Project Cancelation & Beyond: Part 7 of The Rise and Fall of “Stem Cells for Autism” at Duke University
Where do failed autism research projects go to die?
Sometimes they float through the virtual world for decades, like Andrew Wakefield’s “autism-vaccine” paper, waiting for someone to come back round the barn and issue a merciful retraction. Other times, like the research around chelation for autism, they enjoy a brief moment in the sun before they’re crushed with the swish of an NIH bureaucrat’s pen.[1]
But no matter how they seem to end, they leave digital fingerprints. The internet, after all, is forever. The hopeful early messaging from these projects enters the public consciousness and its hold is strong. The Cryo-Cell message sold families on hope, a hope that was narrowly circumscribed through its own suite of products. The messaging was so strong that despite the changes in Duke’s program status, parents are still buying into that brand of hope, only now at unlicensed, direct to consumer clinics.
The end of the EAP
In early 2022, the FDA performed an onsite audit of Duke’s autism-stem cells EAP, which by then had infused close to 600 young children with stem cells at a total cost to families of somewhere around $9 million. At the same time, the IMPACT clinical trials, which had been funded by the Marcus Foundation, ran into a funding slump; according to Frances Verter, a physicist who supported the project, Cryo-Cell had infused the project with funds in mid-2022.
Soon, the Duke team ceased its EAP. When I inquired as to how and why, the FDA directed me to its web page with general information about the criteria for when FDA may delay a proposed clinical investigation or suspend an ongoing investigation. In its official reply, it also noted:
“In certain situations the patient’s doctor, the Institutional Review Board (IRB), or the sponsor may recommend that use of the investigational product be discontinued.”
Duke’s letter to families did not make it clear who pulled the plug on the project. So I contacted Duke, too. The university responded to my questions by stating:
“The FDA has not cancelled Duke’s Expanded Access Protocol for children with autism spectrum disorder. Duke elected to permit access to the therapy solely through its phase 2 randomized clinical trial…conducted under Investigational New Drug (IND) applications through the FDA.”
Per its letter to parents, Duke seemed to acknowledge that the EAP shut down, at least in part, because the results of the clinical trials were just weak. In that letter, the team had 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”). As well, the funder for the first four trials, Bernie Marcus, had announced his funding would cease in 2024, and the earlier shining hope of federal regulators approving dedicated clinics perhaps had become a dim mirage.
So, who ended it? We may never know. While Verter had claimed that “sabotage by competitors” and “gonzo journalism,” I suspect that, far from being catalysts, the program’s critics were contributing to a broader conversation that was already happening. Is it accurate to blame the fall of the program on its critics or on the failure of its team to demonstrate evidence of benefit? Or, could it be a little of both?
But the important question to me has never really been “why was the EAP canceled?” Rather, the question that still haunts me is “why did the FDA and IRB allow it to begin with?” The Duke EAP contradicted the agency’s own requirement of EAPs—that they be for life-saving medicine and for conditions where there is no adequate standard of care. With the trials funded by a sole source donor and investors eager for a licensed product, perhaps the IRB lost sight of the project’s impact on the hundreds of children used as experimental subjects. Perhaps, too, this is easier when the research subjects are autistic kids, due to pre-existing societal biases.
Regardless, and despite negative findings of its four previous clinical trials and the end of the autism EAP, the Duke team launched another trial in 2023 with the hope that this trial would show evidence of benefit. (They also continued to look at whether stem cells could improve other, unrelated conditions). But a look into Duke’s public records showed that Marcus Foundation funding was drying up. As Verter put it: “Mr. Marcus is getting on in years, and his Foundation is undergoing a reorganization to a more formal management structure.” With no more parent funding through the EAP and no more funding from Marcus, the Duke team will now have to search for new benefactors or give up the ghost.
Two narratives--and a lot of hype
In previous posts, I described the success of Stem Cell Institute of Panama and the continued marketing by US unlicensed clinics as part of a ripple effect from the hype around the Duke project. I’ve pointed out, as have other critics, that project leaders have an ethical obligation to clarify when their studies don’t show benefit. Did the Duke team do an adequate job of that?
Having followed media coverage of the Duke project from its beginning, bioethicist Leigh Turner believes the Duke research team participated in media stories coverage that made “hyperbolic claims” about stem cells for autism. “It’s unsurprising,” he told me, “that other parties are happy to take advantage of such hype and put it to commercial use.”
Throughout the IMPACT trials, we’ve been witness to two separate narratives from two connected stakeholders: Duke and Cryo-Cell. On the one hand, we have Duke’s official materials describing Phase III and IV study as being about safety of using stem cells on autistic kids, with the team making no claims that their study is measuring effect on autism symptoms at all.[2] In contrast, Cryo-Cell continued leveraged parents’ emotions while imbuing its products with a promise of hope for changing children’s behaviour.
