For a number of years, mother and father of autistic youngsters have paid between $10,000 and $15,000 to have their children endure unproven stem cell and cord blood treatments at Duke University, via what’s referred to as an expanded access program, or EAP. That follow has attracted criticism from observers and ethicists within the stem cell field, who have requested why Duke was charging cash for a service when its own clinical trials haven’t been very promising. In recent months, Duke has sent letters informing parents that this program is no longer obtainable to autistic children-elevating new questions about what those mother and father, who’d been led to believe the treatment might be a panacea for his or her kids, will do instead.
One of many extra pressing questions is whether dad and mom who can’t entry the remedy though Duke will as an alternative go to a for-profit accomplice with ties to the school. That would be Cryo-Cell International, which beforehand introduced that it had entered into a licensing agreement with Duke permitting it to supply the identical stem cell infusions in personal, for-revenue clinics the company has said it plans to start opening this 12 months. (Duke previously advised Motherboard that “the licensing agreement doesn’t grant Cryo-Cell using Duke’s EAP for the remedy of patients at Cryo-Cell, however will permit Cryo-Cell to develop its personal cell therapy program.”)
Videos by VICE
What many critics of Duke’s stem cell program are wondering is whether or not the top of the EAP may very well be a financial bonanza for Cryo-Cell, to which those parents could flip as a substitute. (Cryo-Cell and its CEO did not reply to several requests for remark from Motherboard. In a 2021 press release, David Portnoy, the company’s CEO, framed the clinics as a technique to treat a big body of patients in want of cellular therapies, saying, “We are fairly happy with this acquisition and look ahead to offering entry to FDA authorized clinical trial packages to treat patients in want of cord blood cellular therapies as soon as doable. The variety of patients waiting for remedy is giant and rising.”)
Duke University’s School of Medicine is residence to the Marcus Center for Cellular Cures, which was established in partnership with and is basically funded by a grant from Home Depot co-founder Bernie Marcus, a fervent believer that stem cells are a cure for a broad number of diseases. (Marcus credits stem cells with treating a chronic lung disease for him and serving to his wife with a knee problem. Even previous to his involvement with stem cell research, he was also an influential philanthropic donor; he established the Marcus Foundation to concentrate on autism and rehabilitation applications 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 each kids and adults. Much of that analysis has been carried out by Dr. Joanne Kurtzberg, a pioneering scientist and researcher who’s studied stem cells for the reason that nineteen nineties. ( (Some, however not all, of the research at the Marcus Center for Cellular Cures is concentrated on products derived from human umbilical cord blood; cord blood contains stem cells. Some of Kurtzberg’s work specifically makes use of monocytes from cord blood and mesenchymal stromal cells, which she, like different specialists in the field, argues are distinct from a “stem cell.”)
Duke additionally has an expanded access program, or EAP-a means to provide individuals who didn’t qualify for clinical trials a way to entry particular treatments, for a worth. Using EAPs isn’t unusual; additionally referred to as “compassionate use,” the FDA says such therapies could also be acceptable for severe or instantly life-threatening diseases, when patients can’t qualify for a clinical trial, and when, because the agency put it, “potential affected person benefit justifies the potential dangers of remedy.” (For instance, there are drug companies which have gotten FDA permission to give patients experimental medicine on an EAP foundation for severe diseases like ALS. A therapy for smallpox was given to monkeypox patients beneath an expanded access settlement.)
Critics of Duke’s specific EAP, which was issued in 2017, have questioned why it was approved by the FDA in any respect, since EAPs are generally used for illnesses and situations where another remedy is not out there. (Experts also say that, basically, specializing in unproven autism “treatments” or “cures” can direct families away from more practical interventions that assist autistic kids lead fulfilling lives, like fashionable applied habits evaluation.)
Since Duke’s EAP was authorised, too, their analysis has not proven encouraging outcomes for autism particularly. In 2020, a randomized clinical trial at Duke of cord blood infusions for autistic children ages 2-7 showed that the infusions “were not associated with improved socialization expertise or diminished autism signs.”
