Controversial cord cell treatment backed by Duke University

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When desperate parents are looking for medical treatment for their kids, especially their autistic kids, they often do two things: They look up information about a program at Duke University, and then, in short order, they go to GoFundMe. The fundraising site is full of pleas that often mirror each other, in essence saying: My child has one hope, and that hope is in stem cell and cord blood treatments.

This hope, though, is attached to treatments that are hotly disputed at best, unproven at worst, and often very expensive in either case. At Duke University’s Marcus Center for Cellular Cures, parents can enroll their children into a number of clinical trials that aim to study the effects of cells derived from umbilical cord blood on treating the effects of autism and brain injuries; adults can also participate in a trial testing whether cord blood can help them recover from ischemic strokes. And when parents can’t get their children into any of these clinical trials, particularly for autism, they often opt for what’s called the Expanded Access Program (EAP), in which they pay between $10,000 and $15,000 to get their kids a single infusion. 

All of the trials use products derived from human umbilical cord blood, which is a source of stem cells as well as other types of cells. The autism trials are using a type of immune cells called monocytes, according to Dr. Joanne Kurtzberg, a well-respected Duke professor who’s conducting clinical trials into whether cord blood can help with autism, and who has been researching stem cells since the early ‘90s.

Now, a for-profit company called Cryo-Cell International with ties to Duke researchers has indicated that it plans to open clinics promoting these treatments, under a licensing agreement with the renowned North Carolina university.

Duke and Cryo-Cell’s rush to monetize a procedure before it’s shown to have solid benefits has created concern, though, across the community of scientists, clinicians, and medical ethicists who study autism treatments.

The hope is that these cord blood infusions can improve some autism symptoms, like socialization and language, or decrease the inflammation that some parents and clinicians think might exacerbate autism symptoms. Early study results, however, haven’t been very promising. A large randomized clinical trial, the results of which were released in May 2020, showed that a single infusion of cord blood was not, in the words of the researchers, “associated with improved socialization skills or reduced autism symptoms.”

This is why Duke’s latest move comes as such a surprise: The university and Cryo-Cell have told investors that they’re planning to open a series of “infusion centers.” At these clinics, Cryo-Cell will use Duke’s technology and methods to offer cord blood treatments for $15,000 per infusion.

In an exuberant presentation for investors, Cryo-Cell said it estimates an annual revenue of $24 million per clinic; it hasn’t disclosed how many clinics it plans to open.

The move follows a June 2020 announcement that Cryo-Cell had entered into an exclusive patent-option agreement with Duke, allowing it to manufacture and sell products based on patents from Dr. Joanne Kurtzberg.

Alycia Halladay is the chief science officer at the Autism Science Foundation, which seeks to guide families into safe, evidence-based treatments for autism. “While stem cell therapies as medical intervention for the ASD are currently being rigorously studied at Duke, it is still too early to say they actually help those on the spectrum,” she told Motherboard. “Making the move at this time to sell this therapy to families across the world is irresponsible and dangerous. It shocks and surprises me that Duke University, an institution with a stellar academic reputation, would enter into an agreement to sell intellectual property for stem cells in autism, especially since Duke has enormous financial incentive to make this profitable for Cryo-Cell.”

“I was horrified when I first heard about the Duke program,” said Anne Borden King. She’s a Toronto-based science writer and the current chair of the Campaign Against Phony Autism Cures. She also hosts the podcast “Noncompliant,” about neurodiversity, where she’s frequently spoken critically about the Duke program.

Borden King has come to believe that Duke is leveraging its reputation to run a program that’s far from ethical. “When you see a university name on a project, you think everything must be on the up-and-up,” she said. “But that’s actually not a safe assumption. Money talks.”

The idea of using therapies derived from umbilical cord blood to treat serious disorders isn’t new, and neither is the controversy around it. There are small stem cell clinics across the world, from Mexico to Panama to the Caribbean, making a host of shaky claims about the benefits that stem cells from cord blood can provide, both autologous ones (stem cells that come from the patient themselves, taken from cord blood banked at birth) and allogeneic (stem cells that come from someone other than the patient). In the past decade, small stem cell clinics have also begun to dot the United States, mostly in strip malls and similarly downmarket locales, offering treatment for things like back pain

Though stem cell clinics have been around for years, the sheen of academic and scientific legitimacy bestowed by Duke onto experimental stem cell and cord blood treatments is very new indeed.

