UC Davis Health System

10/12/2021 | News release | Archived content

Vice tackles dicey Cryo-Cell Duke mega-million peds clinic plan

When I first learned about the multi-$10 million cord cell clinic deal between Duke and Cryo-Cell I couldn't believe what I was reading.

Duke's involved in this?

The problem: marketing unproven cells for kids

Yes, this wasn't some stem cell clinic operating out of a strip mall seeking to make big money off of injecting kids with unproven stuff. Duke and the well-known cord blood biotech plan to try to make huge revenue by infusing perhaps thousands of vulnerable children with unproven cord blood cells.

That doesn't seem right to me. It also doesn't seem so different than some of the seriously problematic stem cell clinics.

Duke & Cryo-Cell using vulnerable kids

My first reaction back then when I didn't even know about the $10s of millions involved in the deal was that this was likely unethical. When I read the SEC records of the company and saw the big money that was central to the deal I was even more concerned.

I've written extensively about this specific dicey deal to infuse autistic kids and other children with unproven cord cells. In my most recent piece I called on the FDA to freeze the Duke expanded access program or EAP that makes this controversial commercial deal possible. EAPs are supposed to be about helping terminally or otherwise seriously ill people get access to a chance for hope via a promising but not yet proven therapy. Also, EAPs are not supposed to be commercial in nature. Duke's deal doesn't seem to fit with an EAP on both of these levels.

The FDA approves and then oversees EAPs. The more I learned about this largely EAP-based deal with Cryo-Cell the more I thought that this couldn't have made it past the FDA, but at some point some version of at least the EAP did.

How is that possible? Was politics involved? We need to learn much more.

Vice digs into the big money deal

There's a new piece out in Vice by journalist Anna Merlan that helps provide more info and perspectives.

It sheds fresh light on this troubling Duke cord blood for autism program that has teamed up with Cryo-Cell. It is run by Professor Joanne Kurtzberg at Duke. I'm very glad to see more attention brought to this situation and highly recommend the piece.

I especially like the many voices that Anna brought into her story.

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."

Anne Border King, who is Chair of the Campaign Against Phony Autism Cureshad this to say:

"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."

The Vice story also quotes professors Leigh Turner and Jeremy Snyder, who have published important work in this area. They cited numerous issues with the Duke and Cryo-Cell effort.

Kurtzberg quotes

Anna's story in Vice also has several quotes from Dr. Kurtzberg that are notable. In the team's newest approach they are using cord blood monocytes to try to treat cerebral palsy and autism. As to the former, Kurtzberg invokes an unproven monocyte inflammation hypothesis:

"In email responses provided by Duke, Kurtzberg told Motherboard that monocytes "can modulate inflammation, especially in the brain. In cerebral palsy, these cells promote repair of myelin-particularly in the motor tracks, which is often damaged during injuries. We have published several papers regarding this, noting the unique properties of monocytes in cord blood."

That seems very speculative to me. I believe all their data on monocytes is only in rodents or cell culture too. Not humans.

And she then talks about autism:

"Autism, Kurtzberg added, "is more complicated because there are multiple etiologies causing this condition. In some instances, there is inflammation of the brain, and cord blood tissue appears to calm down that inflammation in animals and lab models. We are exploring whether cells in cord blood or in cord tissue, we're not sure which, has the ability in certain patients to calm down that inflammation and improve symptoms."

This second quote might seem reasonable at first on its own because it suggests a real clinical trial to test whether cord cells help some autistic kids and appropriately mentions the uncertainties and complexities here, but again Duke and Cryo-Cell are essentially selling this totally unproven offering for $15,000 in parallel to doing trials. Such trials may ultimately show this approach is ineffective or even unsafe in some ways. Cryo-Cell's SEC filings describe the anticipated income from charging families as projected revenue so I don't really see this as patient-funded research.

Weak and negative data

What do the existing data say?

As Anna points out in her Vice story and as I have written several times, one of the biggest problems for Duke and Cryo-Cell is the weak or even discouraging clinical trial data they are standing on with this effort. Even their own trial data is largely disappointing about the utility of using cord blood for autism and other conditions.

Unfortunately for the kids and families involved, the trials don't even ever have to be effective for the kids or even a success at ever getting an FDA approved product for Duke and Cryo-Cell to be raking in big bucks. Even if the trials flounder and keep finding largely negative or inconclusive results, which is not that unlikely, the university and biotech will still have generated possibly $10s of millions in revenue for themselves from the families via the infusion clinics. At least that's my reading of their deal in the SEC document and other information I've seen.

Duke, Cryo-Cell and the FDA

Looking to the future, for Duke officials it seems the question is going to be whether the prospect of possibly tens of millions of dollars will make it worth continuing what seems to me to be a potentially unethical endeavor with Cryo-Cell to open infusion clinics based on weak or even negative data. Duke has already been benefiting from the millions from the Marcus Foundation as well as the biotech deal. It may be hard for an institution to turn its back on such huge money. I'd say sometimes you've got to take the difficult but upstanding path.

However, in a sense it's not really up to Duke.

As I've written before, the FDA can and should stop this now by putting a hold on the program. Then the agency should re-evaluate the EAP as well as the direction it's headed commercially. I hope the agency will err on the side of caution, particularly because we are talking about possibly thousands of children here and their families.

I'll also say this again - the infusion clinic plan doesn't seem so different than what some of the unproven stem cell clinics are doing. The FDA is very vocal about those unproven stem cell clinics and has taken major action recently in that space.

The unproven Cryo-Cell cell infusion clinic is set to open in just months so the agency needs to be quick about this.

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