“It’s not clear to me why this EAP was permitted in the first place given the restricted and customarily discouraging clinical trial knowledge from Duke,” Paul Knoepfler wrote in 2021. “Related trial knowledge 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 extensively learn 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 permitting EAPs is certainly not for the sponsors to make large earnings immediately from it,” he wrote. “While EAPs naturally can have business repercussions and will even assist biotechs in the long run, that’s not the main function. Or it shouldn’t be.”
“Overall,” Knoepfler added, “the concept of a whole lot or 1000’s of vulnerable children being injected with an unapproved drug by way of an EAP potentially mainly for industrial causes just doesn’t seem proper to me.”
(The FDA wouldn’t comment on whether it had any role in the EAP ending for autistic youngsters, telling Motherboard in a press release, “As a basic matter, the FDA is prohibited by law from acknowledging the existence of a biological product file, or if its existence is understood, disclosing any information that could be contained in such application. The company does not touch upon interactions it could or is probably not having with a person sponsor, clinical investigator, or institution.” Duke didn’t reply to a question from Motherboard about whether the FDA has communicated with them in any means in regards to the EAP.)
Earlier this year, parents in stem cell Facebook groups started reporting that Duke had despatched them letters citing that study and saying the EAP was now not accessible for autistic kids.
“The EAP isn’t at present open to children diagnosed with Autism Spectrum Disorder (ASD),” the letter read, in response to a mum or dad who reposted the text of the one they are saying they had obtained. “Results of our most latest randomized study of umbilical cord blood infusion in children with ASD didn’t definitively reveal benefit (Dawson et al, J Pediatrics 2020). Enrollment in a clinical trial provides the one entry to cellular therapy at Duke for qualifying youngsters diagnosed with ASD.”
The significance of the Facebook posts was identified by Anne Borden King, a Toronto-primarily based science writer, podcaster, and advocate; she’s the author of an upcoming book, The Children Do not Consent, which seems at fake autism cures and products, and is the chair of a bunch 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 cost cash for a remedy that thus far has proven no profit in clinical trials.
Borden King displays a number of Facebook teams dedicated to stem cells and associated remedies. She famous that many mother and father didn’t seem deterred by the end of the EAP, or less decided to get their kids these infusions.
“I solely saw one guardian who stated, ‘If Duke is saying this doesn’t work, why are we nonetheless doing it?’” Borden King advised Motherboard.
Borden King additionally says that Duke is accountable for helping to stoke an unrealistic hope in these parents-that their children’s autism might be treated or “cured” with stem cells or cord blood therapies.
“The Duke staff built up this hope and hype by media coverage, communications with parents, communications in dad or mum groups,” Borden King said. She’s concerned that mother and father will as a substitute take their kids to an offshore stem cell clinic-there are several well-identified ones-or else an unregulated one in the United States which will not even be offering what it claims. “And in fact that’s a whole different risk for the youngsters.”
Other educated observers agree.
“It’s a optimistic growth that the EAP has been stopped for kids with ASD,” Knoepfler told Motherboard. “It put children in danger, there was false hope for households, and it was wasting people’s cash.”
Knoepfler points out, although, that Duke is continuing what he sees as unproven EAP therapies on a distinct patient inhabitants.
“Continuing the EAP possibility for kids with cerebral palsy actually doesn’t make any more sense,” he mentioned. “The past Duke trial information on cord cells for CP was typically unfavorable and now they have a newer examine that isn’t encouraging either.” (The study looked at 91 toddlers, and found that while the treatment was secure and nicely-tolerated, “significant changes in motor perform weren’t noticed 6 months after therapy.” It did find, nevertheless, that children who have been handled with cord blood from an unrelated donor did show a greater enhance in gross motor function than untreated youngsters. The Duke researchers mentioned this was a constructive signal should be additional examined in a randomized, placebo-controlled double-blind trial. An independent observer wrote a commentary in the identical situation of the medical journal the place the findings were printed, agreeing that the results justified shifting to a randomized trial.)
The main questions, though, revolve particularly around autism remedies, and the relationship between Duke and Cryo-Cell. As Motherboard previously reported, Joanne Kurtzberg, the Duke researcher, can be the medical director at Cryo-Cell International, a for-revenue firm headquartered in Florida. (Kurtzberg previously instructed Vice that her position at Cryo-Cell was the results of the corporate buying a cord blood bank, Cord:Use, which contracted with a separate cord blood financial institution that she’d based at Duke. “The Cord:Use wage for that place 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 items collected for his or her public bank. When Cryo-Cell purchased Cord:Use, those features and financial arrangements continued without change.”)
