Moral points cloud case report of unproven stem cell remedy for autism | Spectrum
Cell Service: Parent reports suggest stem cell therapy may have reduced their children’s autism characteristics, but experts remain skeptical.
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Unknown financial conflicts of interest and a lack of adequate clarification affect a new study that injected four autistic children with stem cells from their own bone marrow, bioethicists say.
The study, published in Frontiers in Pediatrics in October, has not undergone ethical review and approval by an Institutional Review Board (IRB), a crucial step for research involving human participants.
“Although I am not familiar with their local or institutional regulations, I believe international standards require prior institutional review and approval for this type of experimental procedure,” said Paul Knoepfler, professor of cell biology and human anatomy at the University of California, Davis who blogged critically about stem cell treatments for autism. “There is also no good justification for the experimental treatment they used, which an IRB would have discussed,” says Knoepfler.
In addition, the study’s investigators neither made their connections to an Austrian clinic that sells the unproven therapy, nor did they reveal that the families of the four children paid for the injections – potential financial conflicts of interest. A 2019 independent study of unproven stem cell therapy for autism was withdrawn last week after it also failed to reveal that its participants had paid for the treatment.
The fact that the families of the participants paid for his stem cell procedure does not deny that the families of the study participants are Georg Kobinia, head of the new study, director of the Austrian Society for Regenerative Medicine. In response to email questions from Spectrum, Kobinia confirmed that the participants’ families had paid for the injections but did not respond to questions about the cost of the procedure.
The treatments are ethical, he told Spectrum, because they were only given after other autism therapies had resulted in no changes in children’s behavior and concurrent physical illnesses.
Still, the largest placebo-controlled clinical trial to date of stem cell treatment for autism showed no significant benefits, which calls into question the rationale behind the Kobinia team’s approach.
The children of the new study, aged 3 to 14, received the stem cell injections in Kobinia’s medical practice, Stem Cell Therapy Vienna in Austria. While the children were sedated, Kobinia and his team extracted bone marrow from each child’s hipbone, separated the stem cells, and gave them back to each child via both a spinal tap and an intravenous infusion.
Before treatment and at three-month intervals during the following year, the researchers asked the children’s parents to complete the Autism Treatment Assessment Checklist, a questionnaire asking about changes in a child’s autism-related behavior. None of the children’s parents reported results at all five time points.
All four children’s scores decreased after treatment, suggesting a reduction in autism characteristics and the severity of concurrent physical problems. For example, parents of one child reported an improvement in their son’s digestive problems, which are more common in autistic people than in their non-autistic peers.
The team describes its work as “very promising”. However, the case series does not compare the four treated children with placebo or treatment-as-usual controls, so it is not clear how they can say this or draw specific conclusions, says Knoepfler. “There is still a long way to go before the practice is ready to convince people that this is an effective treatment.”
Additionally, parent-reported results can be unreliable and introduce various types of bias into a study, says Kristen Bottema-Beutel, associate professor of teaching, curriculum, and society at Boston College, Massachusetts. Parents are concerned that their child will benefit from an intervention and can change the way they interact with their child, which can lead to positive change – a form of bias known as placebo-by-proxy, says she. And parents want their children to benefit from an intervention that, even in the absence of real improvements, may unconsciously give higher scores on reports after the intervention, an issue known as detection errors. Bottema Pouch studies conflicts of interest in autism research and is sometimes paid to speak on the subject.
KObinia says the stem cell injection he and his colleagues used is not “experimental therapy” and therefore does not require approval from an IRB. He also says that the retrospective nature of the case report precludes classifying these procedures as processes.
But for a retrospective case series, the children should have received the stem cells – with ethical approval – for a different condition, says Knoepfler. Then Kobinia and his team would have had to dig into the data afterwards to find out if the procedure had inadvertently improved their autism-related behavioral traits.
“Otherwise, anyone without an IRB license could conduct experiments on humans and then simply claim that since it was later reported in a manuscript, no IRB license was required,” he says.
Although the treatments were done in a doctor’s office that sells stem cell therapies for various conditions, and even though the families paid for the procedure, Kobinia and his colleagues reported in their study that “the research was done without commercial or financial resources.” Relationships that could be construed as a potential conflict of interest. ”When asked by Spectrum, Kobinia said there was no conflict because a doctor’s office is not a company or a clinic.
But claiming the location of a doctor “doesn’t solve the conflict of interest problem,” says Arthur Caplan, director of medical ethics at New York University in New York City. “That’s ridiculous.”
Cite this article: https://doi.org/10.53053/ARWI8127
