Why did Duke autism team halt its troubling pay-for-play program?

Almost two years ago I publicly called on the FDA to freeze the unproven cord cell expanded access program (EAP) at the Duke Autism Center. Duke has been infusing kids with autism spectrum disorder (ASD) with unproven cord cells and requiring large payments for this. The cost was as much as $15,000.

A halt and a mystery

The Duke team led by Joanne Kurtzberg has a huge partnership deal with the cord banking firm, Cryo-Cell. The deal made me concerned that commercial interests might have been a driving factor to run the EAP.

The good news today is that Duke halted the autism EAP a few months ago.

What’s a mystery is why they stopped. Did the Duke cell therapy team itself decide to call a halt? Did someone higher up at Duke pull the plug? The FDA stepped in?

Let’s go through what we know including from a new piece written by Anna Merlan at Vice.

Duke Autism Clinic controversy

First, what was so problematic about the EAP that I asked the FDA to stop the program?

In brief, the Duke team knew the cells didn’t help but kept injecting kids with them anyway for years within the scope of the EAP and were charging for it.

Families were also given the impression that via the EAP their child might be helped despite the negative trial data. That’s false hope in my view.

The show The Doctors has run segments on the Duke program, including some seemingly more positive and others quite critical. In one of them included below, a family relates how they were given hope of major positive changes and not informed of risks, but then found that their son Ritchie had significant apparent side effects.

Just as a reminder of the reality here, already many hundreds of vulnerable kids have been through difficult experiences getting unproven cell infusions at Duke. Sometimes they were sedated to make it happen and some kids had side effects to that. We can’t be sure that cord cells are 100% safe in these kids in the long run. Families have been given false hope and lost their hard earned money.

Why did the Duke autism team halt the EAP?

Duke isn’t saying much about the ASD EAP shutdown. All I’ve heard as a possible reason is that their trial data were negative, but Duke has known that for years and kept the EAP going anyway. Did they get more negative data?

Some have asked me if the FDA might have frozen the program, but I haven’t seen any evidence of that.

Without more transparency here we may never know the reason for the halt.

This unexplained shutdown situation kind of reminds me of what happened with the Northwestern Autoimmune Disease HSCT program. After criticism for a variety of issues, eventually Northwestern shut it down. There was never any public explanation as to why. In that case, there’s good reason to think (still not in the public domain but I may one day post on it) that something unusual happened at that HSCT program to lead to the shutdown rather than it being due to criticism from some folks like me. However, no transparency means the public just doesn’t know the reason.

Note that unlike with Northwestern, which received an FDA warning letter, I don’t know of any oversight action by the FDA on this Duke ASD program. Northwestern also shut down its clinical trials program, while Duke is forging ahead still. Notably, unlike Duke, the Northwestern program actually had good data showing efficacy of what they were doing.

EAP for cerebral palsy continues?

Unfortunately, it appears the Duke pediatric cell therapy team is continuing their EAP for cerebral palsy (CP).

Duke’s own clinical trial data on cord cells for CP are generally discouraging. Maybe more clinical trials are justified with some changes in focus, but why continue an EAP?

Why might cord cells help CP? I can’t think of a convincing reason.

Duke and Cryo-Cell were unwilling to say much to Merlan.

What about Cryo-Cell?

As many of you know, I’ve been critical of Cryo-Cell.

I have mixed feelings about private cord cell banking in general (I do strongly support public cord cell banks), but what got me concerned specifically about Cryo-Cell is their plan to open infusion clinics. Their goal is to sell unproven cord cell offerings to children with various neurological conditions including CP and ASD as well as traumatic brain injury.

They would charge around $15,000 for this unproven medical treatment.

The fact that Duke has halted their EAP for autism could be a blow to Cryo-Cell’s plans or it could help the firm by creating more demand.

I hope they never open the planned cord cell clinics unless they have somehow gotten dramatically better data, which seems very unlikely.

Unproven clinics?

The halt by Duke could have another complication. It might drive demand to what I see as riskier offshore stem cell clinics that claim to treat autism.

For instance, I have big concerns about the Stem Cell Institute in Panama. They sell lab-grown cord cells as a supposed autism treatment but in my view have no good data to back that up.

There are still clinics in the U.S. selling supposed cell therapies for autism and CP too.

Looking ahead

Duke should do some soul-searching on this pediatric cell therapy program more generally and its history overall during the last 10-20 years.

It’s concerning that along the way the line between therapy and experiments seems to have been blurred more than once. Check out this The Plain Dealer investigative piece on Duke experimental pediatric cell therapy work. Reading it I feel some deja vu about where the Duke team is now in 2023 even if the cell types and conditions are different. They seem to have trouble keeping experiment and hopeful therapy separated.

Looking ahead, if we could jump in a time machine 15-20 years into the future, will the Duke team have produced a new FDA-approved cell therapy that is safe and effective for some kids for something? I hope so.

If not and the line between unproven experimental cell therapy offerings and actual helpful treatments for kids is still blurred at Duke even then, it’s going to be more painful deja vu.

The FDA has a role and responsibility here too. It’s not as though Duke and Cryo-Cell can just do whatever they want regardless of the data.  Can we expect more from the agency?