Cryo-Cell introduced in 2021 that it had entered into an “exclusive license agreement” with Duke, “which grants us the rights to proprietary processes and regulatory information related to cord blood and cord tissue developed at Duke.” It deliberate to make use of those applied sciences, it stated, to open a stem cell infusion clinic. The company initially said it might open the clinic in January 2022, and has now pushed back the date until 2023; the primary clinic will reportedly be located in Durham, North Carolina, the place Duke can also be situated.
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 yr, based mostly on the number of children diagnosed every year with autism, cerebral palsy, and traumatic brain harm.
Treatment for autism still appears 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 settlement with Duke. The filing mentioned 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 brand new amended licensing agreement additionally added that the corporate will, Cryo-Cell wrote, “open a producing facility for the licensed product prior to the initiation of a Phase III clinical trial utilizing ctMSC’s.”
Finally, the SEC filing indicates that Cryo-Cell will now present funding to complete one among Duke’s research, the Impact examine, which is learning the consequences of remedies dervied from mesenchymal stromal cells, a type of cell derived from human umbilical cord tissue, on autistic children ages 4-11. That settlement was efficient on March 2, 2023, based on the filing. The corporate will make 14 equal monthly funds to Duke “commencing in April 2023,” the filing acknowledged, “and a final cost upon the submission of a draft proposed publication for peer assessment and delivered to the company of the accomplished Impact Study. Duke agrees this shall be no later than September 30, 2024. The info Safety Monitoring Board for the Impact Study not too long ago has decided that the trial’s targeted accrual has been reached and consists of 137 patients.”
On its web site, below a piece labeled “innovative therapies,” Cryo-Cell said clearly that it believes it could actually use Duke’s EAP to supply similar therapies at its own clinic.
“Cryo-Cell also plans to open clinics the place patients can receive these therapies on clinical trials or as a part of an expanded access protocol before they are accredited by the FDA as standard therapies,” 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 remedies to patients with conditions for which there are restricted FDA-accredited therapies, including cerebral palsy, autism, and other neurological circumstances.”
(There is an asterisk next to the phrase “standard treatments” that reads, “Patients don’t should be prospects of Cryo-Cell’s cord blood and cord tissue banking companies to have entry to remedy.”)
In response to questions submitted to Duke Health’s media office, the university gave responses attributed to Kurtzberg. Asked when the choice was made to discontinue the EAP program for autistic youngsters, and what info factored into it, she responded, “Several hundred patients were accrued to the EAP protocol, offering adequate data to tell the design of future part 2 and three clinical trials.”
Duke and Kurtzberg declined to touch upon a observe-up query about why the EAP continued until the tip of 2022 if stem cell infusions had been discovered to be ineffective for autism in a 2020 examine.
Kurtzberg said follow-up care is being provided to the patients who have been part of the EAP. “According to the formal protocol beneath the IND with the FDA, beforehand treated patients are followed with periodic, remote check-ins with examine employees for 2 years.”
For now, Duke is continuing to study the advantages of cord blood treatments on toddlers, younger children, and younger adults, Kurtzberg added, however the toddler and younger child trials are not taking new members:
Currently, there are three trials underway for toddlers (TACT NCT04294290), young youngsters (Impact NCT04089579) and younger adults (Aims NCT04484077) with autism testing cord tissue MSCs, but the toddler and young children trials have completed accrual. These patients continue therapy and comply with-up. The grownup trial remains to be open to enrollment. Future cell therapy research might be based mostly on the outcomes of these trials.
Cryo-Cell didn’t reply to a number of requests for remark from Motherboard about what the Duke EAP choice means for the corporate or its said plans to offer infusion treatments for autism. Its CEO, David Portnoy, also didn’t respond to direct requests for comment. Duke also refused to comment or provide comment from Kurtzberg concerning the university’s relationship with Cryo-Cell, writing solely, “Please direct questions on Cryo-Cell to the company. Duke Health has no additional remark past the responses we sent beforehand.”
The lack of readability has left some observers wondering whether mother and father who can not pay to have their kids treated at Duke will merely go to Cryo-Cell as an alternative.
