Duke Has Quietly Discontinued a Costly, Unproven Autism Treatment

For several years, parents of autistic children have paid between $10,000 and $15,000 to have their children undergo unproven stem cell and cord blood treatments at Duke University, through what’s called an expanded access program, or EAP. That practice has attracted criticism from observers and ethicists in the stem cell field, who have asked why Duke was charging money for a service when its own clinical trials have not been very promising. In recent months, Duke has sent letters informing parents that this program is no longer available to autistic children—raising new questions about what those parents, who’d been led to believe the treatment might be a panacea for their kids, will do instead. 

One of the more urgent questions is whether parents who can’t access the treatment though Duke will instead go to a for-profit partner with ties to the school. That would be Cryo-Cell International, which previously announced that it had entered into a licensing agreement with Duke allowing it to offer the same stem cell infusions in private, for-profit clinics the company has said it plans to begin opening this year. (Duke previously told Motherboard that “the 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.”) 

What many critics of Duke’s stem cell program are wondering is whether the end of the EAP could be a financial bonanza for Cryo-Cell, to which those parents may turn instead. (Cryo-Cell and its CEO did not respond to several requests for comment from Motherboard. In a 2021 press release, David Portnoy, the company’s CEO, framed the clinics as a way to treat a large body of patients in need of cellular therapies, saying, “We are quite pleased with this acquisition and look forward to providing access to FDA approved clinical trial programs to treat patients in need of cord blood cellular therapies as soon as possible. The number of patients waiting for treatment is large and growing.”) 

Duke University’s School of Medicine is home to the Marcus Center for Cellular Cures, which was established in partnership with and is largely funded by a grant from Home Depot co-founder Bernie Marcus, a fervent believer that stem cells are a cure for a broad variety of diseases. (Marcus credits stem cells with treating a chronic lung disease for him and helping his wife with a knee problem. Even prior to his involvement with stem cell research, he was also an influential philanthropic donor; he established the Marcus Foundation to focus on autism and rehabilitation programs for veterans, and founded the Marcus Autism Center in 1991, which is now part of Children’s Healthcare of Atlanta. He’s also given lavishly to Jewish and Israel-oriented projects, and to Donald Trump and other Republican politicians.)  

For years, Duke has run clinical trials on the effects of stem cells and other cells derived from human umbilical cord blood on issues ranging from autism to cerebral palsy in both children and adults. Much of that research has been conducted by Dr. Joanne Kurtzberg, a pioneering scientist and researcher who’s studied stem cells since the 1990s. ( (Some, but not all, of the research at the Marcus Center for Cellular Cures is focused on products derived from human umbilical cord blood; cord blood contains stem cells. Some of Kurtzberg’s work specifically uses monocytes from cord blood and mesenchymal stromal cells, which she, like other experts in the field, argues are distinct from a “stem cell.”) 

Duke also has an expanded access program, or EAP—a way to give people who didn’t qualify for clinical trials a way to access specific treatments, for a price. The use of EAPs isn’t unusual; also called “compassionate use,” the FDA says such treatments may be appropriate for severe or immediately life-threatening diseases, when patients can’t qualify for a clinical trial, and when, as the agency put it, “potential patient benefit justifies the potential risks of treatment.” (For example, there are drug companies which have gotten FDA permission to give patients experimental drugs on an EAP basis for serious diseases like ALS. A treatment for smallpox was given to monkeypox patients under an expanded access agreement.)  

Critics of Duke’s specific EAP, which was issued in 2017, have questioned why it was approved by the FDA at all, since EAPs are generally used for illnesses and conditions where another treatment is not available. (Experts also say that, in general, focusing on unproven autism “treatments” or “cures” can direct families away from more effective interventions that help autistic kids lead fulfilling lives, like modern applied behavior analysis.)  

Since Duke’s EAP was approved, too, their research has not shown encouraging results for autism specifically. In 2020, a randomized clinical trial at Duke of cord blood infusions for autistic kids ages 2-7 showed that the infusions “were not associated with improved socialization skills or reduced autism symptoms.”

