; then, a formal letter was sent to them from Duke in January. After waitlisted parents shared copies of Duke’s letter with me via Instant Messenger in January, I grabbed screenshots and tipped off media. The public needed to know. The journalist who broke the story was Anna Merlan at Vice, whose research brought further issues to light (as we will see below.)
In their January letter to parents, after noting that the EAP is no longer available, the Duke team reminded parents that results of its 2020 randomized study had not demonstrated benefit. As study authors had concluded:
“Overall, a single infusion of [cord blood] was not associated with improved socialization skills or reduced autism symptoms.”
Shortly following the release of the Vice feature, Frances Verter, Director of the Parents Guide to Cord Blood (PGCB) website, which was associated with the Duke team, penned an editorial criticizing Merlan’s “gonzo journalism.” To Verter, the project’s demise was not due to the failure of the team to prove its hypothesis that stem cells can treat autism. Instead, the project’s closure was blamed on outside players:
“The first wave of opposition to the Duke EAP for Autism came from a few professionals who work in the cell and gene therapy (CGT) field,” she wrote. “They claimed that the price of the treatment was outrageous and that the program was taking advantage of vulnerable parents.”
Verter left out the other concern that stem cell biologists and experts like Arnold Kriegstein and Paul Knoepfler had stressed: with no plausible mechanism for it to work, and no compelling evidence through many trials, the hype around stem cells for autism could be impacting the credibility of the stem cell field while also causing harm to children and families.
Verter also asserted there was a second player in the program’s demise: media.
“The second wave of opposition to the Duke EAP for Autism came when a couple of CGT professionals recruited the help of a journalist at an on-line magazine which thrives on reporting about conspiracy theories,” she wrote, referring to Vice. “In October 2021 the autism treatments at Duke were splashed across the media as a “big business” centered around an “unproven treatment.”
Then the neurodiversity movement came under fire.
”The third wave of opposition came when the press coverage reached the community of advocates for neuro-diversity. [sic] These are organizations of adults on the ASD spectrum who argue that autism should be accepted, that they do not need to be ‘cured.’ …It is sad when adults that are on the high-functioning end of the spectrum attack people who are trying to help children that fall on the low-functioning end of the spectrum.”
Verter was partially right. The neurodiversity movement does not like it when young autistic children are subject to these types of experiments, nor the myth that we can be cured. She did miss the detail that Phases 2 and 3 trials were actually performed on children with IQs over (not under) 70.
In any case, neither neurodiversity advocates nor outspoken critics from the science community approved of the use of young children of any IQ as subjects for this research into the hypothesis that one could undo a child’s autism with an infusion of stem cells from a non-autistic child. It didn’t seem scientific so much as belief-based, harkening a story as old as the religious exorcism, with autism framed as the demon roiling through the veins of children, purgeable and replaceable through a transfusive miracle of medical science.
But while autistics and ethicists were pleased with the news of the EAP shutdown, Cryo-Cell probably was not. The company’s value tumbled by 45 percent between September 2022 and February 2023, the period when waitlisted families started receiving calls from Duke.
Cryo-Cell provided almost no public information about the shift, which was odd since Cryo Cell’s Securities Exchange Commission stock disclosures detailed tens of millions in deals with Duke, although when I emailed the company in early 2023 about the shutdown, they seemed to be distancing themselves from Duke. As Knoepfler has noted, Cryo-Cell’s SEC filings at that time indicated that the Duke deal “was continuing (although there are amendments) and clearly involves ASD and cord cells,” with the relationship with Duke referenced repeatedly in the documents. But the official relationship appeared to be shifting.
As to Bernie Marcus, the sole-source funder of the Duke team’s earlier experimental trials, there were no statements from his camp at the time, either. Marcus, a co-founder of Home Depot with an interest in both autism and stem cell technologies, had quietly withdrawn funding for the Duke team as of 2024, after funding tens of millions in studies in the previous decade.
The Duke project was always kind of an anomaly. First, it defied the FDA’s own boundaries on the use of stem cells. The FDA has typically restricted stem cell research to cases where there is no other reasonable standard of care, in life-threatening conditions. Outside of drug trials, the agency has only approved stem cells for use in “disorders affecting the blood forming system.” Autism is not such a disorder.
FDA approvals for Duke’s Expanded Access Program (EAP) had also seemed odd, as FDA regulations state that EAPs (which allow researchers to charge study participants for study costs) are only generally allowable by FDA for conditions where there is no standard of care and where the condition is life-threatening. The thing is, there are standards of care for autism, and autism is not a life-threatening condition. Furthermore, the research was being done on vulnerable subjects (children).
For years, the Duke team had been focused on a message of promise that their trials (called IMPACT) and then their EAPs could provide proof of concept—but according to Duke’s own trial results and letter to parents, they never did. To critics, it seemed like families had been paying for little more than faint hope. But how did this journey of hope (and hype) go so far? And what, ultimately, led to its end?
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 This section likely references Vice, as its headline is echoed in Verter’s description.
 Not only were neurodivergent people subjected to real exorcisms throughout history, but some still are. An evangelical Christian group founded in Denmark has recently traveled Europe offering exorcisms on autistic children (as well as gay people) by casting out “demons”. In a horrific case in 2004, a Milwaukee preacher was tried and convicted for suffocating an autistic child to death during an exorcism to “cast out the Devil”. In a way, autism scammers have reinvented the exorcism--instead of removing demons, they now promise to remove mercury, parasites or neurotoxins.