Leigh Turner is the govt director on the bioethics program at University of California Irvine and a professor in its public health division. He’s studied the stem cell business for years, and says that the tip of the autism-specific EAP at Duke may, “in a method, manufacture demand for Cryo-Cell,” or for comparable companies abroad.
“Are mother and father who’re satisfied it is a therapy they wish to get for their children going to go to Panama or other jurisdictions?” he added. “That’s a possibility.” But there can also be the possibility, Turner provides, that “It may also reduce the numbers. There could also be people who aren’t willing to get on a airplane to go to Panama. There may very well be a diminution.”
“Panama” refers to the most effective-recognized stem cell clinics exterior the United States, the Stem Cell Institute in Panama. Marcus, the house Depot co-founder, has been treated there, and as Business Insider reported in 2021, the institute’s founder, Neil Riordan, counts Marcus and quite a lot of celebrities as mates. 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 basis for funding.
“I know Dr. Kurtzberg at Duke,” he wrote. “Bernie Marcus, who funded the Duke trials, has been a patient in Panama (public info) and saw kids with autism benefitting from remedy with umbilical MSCs first hand. I was requested to assessment the Duke proposal for the Marcus Foundation and was at the inspiration Board assembly that led to the funding.”
In other words, the decisions made at Duke, and the analysis findings there, clearly have implications outside of academia, in a more revenue-pushed sector of science. For that motive, Anne Borden King says, Duke ought to proceed to be extraordinarily clear with parents that cord blood infusions haven’t any confirmed profit for their kids earlier than they keep spending their money on these therapies.
“They’ve led people down this trail of hope,” she says. “It is their accountability to be clear with dad and mom so that they don’t continue down this highway.”
Alycia Halladay is the chief science officer at the Autism Science Foundation, which additionally expressed concern about the EAP program. ASF funds autism research, offers evidence-based mostly guidelines for folks on remedies and behavioral therapies that might help their kids, and speaks out frequently on what it calls “non-proof primarily based remedies.” It’s previously stated of stem cell therapies, “There isn’t any proof that the remedy is protected or effective for autism, and no guarantee that the stem cells utilized in these countries are even human.”
“We are relieved to see that children are now not collaborating in the expanded access protocol which charged hundreds of dollars to mother and father of kids to obtain stem cell therapies which haven’t proven to be efficient,” Halladay told Motherboard. “The advertising of this product for the therapy of ASD was extremely premature and never in the best curiosity of the group. If it have been to restart, it might have to be justified by a randomized control trial to show the efficacy for these with ASD and make clear who on the spectrum would benefit from such therapy.”
She added, “Unfortunately, Cryo-Cell’s relationship to Duke University led households to imagine that it had been studied to the level of scientific rigor wanted to ensure it was each protected and efficient. We hope that households who have obtained this therapy proceed to be monitored for longer-term results, together with opposed events.”
XFacebookShareCopied to clipboardOne electronic mail. One story. Every week. Join the VICE newsletter.
By signing up, you comply with the Terms of Use and Privacy Policy & to obtain digital communications from VICE Media Group, which can embody advertising and marketing promotions, commercials and sponsored content material.
MoreLike This
‘Designer Cells’ May very well be the important thing to Autoimmune Disease Treatment
11.22.24 By Sammi Caramela
–
Man’s Brain Tumor Shrinks by Half Amid Clinical Trial
10.30.24 By Paige Gawley
-The FDA Just Approved a Schizophrenia Drug That Works Differently
09.27.24 By Sammi Caramela
-Alex Jones Owes Her Millions of Dollars. She Has to Crowdfund Her Cancer Treatment on GoFundMe.
05.10.23 By Anna Merlan
-A Plastic Surgeon Accused of Vaccine Fraud Has Become a Hero to Republicans and Anti-Vaxxers
03.13.23 By Anna Merlan and Tim Marchman
-Scientists Begin Trials for Transfusions of Lab-Grown Blood Cells
11.14.22 By Hannah Docter-Loeb
One e mail. One story. Every week. Sign up for the VICE e-newsletter.
If you loved this short article and you wish to receive more info concerning https://autismtreatmentcare.com/ i implore you to visit the web-site.
