A Paradigm Shift in Autism Research: Pt 6 of The Rise and Fall of “Stem Cells for Autism” at Duke University
This is the sixth 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.
A shift in research: Acknowledging children’s rights and embracing participatory research
The controversy around Duke’s failed stem cells-autism trials is not just about the risks of the procedure, nor is it merely about the dangers of selling false hope to parents for $15,000 per family. The trials are a touchstone for broader issues on how and why autism research gets funded, the rights of autistic and child research subjects, and the emerging subjectivity of autistic people within research.
Basic ethics tell us that clinical trials should always be performed on adults, when possible, rather than children. But in what special cases is it ethical to run a study on non-consenting children? If it involves potentially lifesaving medicine, it may make sense, depending on matters of plausibility and risk. However, using stem cells on autistic kids has no lifesaving potential at all, in addition to carrying significant risk and having no plausibility.
So, why did Duke’s Institutional Review Board (ethics board) allow the team to use children as subjects? Why did the FDA approve the initial trials? And then why did the FDA approve a pay-to-play trial, where parents paid $15,000 for a doomed and dangerous experiment? Why wasn’t the issue of proxy consent flagged by anyone involved? Why did it take an uproar from stem cell biologists, ethicists and the autistic activists to shut it down, after two years and millions of dollars, from hundreds of families, poured down the drain?
The problem with the proxy consent loophole
It is clearly impossible to find a pool of freely-consenting autistic adults to volunteer for such a flawed and risky study. Should we view the impossibility of finding consenting autistic adults for the Duke experiments as merely a roadblock that researchers had the right to dodge, through a proxy process, or was that very lack of a freely-consenting population a critical piece of information that should have been carefully weighed alongside the program’s risks and benefits?
With no informed, consenting autistic adults who would agree to the study, the Duke team’s only way to gather study subjects was via what I’ve labeled the “proxy consent loophole”. The proxy consent loophole builds off legitimate uses of proxy consent (such as a parent consenting to emergency surgery for their baby) and expands it to justify parents proxy consenting to have their children enrolled in non-essential, experimental scenarios that no child would ever choose to endure. In this case, parents were convinced to consent on their children’s behalf, in part, by the rhetoric of hope that the Duke and Cryo-Cell team were spreading through media and the Cryo-Cell website.
But the children did not consent.
Where does this all take us from a legal and rights perspective? If the only way to recruit the study participants was through the proxy consent of parents being promised “hope,” was the study ultimately coercive? Were the children’s rights violated? Could they file a human rights related class action lawsuit? If Duke’s research did not meet the needs of participants, whose needs did the research meet?
What the Duke debacle has made clear to me is that despite decades of ethical discussions around issues of informed consent for human subjects in drug trials, the autonomy and informed consent rights of autistic children have too often been ignored.[1] More than half a century ago, the Belmont Report of the National Commission for the Protection of Human Subjects was established to prevent exploitative research. The report defined the terms of “diminished autonomy” to include “circumstances that severely restrict liberty.”
Children clearly have diminished autonomy, not only because they lack the experience and cognitive capacity to make many informed decisions, but because their autonomy is also severely limited within a research structure that offloads their consent to the nearest proximate caregiver. The Belmont Report would indicate that the default proxy answer for children to participate in a study like the Duke stem cell trials is a clear “no.”
A paradigm shift, towards participatory research
When media reported on the closing of the team’s EAP--six months after parents received their cancelation letters--its sole defense came from the website of a project supporter, physicist Frances Verter, who claimed the project had been “sabotaged by rival researchers…targeted by a magazine that prides itself on gonzo journalism,” and that “high functioning” autistic people had been part of the conspiracy.
Verter’s two-dimensional critique sidesteps the fact that the mess at Duke signifies something larger than itself. If we look to the comments on the petition against Duke’s trials, we see deeper questions about the nature of autism research itself. “My son deserves the right to be himself,” writes one petition-signer. “So do the children being subjected to this procedure.” An autistic signer was even more direct:
“Autistic people don’t need a cure. We are not your lab rats.”
Until very recently, much autism research has focused on autistic participants as objects of study (like the “lab rats” comment suggests)--not as active participants with the right to consent. It is even more rare to see autistic adults advising on the course of a project or contributing to the discussion about research goals. This is frankly astonishing considering that autistic people are being portrayed as the end users of the therapies being trialed—this is, supposedly, all being done in the name of curing them!
But here’s a thought exercise in trying to imagine the almost unimaginable: Envision a scenario in which researchers communicate with autistic children to ask them what is missing in their lives and what kind of research questions they should explore to begin with. This experiment is being played out in real time for adult autistics, through new excursions into participatory research. As researcher Kristen Bottema Beutel of the Lynch School of Education has described it:
“Exclusively focusing on deficits does not represent autistic people or autism accurately; instead it reflects the interests of primarily non-autistic researchers. …The assumed validity of these theories further encourages poor research practices and confirmation bias (e.g., elective reporting, p-hacking, hypothesizing after results are known, etc.). …In contrast, autistic accounts of autism tend to be broad in scope, rather than deficit-focused. [C]o-produced work tends to advocate for a holistic approach to understanding autistic strengths and challenges.”
We are at the dawn of participatory autism research, with a growing openness to research that considers the research priorities of autistic people themselves.[2] Participatory research goes beyond merely surveying autistics on research priorities towards being more collaborative, with researchers even providing training modules for research participants so that, as persons outside of academia, they can understand the research process, which informs their involvement in the project.[3]
As Shannon Rosa, editor of The Thinking Person’s Guide to Autism, who has been attending the International Society for Autism Research (INSAR) since 2011, told me:
In recent years, we have more autistic researchers and more autistics in general involved in directing the course of research. And with this shift has come a focus on improving quality of life, because that's what autistic people themselves tend to want from research.
