The curious case of ClinicalTrials.gov, where dubious stem cell therapies seem legit

Earlier this year, doctors reported the case of three women who went blind after having stem cells derived from their own fat injected directly into their eyeballs—a procedure for which they each paid $5,000. Piecing together how those women came to pay for such a treatment, the doctors noted that at least one of the patients was lured by a trial listing on ClinicalTrials.gov—a site run by the US National Institutes of Health to register clinical trials. Though none of the women was ever enrolled in the trial—which never took place and has since been withdrawn—it was enough to make the treatment seem like part of legitimate, regulated clinical research.

But it wasn’t. And, according to a new analysis in the journal Regenerative Medicine, it’s not the only case of dubious and potentially harmful stem cell therapies lurking on the respected NIH site.

At least 18 ostensible trials listed on the site offer similar stem cell treatments that participants must pay to receive—unlike most trials, which compensate rather than charge participants for experimental treatments. These trials, sponsored by seven companies total, claim to be developing therapies for a wide range of conditions, like erectile dysfunction, type II diabetes, vision problems, Parkinson’s disease, premature ovarian failure, and chronic obstructive pulmonary disease (COPD). However, these trials are largely not backed by preliminary research. None of them has Food and Drug Administration approval—even though the agency has published a draft guidance that suggests these treatments are subject to FDA regulation. And some of the studies are only granted ethical approval by review boards with apparent conflicts of interest and histories of reprimands from medical boards and the FDA.

“Some of these studies, I mean, they’re just so outrageous,” article author Leigh Turner, a bioethicist at the University of Minnesota, told Ars.  But, to hook patients, a listing on ClinicalTrails.gov is helpful to stem cell clinics, he explains. “A key thing that these businesses need to do is they need to look legitimate, they need to look credible,” he explains. “Listing studies on ClinicalTrials.gov is in some respects really clever from a marketing perspective—and really dangerous.”

They’re taking a federally funded website supporting clinical research, he said, and using it as a “marketing platform.”

Getting on the list

In his article Wednesday, Turner argued that the NIH should tighten restrictions on registering studies on the site. Right now, registration is largely based on the honor system. It’s up to study sponsors to accurately note whether their study is subject to FDA regulation.

“Some of these studies, I mean, they’re just so outrageous”

Many of the stem cell clinics argue that their work is not regulated by the FDA because they treat each patient with the patient's own stem cells, called autologous-derived stem cells. In many cases, tissue is harvested from a patient—often fat tissue collected using liposuction—then the tissue is processed in some way to obtain stem cells, which get injected back into the patient to treat any of several conditions. This was the case for the three blinded patients who received treatment at a clinic called US Stem Cell (previously known as Bioheart, Inc.).

Autologous treatments are exempt from FDA regulation if the cells are “no more than minimally manipulated” before they go back into the patient, the FDA noted to Ars. But a draft guidance that the agency has not yet enforced states that processing and isolating stem cells from tissue, specifically fat tissue, is not exempt. According to the draft guidance, this procedure involves cells that are “more than minimally manipulated,” because it alters the “original relevant characteristics” of the human tissue.

In a comment to Ars, the FDA said that, once the guidance is finalized, it will be able to apply the proper oversight to these stem cell treatments. In the meantime, it said:

“We recognize that there are a number of clinics operating, which do not register with FDA. Consumers are encouraged to contact FDA and the appropriate state authorities in their jurisdictions to report any potentially illegal or harmful activity related to stem cell based products. We also encourage patients and health care providers to report adverse events associated with cellular therapies to FDA.”

For now, stem cell clinics are skirting FDA oversight. Without enforcement, the only barrier to registering on ClinicalTrails.gov is to have an IRB—institutional review board—sign off. These boards provide ethical oversight of a clinical trial and must be registered with the FDA.