Summary: umbilical cord blood treatments for autism that has not shown benefits will be marketed in clinics owned by a company with ties to the researchers.
https://www.vice.com/en/article/qj87xm/ ... g-business
Duke and Cryo-Cell’s rush to monetize a procedure before it’s shown to have solid benefits has created concern, though, across the community of scientists, clinicians, and medical ethicists who study autism treatments.
The hope is that these cord blood infusions can improve some autism symptoms, like socialization and language, or decrease the inflammation that some parents and clinicians think might exacerbate autism symptoms. Early study results, however, haven’t been very promising. A large randomized clinical trial, the results of which were released in May 2020, showed that a single infusion of cord blood was not, in the words of the researchers, “associated with improved socialization skills or reduced autism symptoms.”
One has to scroll far down the article for FDA information:
Knoepfler has questions, he said, about whether the FDA is “carefully monitoring” the Expanded Access Program at Duke, which it does have to approve, at least initially. “Are they still aware Duke and Cryo-Cell want to expand this greatly?” he asked. “Is the FDA aware of the fact that hundreds of kids have gotten infused and the plans could be for thousands more? That’s where I don't have a good sense. A lot of these things are hard to tease apart. There’s different confidentiality issues.”
One especially tricky thing, he added “is that Duke must have gotten what’s called an IND, or investigational new drug applications, for cord blood or stem cells related to cord blood. That’s the basis for running a clinical trial. And anything related to an IND, the FDA views as confidential.” Indeed, in investor documents, Cryo-Cell says plainly that it plans to administer stem cell infusions under the permissions granted by Duke and Kurtzberg’s IND approval. It has gotten, Cryo-Cell’s documents add, $5 million in startup funding from the Marcus Foundation.
The process from IND to full approval can take years. This is basically an end-run around the process, bringing a treatment to market without full FDA approval. Possibly the company will get a sternly-worded letter at first, and fines later, but they might consider that to be the cost of doing business.