“It’s not clear to me why this EAP was approved in the first place given the limited and generally discouraging clinical trial data from Duke,” Paul Knoepfler wrote in 2021. “Related trial data from others has been discouraging too.” 

Knoepfler is a professor at UC Davis in the Department of Cell Biology and Human Anatomy, and writes a widely read blog on stem cell therapies, The Niche, where in 2021 he called on the FDA to freeze Duke’s EAP, arguing it was being inappropriately commercialized..

“The spirit of allowing EAPs is definitely not for the sponsors to make big profits directly from it,” he wrote. “While EAPs naturally can have commercial repercussions and could even help biotechs in the long run, that is not the main purpose. Or it shouldn’t be.”

“Overall,” Knoepfler added, “the idea of hundreds or thousands of vulnerable kids being injected with an unapproved drug via an EAP potentially mainly for commercial reasons just doesn’t seem right to me.”

(The FDA would not comment on whether it had any role in the EAP ending for autistic kids, telling Motherboard in a statement, “As a general matter, the FDA is prohibited by law from acknowledging the existence of a biological product file, or if its existence is known, disclosing any information that may be contained in such application. The agency does not comment on interactions it may or may not be having with an individual sponsor, clinical investigator, or establishment.” Duke did not respond to a question from Motherboard about whether the FDA has communicated with them in any way about the EAP.)

Earlier this year, parents in stem cell Facebook groups began reporting that Duke had sent them letters citing that study and saying the EAP was no longer available for autistic kids. 

“The EAP is not currently open to children diagnosed with Autism Spectrum Disorder (ASD),” the letter read, according to a parent who reposted the text of the one they say they had received. “Results of our most recent randomized study of umbilical cord blood infusion in children with ASD did not definitively demonstrate benefit (Dawson et al, J Pediatrics 2020). Enrollment in a clinical trial provides the only access to cellular therapy at Duke for qualifying children diagnosed with ASD.” 

The significance of the Facebook posts was pointed out by Anne Borden King, a Toronto-based science writer, podcaster, and advocate; she’s the author of an upcoming book, The Children Do Not Consent, which looks at fake autism cures and products, and is the chair of a group called the Campaign Against Phony Autism Cures.  Borden King has been a vocal critic of the EAP at Duke, arguing that it’s inappropriate for Duke to charge money for a treatment that thus far has shown no benefit in clinical trials.

Borden King monitors several Facebook groups devoted to stem cells and related treatments. She noted that many parents didn’t seem deterred by the end of the EAP, or less determined to get their kids these infusions. 

“I only saw one parent who said, ‘If Duke is saying this doesn’t work, why are we still doing it?’” Borden King told Motherboard. 

Borden King also says that Duke is responsible for helping to stoke an unrealistic hope in these parents—that their children’s autism can be treated or “cured” with stem cells or cord blood treatments.  

“The Duke team built up this hope and hype through media coverage, communications with parents, communications in parent groups,” Borden King said. She’s concerned that parents will instead take their kids to an offshore stem cell clinic—there are several well-known ones—or else an unregulated one in the United States that may not even be offering what it claims. “And of course that’s a whole other risk for the kids.” 

Other knowledgeable observers agree.

“It's a positive development that the EAP has been stopped for kids with ASD,” Knoepfler told Motherboard. “It put kids at risk, there was false hope for families, and it was wasting people’s money.” 

Knoepfler points out, though, that Duke is continuing what he sees as unproven EAP treatments on a different patient population. 

“Continuing the EAP option for kids with cerebral palsy really doesn’t make any more sense,” he said. “The past Duke trial data on cord cells for CP was generally negative and now they have a newer study that isn’t encouraging either.” (The study looked at 91 toddlers, and found that while the treatment was safe and well-tolerated, “significant changes in motor function were not observed 6 months after treatment.” It did find, however, that children who were treated with cord blood from an unrelated donor did show a greater increase in gross motor function than untreated children. The Duke researchers said this was a positive sign should be further tested in a randomized, placebo-controlled double-blind trial. An independent observer wrote a commentary in the same issue of the medical journal where the findings were published, agreeing that the results justified moving to a randomized trial.) 

