Neither US Stem Cell Clinic nor California Stem Cell Treatment Centers responded to requests for comment.
The International Society for Stem Cell Research commended the FDA for its policy direction and enforcement efforts. President Hans Clevers said the society has been “very concerned” about reports of patients using unproven stem cell therapies.
“Many of these patients have suffered great harm, and even death as a result of using unproven stem cell therapies,” Clevers said in a statement. “We are hopeful that increased regulatory enforcement against clinics offering unproven treatments will deter this practice and help protect patients.”
“I’ve directed the FDA to launch a new working group to pursue unscrupulous clinics through whatever legally enforceable means are necessary to protect the public health,” Gottlieb wrote. “We have examples where some of these unproven treatments have clearly harmed patients.”
“I wish it hadn’t taken this long,” said Leigh Turner, associate professor at the Center for Bioethics at the University of Minnesota. “This is a space where the FDA could have taken action four or five years ago as far as making this a policy priority.”
Turner said he sees the steps announced Monday as both important and necessary, yet he remains skeptical.
“There are important distinctions to be made, and the FDA seems to be making these distinctions in terms of suggesting that they are putting together this working group, a task force, going after businesses marketing unproven interventions, going after businesses making illegitimate or unwarranted claims about stem cell treatment,” he said.
What will trigger regulation?
Gottlieb wrote in his statement “it’s incumbent upon the FDA” to make sure the existing legal and regulatory framework is properly defined, with “bright lines” separating individualized or tailored therapies surgeons are permitted to use from new treatments subject to regulation. Because the field of regenerative medicine is rapidly evolving, he said, close calls may be frequent between what constitutes an individualized treatment and what constitutes an unapproved, possibly harmful medical product.
Turner said Gottlieb’s statement allowed for “a bit of slippage as far as what exactly the FDA is going to do and which businesses they are going to target.”
Questions remain as to whether the warning letter is a sign of more letters to come and whether we will see “a dramatic increase” in such activities from the FDA, Turner said.
FDA spokeswoman Lyndsay Meyer wrote in an email that the agency “will seek to take additional actions in the coming months as we address this field, and target those who are clearly stepping over the line.”
Yet, Turner asked, what is enough to trigger FDA regulation? Are marketing campaigns and commercial activity enough?
“Or do we actually require people being blinded before the FDA does something?” he asked, noting that “there’s a considerable amount of uncertainty in terms of what we should expect in the months ahead. The statement itself doesn’t provide clear answers to all those questions.”
According to Susan L. Solomon, CEO of the New York Stem Cell Foundation, a nonprofit research organization, “the regulation of these clinics is very difficult, so the announcement today that the FDA will be stepping up their oversight should be welcomed and applauded.”
Overall, Turner said, the agency’s actions should not give all stem cell treatments or doctors performing these regenerative therapies a bad name. There are already effective treatments. “If we think about bone transplants as stem cell transplants, it’s standard of care for certain diseases,” he said.
Solomon agreed: “There are extremely promising studies and research using stem cells to treat macular degeneration, multiple sclerosis, diabetes and many other devastating diseases. I cannot emphasize enough how exciting and promising the research is.
“However, anyone advertising a ‘cure’ today is simply taking advantage of patients for their own financial gain,” she said.
What patients need to know
Turner acknowledges the difficulty for patients, who may not easily recognize which stem cell therapies are approved and beneficial and which are not.
“If you see a business that’s making all sorts of dramatic marketing claims across disease categories, claiming to use fat as a treatment for all sorts of indications, these are all signs to be wary of,” he said.
Turner acknowledged stem cell treatments as “a very promising area of research,” and over time, he expects to see more FDA-approved therapies in the marketplace. The problem, he said, is that many American businesses making claims about stem cell treatments lack proper scientific safety and efficacy data.
“Why, for example, didn’t California Stem Cell Treatment Centers get a warning letter for all the other treatments they are doing? It leaves me a bit perplexed,” he said. “Why is the FDA so focused on these vials and not on the broader array of marketing claims that California Stem Cells was making?”
Solomon said that “by providing unproven treatments to chronically ill or injured patients,” these clinics are not only taking advantage of patients, they are “muddying the scientific waters of clinical trials that are trying to show whether a treatment does or does not work.”
In its statements Monday, the FDA notes the “handful of bad actors” in the stem cell space, Turner observed. (Meyer repeated the FDA’s assertion that it’s only “a small number of unscrupulous actors who have seized on the clinical promise of regenerative medicine.”)
Ultimately, Turner is glad for the FDA’s actions.
“I hope this is a sign that the FDA is going to do a lot more and better regulate this market space so we’ll see whether or not that happens,” he said. “It’s easy to make these bold announcements. The question is going to be whether anything really comes of it.”
Correction: Previous versions of this story included quotes attributed to David McKeon, a spokesman for the New York Stem Cell Foundation; they should have been attributed to foundation CEO Susan L. Solomon.