Rosa has been observing changes in the course of autism research, away from cure-based explorations and towards quality of life-focused projects, as well as studies on co-occurring conditions such as epilepsy. Rosa notes that a decade ago, most INSAR presentations were still focused on treating “symptoms” of autism itself. But within the broader scientific community, that language has been changing as some research moves away from a cure-based approach (what some call the medical model of autism) towards a disability-access focus, where researchers focus on quality of life (what some call the social model of disability).
To Rosa, it is a seismic shift in the focus of autism research over the course of just a few years.
“We’ve learned a lot through participatory research about the actual needs of the autistic community and again and again we see that autistic people aren’t looking for a cure for who they are.”
INSAR has even adopted some of the communication and sensory accommodations that have been in practice at autistic-led gatherings for years, such as sensory-friendly quiet rooms when people need breaks. These changes have come from neurodiversity advocates and activists, as well as an influx of researchers who are openly autistic themselves. As autism research becomes more welcoming to autistic researchers, we can see more emerging autistic scholars migrating from other areas of research towards autism-specific research. This is changing the pitch of autism research away from cause/cure, towards a quality of life focus.
Rosa has also been co-host of AutINSAR, a series of discussions in which autistic people and/or researchers and other community members get together virtually during the INSAR conference to discuss how autism research can better serve autistic people. According to Rosa, the most common research concerns raised by autistic people are around mental health (including suicidality), followed by sleep disruption, medication, best practices for job interviews and workplace accessibility, and research into seizures (a co-occurring condition for some autistic people).
The double-empathy problem
Autism researchers have also been looking at autistic communication in a new way: by studying and addressing communication differences between autistic and non-autistic people (sometimes called “the double empathy theory”), instead of focusing only on changing autistic people’s communication behaviours to mimic those of non-autistic people. Research that focuses on communication access also has clear results, for example in the development of communication technologies for non-speaking people (such as text-to-speech apps), across various disabilities.
New research also makes it clear that non-autistic people tend to understand each other better than they do autistic people—and that non-autistic people tend to misunderstand autistic people and even view them negatively.[4]
This raises the obvious question: If non-autistic people are misunderstanding what is essential for autistic people to have a quality of life, to self-regulate and to feel good, does it make any sense to have non-autistics determining the goals of autism research?
It is notable that there were no autistic advocates in support of the Duke stem cell experiments--because from an autistic perspective, these experiments looks like torture. The dominant view from our community is that research should focus on quality of life, rather than imagined autism cures and licensed hope-infusion clinics.
“I’m grateful to see autism research increasingly addressing my son’s traits through the lens of understanding rather than as a laundry list of ‘deficits’,” says Rosa, adding that parent-child relationships are strengthened when parents accept their child’s autism. As autism mom Ariane Zurcher told Alison Opar, writing for The Atlantic: “My entire focus changed. Instead of fighting against Emma’s neurology and trying to cure this heinous disorder, I started finding ways to help her flourish.” Opar continued:
“During a conversation in 2014 about past treatments, Emma seemed to understand exactly why her mother pursued so many therapies over the years. ‘You thought my autism was hurting me and that you needed to remove it, but you did not understand that it is a neurological difference,’ she typed. ‘Fear caused you to behave with desperation.’”
If researchers are allowed to tap into parents’ fears and sell them hope—rather than tuning into autistic people’s real lives and needs--the pseudoscience hype will continue to expand, including for clinics outside of regulators’ reach.
Meanwhile, the autistic children who are subjects of trials such as the Duke project are seeking something that can’t be commodified as a product or licensed to an infusion lab. They want to be accepted and included as who they are, not the imagined child that’s conjured in a laboratory.
Research based on this humanizing, participatory model centers autistic people as subjects, rather than objects to manipulate or “repair.” [5] Mary Doherty, a UK-based MD who is autistic, summed it up:
“As a community, autistic people have documented our research priorities, and…‘cures’ do not feature prominently. We want research that will directly and positively affect our lives.”
If researchers follow the lead of AutINSAR’s work, then projects such as the Duke-Cryo-Cell partnership could have a harder time gaining a foothold—or any institutional support—for their endeavors. As a parent now engaged in research, Rosa summed up her hopes for the future of autism research this way:
“I am furious every time a research project derails that future with a ‘cure’ approach. If I’m seeing $15 million in research, I’m asking, how could we be spending that $15 million to improve my son’s daily quality of life through supports and accessibility—not a wild goose chase with stem cells.
“‘Hope’ for me means a continuing social shift in understanding autism as a neurology to be accommodated.”
Coming Saturday. Conclusion: Where does failed autism research go to die?
[1] See the previous chapter and the next chapters for more examples.
[2] For more on autistic participatory research, which I discuss more in the last chapter of this book, please see the work of Sue Fletcher Watson on this topic.
[3] An example of this training is the Research 101 online module, which Jackie Ryan and colleagues have described in their work.
[4] Research (e.g., Sasson, 2021, Crompton, 2019, 2021) has shown that non-autistic individuals have trouble understanding how autistic individuals feel or what they think and tend to make snap judgements or impressions about autistic people, as well that the social interaction quality between two autistic people is actually as strong as between two non-autistic people. This knowledge can aid the broader body of research. I discuss many new projects further in the last chapter of my forthcoming book (2024): The Children Do Not Consent.