The main questions, though, revolve specifically around autism treatments, and the relationship between Duke and Cryo-Cell. As Motherboard previously reported, Joanne Kurtzberg, the Duke researcher, is also the medical director at Cryo-Cell International, a for-profit company headquartered in Florida. (Kurtzberg previously told Vice that her position at Cryo-Cell was the result of the company acquiring a cord blood bank, Cord:Use, which contracted with a separate cord blood bank that she’d founded at Duke. “The Cord:Use salary for that position was paid directly to the Family Support Program for the Pediatric Transplant and Cellular Therapies program at Duke,” she wrote, “and Cord:Use paid Duke for processing the units collected for their public bank. When Cryo-Cell bought Cord:Use, those functions and financial arrangements continued without change.”) 

Cryo-Cell announced in 2021 that it had entered into an “exclusive license agreement” with Duke, “which grants us the rights to proprietary processes and regulatory data related to cord blood and cord tissue developed at Duke.” It planned to use those technologies, it said, to open a stem cell infusion clinic. The company initially said it would open the clinic in January 2022, and has now pushed back the date until 2023; the first clinic will reportedly be located in Durham, North Carolina, where Duke is also located. 

In an exuberant investor presentation, which has now been taken offline, Cryo-Cell dubbed 2021 “our pluripotent year” and included a slide showing that it expected an annual revenue of $24 million per clinic per year, based on the number of children diagnosed each year with autism, cerebral palsy, and traumatic brain injury. 

Treatment for autism still seems to be a large part of what Cryo-Cell envisions for its future. In a March SEC filing, Cryo-Cell reported that on February 17, 2023, the company entered into a second licensing agreement with Duke. The filing said that Cryo-Cell would pay a new fee to Duke “upon FDA approval for the first licensed product comprising cord tissue derived MSC (“ctMSC”) for autism spectrum disorder.”

The new amended licensing agreement also added that the company will, Cryo-Cell wrote, “open a manufacturing facility for the licensed product prior to the initiation of a Phase III clinical trial using ctMSC’s.” 

Finally, the SEC filing indicates that Cryo-Cell will now provide funding to complete one of Duke’s studies, the IMPACT study, which is studying the effects of treatments dervied from mesenchymal stromal cells, a type of cell derived from human umbilical cord tissue, on autistic kids ages 4-11. That agreement was effective on March 2, 2023, according to the filing. The company will make 14 equal monthly payments to Duke “commencing in April 2023,” the filing stated, “and a final payment upon the submission of a draft proposed publication for peer review and delivered to the Company of the completed IMPACT Study. Duke agrees this will be no later than September 30, 2024. The Data Safety Monitoring Board for the IMPACT Study recently has determined that the trial’s targeted accrual has been reached and consists of 137 patients.” 

On its website, under a section labeled “innovative therapies,” Cryo-Cell stated clearly that it believes it can use Duke’s EAP to offer similar therapies at its own clinic. 

“Cryo-Cell also plans to open clinics where patients can receive these treatments on clinical trials or as part of an expanded access protocol before they are approved by the FDA as standard treatments,” the page reads. “Leveraging the Duke agreement, Cryo-Cell plans to explore, test, and administer umbilical cord blood and cord tissue derived stem cell therapy treatments to patients with conditions for which there are limited FDA-approved therapies, including cerebral palsy, autism, and other neurological conditions.” 

(There is an asterisk next to the word “standard treatments” that reads, “Patients do not have to be customers of Cryo-Cell’s cord blood and cord tissue banking services to have access to treatment.”)

In response to questions submitted to Duke Health’s media office, the university gave responses attributed to Kurtzberg. Asked when the decision was made to discontinue the EAP program for autistic kids, and what information factored into it, she responded, “Several hundred patients were accrued to the EAP protocol, providing sufficient data to inform the design of future phase 2 and 3 clinical trials.” 

