Unapproved stem cell treatments touted on federal database

Stem cell clinics offering unapproved treatments for ailments from hip pains to autism to erectile dysfunction increasingly are using a federal clinical-trials database as a marketing tool — a strategy that confuses patients and exposes them to “unjustifiable” safety risks and high costs, according to a new study.

At least 18 purported clinical trials — all of which involve unregulated therapies and require patients to pay to enroll — are listed on ClinicalTrials.gov, the comprehensive registry for public and private clinical trials that is run by the National Institutes of Health, according to an article published in the journal Regenerative Medicine.

Leigh Turner, who authored the study and is an associate professor at the University of Minnesota's Center for Bioethics and School of Public Health, said NIH should use much tougher screening tools to exclude from its database unapproved treatments, especially ones that require patients to pay to participate.

“Do we really want ClinicalTrials.gov to be ‘caveat emptor,' where no one is paying attention to the substance of studies being listed?” said Turner. “A lot of these studies are just marketing pitches designed to appeal to people with COPD, multiple sclerosis and Parkinson's disease.”

In recent years, hundreds of stem cell clinics have proliferated in the United States, but very few of their treatments have been cleared by the Food and Drug Administration.

The clinics insist that they do not need FDA approval because they are practicing medicine, which is outside the agency's purview. They also say they are exempted from agency rules because they don't alter the stem cells much, which is in dispute.

Experts say that FDA guidance indicates that the procedures should be regulated like a new drug — which means the clinics should be submitting their treatments to the agency for approval. But FDA's apparent reluctance to crack down on the clinics has created confusion — and opportunity.

The NIH clinical trials website operates largely on an honors system in which the government relies on the companies and researchers to answer accurately whether a product requires FDA review. Turner says the system isn't working.

“Absent evidence that the FDA has reviewed such studies, approved all charges to research subjects and permitted clinical research to proceed, such studies should not be accepted for inclusion and public listing,” he wrote in the article.

In a statement, the NIH said it “agrees that it is of paramount importance for potential research participants to know about the potential benefits and risks of participating in clinical research studies.”

The agency noted that it added a prominent disclaimer to the ClinicalTrials.gov homepage in March 2017 stating: “Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted health care professional before volunteering for a study.”

That disclaimer followed the publication of an article in the New England Journal of Medicine that reported three women were blinded after receiving stem cells from fat in a procedure touted as part of a clinical trial at a South Florida clinic. The women had paid $5,000 per person for the therapies that were supposed to treat macular degeneration. At least one of them heard about the procedure on ClinicalTrials.gov and thought she was taking part in a government-sanctioned study, the authors of the article said.

Most legitimate trials do not charge patients to participate, though patients might face incidental costs such as travel. By contrast, participants in the “pay to participate” studies for stem cell procedures are often charged thousands or tens of thousands of dollars, Turner said.

In many of the clinics, the procedures involve liposuction, in which fat cells are extracted, processed and then injected into other parts of the body.

“This is an important problem that needs to be addressed,” said Timothy Caulfield, a law professor at the University of Alberta who wasn't involved in the study and has called for tighter regulation of unproven stem cell treatments. “Many of us have noticed that Clinicaltrials.gov is being used as a marketing ploy to give a veneer of scientific legitimacy but this is the first paper to provide concrete evidence on how it plays out.”

As part of his research, Turner found seven studies in which the listings on ClinicalTrials.gov acknowledged that patients are charged to take part in the trials — five by the Ageless Regenerative Institute and two by the Lung Institute. He found another 11 studies in which the listings don't mention payment but other publicly available information makes it clear patients are charged.

Ageless is using stem cells in people with diabetes, erectile dysfunction and other problems while the Lung Institute is focusing on COPD. Neither company answered inquiries about their trials.

ClinicalTrials.gov, which was created by a 1997 federal law and made available to the public in February 2000, is a searchable database of clinical trials in the United States and elsewhere. It consists of trials that are funded by public and private organizations around the world.

Turner was the co-author of a study published last summer in the journal Cell Stem Cell that showed that unregulated stem cell clinics are proliferating across the United States. According to that study, at least 351 companies with 570 clinics are marketing unapproved treatments for conditions such as osteoarthritis, Alzheimer's, autism and injured spinal cords, as well as for cosmetic enhancements. And the number is probably higher now, he said.