December 8, 2021

Therapeutic misconception & the planned Duke Cryo-Cell cord clinic

Therapeutic misconception & the planned Duke Cryo-Cell cord clinic


One of the core problems in the world of unproven stem cell clinics is something called therapeutic misconception. I’ll talk more about that in a minute, but first, let me tell you what brought this to mind now.

Surprisingly, the story starts at Duke University.

A Duke cell therapy team and the biotech Cryo-Cell have big plans for an unproven, for-profit cell therapy clinic for kids to open in just a few months. One of my main concerns with the Duke Cryo-Cell plan is that their efforts are based on minimal, often conflicting data, and corporate profit seems to be a central goal (e.g., see image below from a company presentation).

Here on The Niche, I’ve been critical of their efforts. I knew I would get some pushback on my criticisms of what they are planning and I got some intense reactions. These responses have included hate mail and personal attacks.

Therapeutic misconception & the planned Duke Cryo-Cell cord clinic
Cryo-Cell presentation slide focused on revenue. The talk didn’t mention a central goal of helping children, at least not that I heard. Screenshot. Note that an FDA-cleared IND or compassionate use program is not equivalent to a final drug approval. The cord cell drug in this case has little concrete data behind it.

Parent’s Guide to Cord Blood Foundation: Duke Cryo-Cell defender

Now Frances Verter of Parent’s Guide to Cord Blood Foundation wrote a long piece in part to try to rebut my concerns and criticisms.  I disagree with much of what she wrote, but at least she was civil and carefully explained her thinking.

In the article, Verter lays out her arguments both for why she thinks I’m wrong and the reasons why the Duke Cryo-Cell effort is supposedly worthwhile.

The cord blood product in question is not approved as a drug by the FDA and is not clearly scientifically proven. It is just part of a compassionate use program.

Verter’s piece lists many clinical studies and some results in this general area of clinical research, but I’d say there’s no clearly convincing data. Overall, there’s really “no there there” to support a big new clinic.

Stem cell clinic parallels

It’s interesting that to make some points she in part quotes stem cell clinic doc Chris Centeno.

Centeno has been critical of me for using the term “unproven” about stem cell clinics like his cell therapy clinic chain Regenexx. His argument seems to be that much of accepted modern medicine is still unproven by data. I disagree.

By bringing Centeno into this Verter just adds another parallel between the Duke Cryo-Cell effort and the existing stem cell clinics. If the FDA allows the Cryo-Cell clinic to open I’d imagine that hundreds of stem cell clinics across the U.S. will applaud.

It all goes toward the risky idea that solid clinical trials and clearly encouraging data just aren’t necessary for cell therapies prior to selling them.

In another parallel, Verter emphasizes parent testimonials from families who already got off-study infusions from Duke and unproven stem cell clinics often rely on such testimonials for marketing.

Duke, Cryo-Cell, & therapeutic misconception

Now let’s bring therapeutic misconception back into the discussion.

A friend mentioned to me that Verter’s new piece is an example of therapeutic misconception. What is that?

The most basic definition seems to be when patients misunderstand clinical research as though it was a beneficial treatment.

How does it apply here?

Research by Duke/Cryo-Cell is being falsely equated with a promising treatment for kids. In reality, it’s just research. In this case, in my view, the misconception starts with the groups marketing the research as though it was a treatment. Then this confuses the patients (or parents) leading to false hope.

Unfortunately, this kind of conflation of cell research with cell treatments is pervasive.

It is particularly common in the unproven stem cell clinic world. Many clinics wrongly market experimental stem cells as a beneficial treatment but somehow also research. The clinics’ intent seems to be to encourage therapeutic misconception among potential customers. Maybe we need a new term for when a sponsor is encouraging therapeutic misconception?

As to Verter’s piece, I’m also concerned by her inclusion of pictures of happy children and parents both in this piece and others on Duke/Cryo-Cell. Is that to send the message that the experimental cord infusions are treatments that will bring family happiness?

Demand doesn’t equal justification for unproven clinics

Another issue with Verter’s article is that it wrongly seems to argue that parental demand justifies the planned unproven clinic.

Just here in the U.S. there is a vast number of Americans desperately seeking treatments for other conditions, but do we open for-profit clinics with unproven cell therapies for heart disease, paralysis, and many other conditions? Wait, with this idea we’re back to the huge American unproven stem cell clinic industry again, right?

Would the FDA give an explicit OK for unproven cell therapy clinics for millions of customers around the U.S. for diverse conditions? No way.

So why should Duke/Cryo-Cell be a special case where the agency lowers the bar for a clinic? It shouldn’t.

Money issues: Cryo-Cell funding for Parent’s Guide & more

If this was all about just doing clinical trials it would be one thing, but again we’re talking about for-profit clinics.

It struck me that Verter’s article acknowledged that her organization gets substantial annual payments from Cryo-Cell.

Since the Duke team is also financially tied with Cryo-Cell and has also been getting millions of dollars from a philanthropic organization for their cord blood research on kids, I believe money is a big motivating factor driving the rush to open a new clinic.

I’m not clear why, but Verter argues the financial aspects of the deal should be considered separately from the ethical issues:

At the end of the day, the ethical aspects of the Duke EAP need to be separated from the financial aspects. An ethicist can make a strong case that it is unethical to charge families for compassionate treatment of a child that has disabilities which impact daily life. But the financial reality is that Duke does not have the resources to scale up their EAP. This means that the Duke EAP will never get bigger unless another entity, such as Cryo-Cell, licenses the technology and offers it commercially.

I appreciate that she acknowledged the ethically thorny side of charging for compassionate use.

However, it’s puzzling to me that she thinks that the Duke EAP needs to get much bigger, but it may go back to her belief that the Duke offering is promising or that parents need something even if it is a shot in the dark.

I also noted that Verter claims that the FDA allows firms running compassionate use programs to profit from the efforts. The regulatory language she linked to is a little vague even if it does mention profit in passing. She might be technically right.

Still, historically, has any compassionate use program before ever splashed around huge financial hopes in the tens of millions of dollars in income all from their “compassionate” clinic based on so little data? Has any compassionate use program opened a clinic hoping for thousands of paying customers and the customers are kids? If so, I’ve never seen anything like that.

It seems radical to me. Have there been other such cases?

A place for compassionate use more generally

To be clear, I believe there can be a place for compassionate use (more technically called “expanded access”) for certain seriously ill patients without other options.

However, caution is needed in evaluating such programs and monitoring them as they may evolve over time.

When a sponsor is planning to enter into the squarely for-profit realm with such programs they should be subject to even more oversight and scrutiny first.

Otherwise, participants getting the experimental offering risk become more like customers.

Will the unproven clinic open in early 2022?

I have called for the FDA to freeze the Duke compassionate use program to give time for the agency to investigate before the dubious Cryo-Cell clinic opens.

Verter argues it isn’t likely the FDA will do that.

If she’s right about that, then the agency will have undermined their own stance on cell clinics overall.

Also, I believe the FDA can then expect a wave of new requests from other biotechs or universities (or stem cell clinics) for more for-profit but unproven cell therapy clinics for heart disease or diabetes or dozens of other conditions based on minimal or conflicting data under the umbrella of compassionate use. Is it going to okay those too?

Such a trend could weaken the FDA approval system overall.

As I’ve said before, this is about real people in the form of potentially thousands of vulnerable children who might get experimental injections portrayed as treatments.

Verter’s piece doesn’t make it clear to me how that can be a good thing.

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