Duke and Kurtzberg declined to comment on a follow-up question about why the EAP continued until the end of 2022 if stem cell infusions were found to be ineffective for autism in a 2020 study.

Kurtzberg said follow-up care is being provided to the patients who were part of the EAP. “According to the formal protocol under the IND with the FDA, previously treated patients are followed with periodic, remote check-ins with study staff for two years.”

For now, Duke is continuing to study the benefits of cord blood treatments on toddlers, young children, and young adults, Kurtzberg added, but the toddler and young child trials are no longer taking new participants: 

Cryo-Cell did not respond to multiple requests for comment from Motherboard about what the Duke EAP decision means for the company or its stated plans to offer infusion treatments for autism. Its CEO, David Portnoy, also did not respond to direct requests for comment. Duke also refused to comment or provide comment from Kurtzberg about the university’s relationship with Cryo-Cell, writing only, “Please direct questions about Cryo-Cell to the company. Duke Health has no further comment beyond the responses we sent previously.” 

The lack of clarity has left some observers wondering whether parents who can no longer pay to have their kids treated at Duke will simply go to Cryo-Cell instead.

Leigh Turner is the executive director at the bioethics program at University of California Irvine and a professor in its public health department. He’s studied the stem cell industry for years, and says that the end of the autism-specific EAP at Duke could, “in a way, manufacture demand for Cryo-Cell,” or for similar companies abroad.   

“Are parents who are convinced this is a treatment they want to get for their children going to go to Panama or other jurisdictions?” he added. “That’s a possibility.” But there is also the possibility, Turner adds, that “It may also reduce the numbers. There may be individuals who aren’t willing to get on a plane to go to Panama. There could be a diminution.” 

“Panama” refers to one of the best-known stem cell clinics outside the United States, the Stem Cell Institute in Panama. Marcus, the Home Depot co-founder, has been treated there, and as Business Insider reported in 2021, the institute’s founder, Neil Riordan, counts Marcus and a number of celebrities as friends. In a response to another article written about his clinic, Riordan claimed not just to know Marcus, but Kurtzberg at Duke, and even claimed to have reviewed Duke’s proposal to the Marcus foundation for funding.

“I know Dr. Kurtzberg at Duke,” he wrote. “Bernie Marcus, who funded the Duke trials, has been a patient in Panama (public information) and saw children with autism benefitting from treatment with umbilical MSCs first hand. I was asked to review the Duke proposal for the Marcus Foundation and was at the Foundation Board meeting that led to the funding.”

In other words, the decisions made at Duke, and the research findings there, clearly have implications outside of academia, in a more profit-driven sector of science. For that reason, Anne Borden King says, Duke should continue to be extremely clear with parents that cord blood infusions have no proven benefit for their kids before they keep spending their money on these treatments.

“They’ve led people down this trail of hope,” she says. “It is their responsibility to be clear with parents so they don’t continue down this road.” 

Alycia Halladay is the chief science officer at the Autism Science Foundation, which also expressed concern about the EAP program. ASF funds autism research, provides evidence-based guidelines for parents on treatments and behavioral therapies that could help their kids, and speaks out frequently on what it calls “non-evidence based treatments.” It’s previously said of stem cell therapies, “There is no evidence that the treatment is safe or effective for autism, and no guarantee that the stem cells used in these countries are even human.” 

“We are relieved to see that children are no longer participating in the expanded access protocol which charged thousands of dollars to parents of children to receive stem cell therapies which have not shown to be effective,” Halladay told Motherboard. “The marketing of this product for the treatment of ASD was incredibly premature and not in the best interest of the community.  If it were to restart, it would need to be justified by a randomized control trial to prove the efficacy for those with ASD and clarify who on the spectrum would benefit from such treatment.”

She added, “Unfortunately, Cryo-Cell’s relationship to Duke University led families to believe that it had been studied to the level of scientific rigor needed to ensure it was both safe and effective.   We hope that families who have received this therapy continue to be monitored for longer-term effects, including adverse events.”