As the cord blood market has grown, autistic children—and their often desperate, always well-meaning parents—have since begun to comprise a core market for those therapies, even as the benefits of the treatments remain largely speculative. And as Business Insider’s Tom Porter reported in January, experts in the field say that the fly-by-night clinics in other countries are getting an unintended legitimacy boost from Duke. In GoFundMe appeals, parents talk about going to overseas clinics after being unable to get into Duke, or because they felt they saw promising results from a Duke infusion and wanted another.

Autistic kids and their parents are already extremely vulnerable from the start, Borden King says, due to the sheer amount of misinformation and pseudoscience that’s directed their way. She began running the Campaign Against Phony Autism Cures three years ago, after her son received an autism diagnosis.

The results were unambiguous overall, in the view of experts in the field. “This is a uniquely solid study in this area so in my view there’s not much room for hedging bets like ‘Well, maybe trying things a little different will work’ anymore,” Paul Knoepfler wrote on The Niche. “The key data in the paper are in Table II (screenshot below) showing across the board that there is no benefit of cord blood for these patients.”

“The first thing that jumped out at me when I read Duke’s study was they reported kids having health issues during infusion, like severe allergic reactions—and also being agitated and upset by the procedure,” Borden King said. (Duke’s researchers said some children experienced “agitation during IV placement and infusion” and one child experienced an allergic reaction, which was immediately treated, but they characterized most of the adverse events seen during the study as “nonserious,” and that overall the infusions were “safe and well tolerated.”)

There’s one main reason why Duke has been able to run so many clinical trials relating to stem cells: Bernie Marcus, the Home Depot founder. Marcus suffered from bronchiectasis, a condition where the lungs become damaged and clogged with mucus, and sought treatment from the Stem Cell Institute (STI) in Panama, according to a blog post on its website. STI is controversial, to put it lightly.

Bernie Marcus’ efforts in this area led, in 2018, to the founding of the institution at Duke that bears his name: the sunnily named Marcus Center for Cellular Cures.

“I think [that name] is hugely problematic,” says Jeremy Snyder, a professor in the Faculty of Health Sciences at Simon Fraser University whose research focuses on public health ethics and medical tourism, among other topics. That’s a longstanding complaint,” Snyder says, of the Marcus Center’s incredibly optimistic name. “You’ll see this in the cancer realm quite a bit. Marketing divisions of large hospitals put out commercials and different marketing materials, and you get a lot of that misleading language, playing on these tropes of hope, fighting, cures

As the years have passed, Turner, Knoepfler, and Snyder have all begun asking the same basic question about the Duke program: Why is an intervention that hasn’t shown much in the way of results not only still operational, but also charging participants a lot of money to get access to it?

In a recent blog post on The Niche, Knoepfler called on the FDA to freeze Duke’s EAP and closely examine its deal with Cryo-Cell.

In the end, the issues here extend beyond just Duke, and even the world of stem cell research, Borden King argues, noting they touch on the very nature of scientific credibility itself.

“When people think of pseudoscience, they tend to think of things like a cheesy salesman selling fake pills online,” she said. “But in my work, I see quite a few examples of autism scammers hiding behind university credentials, building hype through a university. This all has a broader impact on the credibility of our universities, which are supposed to be grounded in science. Especially at this time, when we’re fighting a pandemic and we need clear boundaries around science versus pseudoscience, the impact of projects like this just really ripples out.”

A full statement from Duke University is below:
Researchers at Duke University have studied the use of cord blood cells for pediatric blood cancers and have explored potential therapeutic applications of these cells for neurological disorders in children arising from inborn errors of metabolism, traumatic brain injury, cerebral palsy, autism and neuroinflammatory conditions.

Duke researchers are active in conducting randomized controlled clinical trials evaluating cord blood cell therapies for these applications. Data from the studies are submitted for publication in peer reviewed journals and are rigorously assessed by the scientific community and the FDA. Much remains to be learned about how these cells affect the brain and about clinical outcomes, and Duke investigators are committed to ongoing research to close these gaps in knowledge.

For some children who do not qualify for clinical studies, but who have few other options for treatment of their conditions, the FDA has authorized Duke to offer cord blood cells through an expanded access protocol (EAP) monitored by the FDA.

In recent months, Duke has entered into a licensing agreement with Cryo-Cell International to provide regulatory information and other data that would help advance the company’s effort to develop a cell therapy program eligible for approval from the FDA. This licensing agreement does not grant Cryo-Cell the use of Duke’s EAP for the treatment of patients at Cryo-Cell, but will allow Cryo-Cell to develop its own cell therapy program.

Duke is committed to ensuring that scientific evidence guides the further study and use of these investigational therapies. As new findings provide additional insights, Duke will continue to evaluate how best to move forward, both with research and with FDA-approved clinical applications.

deeply disturbing on many levels. thanks as always for posting timely information.