As we saw in previous posts, Cryo-Cell’s marketing is based on a false idea that autistic children cannot feel or express love unless they have been fixed.[3] This is the idea that powers so much of autism pseudoscience—that a cause was found, that a cure was developed and the children will be cured of autism. In 2021, Cryo-Cell stated plans to become “an autonomous, vertically integrated cellular therapy company that will treat patients,” with clinic space devoted to autism-related infusions.
The company also positioned stem cells as a potential treatment for many conditions not approved by the FDA. It was making a big promise: that infusing autistic children with cord blood could transform them into more expressive, loving children. As children’s advocate Melissa Eaton, who has been inside the parent groups for years, told me:
“Parents in the groups I followed expressed again and again their hope that stem cells would enable their children to speak more, to have less sensory sensitivities and to have cognitive improvements.”
In the Facebook groups, parents struggled to piece together treatment programs for their children and to find signs of change—many of which could be imagined through a placebo effect. Even the smallest shift in behaviour would take on a larger meaning for some, things like trying a new food in the weeks after an injection, for example.
The name of Duke’s centre, The Marcus Center for Cellular Cures, also belies the nature of what’s happening within the centre’s walls. Research is “to find out if this thing even works,” bioethicist Jeremy Snyder says, “If you’re getting ahead of yourself and calling it a cure, that’s deeply unethical.” When asked about this by Vice, Duke’s media team stated that: “‘Cures’ is an aspirational term, not a statement about the current standard of care.”
The payout for parents’ efforts to fund autism-stem cell infusions, whether through GoFundMe (or, like one parent discussed in this series, through a cashed-out life insurance policy) ended up being nil. “Many parents [are] paying large sums because they are convinced that they are helping their children undergo an effective treatment for autism,” said Turner in 2021. “They are instead paying for access to an experimental intervention that might have no benefit.”
“Uniquely human”: Understanding the experiences & rights of autistic children
Although some researchers have presented autism as if it were cancer or another condition in which stem cells are legitimately used, autism—it should go without saying, really—is not cancer and it cannot be removed or replaced. Yet too much of research, whether focused on prenatal testing, diagnosing or treating autism, seems to approach autism as an entity unto itself—removable, transformable, exorcisable—rather than an immutable part of someone’s being.
The narrative of autism as a removeable entity is not only wrong, it is also anathema to promoting a healthy sense of self for the child and even a healthy relationship with parents. Psychologist Barry Prizant expresses this in his seminal work on autism, Uniquely Human:
“Our attitudes about and perspectives on people with autism and their behaviour makes a critical difference in their lives and in ours.”
Prizant’s use of the word human in his book title is not happenstance: it is in response to a research and services world that too often has dehumanized autistic children. As we see in the next chapter, the work of early researchers consistently portrayed autistics as incorrigible “monsters” or as mouldable, non-human subjects in need of behaviourist training.
That approach has translated into the deficit-based model of autism services and research, where autistic children were seen as deficient beings in need of repair, rather than human beings with both strengths and challenges. It is no surprise that the critic on the petition saw the Duke project as treating autistic children as though they were lab rats. Given this history and the children’s lack of agency, it is an apt metaphor.
When I look back at the past few years of researching Duke’s project I am left with the question: Why? What is there to show for all the years of study, the tens of millions spent, lives upturned and families’ bank accounts cleared out in the quest for autism hope? As I write this, parents are continuing to take a leap of faith, proxy consenting to risky stem cell procedures on their kids at unlicensed clinics that were nourished by the near-decade of hype around the Duke trials. These parents have been persuaded by false hope to sign the dotted line. If the children aren’t given the option to consent, who will say no for them?
This has been the seventh& final instalment in a series about Duke University’s experiments on autistic children, in which young children were injected with stem cells to see if it would stop them from being autistic. The series looks at the nine 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.
Miss a section? Start reading at the beginning.
More topics to come in 2024!
[1] Please see chapter 5 for discussion of both Wakefield’s paper and the chelation-for-autism debacle.
[2] According to their materials, Duke’s Phase III trials were based around the goal of determining “the safety and tolerability of a single intravenous dose of Human Umbilical Cord Tissue Derived Mesenchymal Stromal Cells (hCT-MSC) in adults with autism spectrum disorder (ASD).”
[3] This is a longstanding myth that was harkens back to Ole Ivor Lovaas’ reading of autism in the 1960s, as I discuss in Chapter 5 of the book.