After standing on the sidelines while a major public health crisis developed, the Food and Drug Administration finally has launched what could be a major crackdown on clinics hawking questionable and potentially dangerous stem cell therapies.
The FDA announced Monday that it had seized nearly 500 doses of a smallpox vaccine that was to be combined with stem cells derived from body fat and administered to patients in clinics in Beverly Hills and Rancho Mirage. The agency also published a blistering warning letter delivered to U.S. Stem Cell Clinic of Sunrise, Fla., for "marketing stem cell products without FDA approval."
Perhaps most important, the agency combined those announcements with a hair-raisingly stern statement from its new commissioner, Scott Gottlieb. "Stem cell clinics that mislead vulnerable patients into believing they are being given safe, effective treatments that are in full compliance with the law are dangerously exploiting consumers and putting their health at risk," Gottlieb said, promising that the FDA "will be stepping up our enforcement actions against clinics that abuse the trust of patients and, more important, endanger their health." His goal, he said, is to "make sure the agency is separating the promise from the unscrupulous hype" surrounding stem cell science.
The FDA's action is long overdue. Clinics across the country now offer treatments using stem cells for an immense and growing range of diseases and conditions — stroke, multiple sclerosis, diabetes, Parkinson's, Alzheimer's and more. They charge desperate patients as much as $15,000, not including travel expenses, for treatments that are seldom covered by insurance. What's more, they're not approved by the FDA and are backed by virtually no scientific evidence to prove their efficacy.
Until now, the FDA's approach to this noisome corner of the healthcare landscape has been hands-off. Over the last five years, according to Leigh Turner, a bioethicist at the University of Minnesota, the number of these clinics has grown to more than 600 but the FDA has issued only five warning letters, only one of which resulted in the shutdown of a clinic network. (Turner and Paul Knoepfler of UC Davis collaborated on a 2016 paper that documented the expansion of the direct-to-consumer stem cell industry.)
"You want the FDA to provide oversight and enforcement," Turner told me. "If they stand on the sidelines and don't do anything meaningful, that becomes a kind of green light encouraging people to come pouring into the marketplace and make their money as long as they can."
If the FDA is serious about stepping up enforcement of laws and regulations governing direct-to-consumer marketing of questionable stem cell therapies, its path may be long and arduous, requiring every ounce of gumption Gottlieb can muster. That's because the industry maintains that its treatments aren't drugs subject to FDA oversight because they're derived from the patients' own tissues.
"The FDA has wrongly defined these in-clinic procedures as a drug," Kristin Comella, chief science officer of U.S. Stem Cell, fired back at the agency in a letter. "If the federal government were to interfere with a person's ability to obtain and utilize their own cells in their body to heal themselves, this could be a gross violation of the constitution."
One target of the FDA's California raid also responded angrily. "I think the FDA's got some political agenda, and they're taking it out on us," Mark Berman, co-founder of California Stem Cell Treatment Centers, which operates the Beverly Hills and Rancho Mirage clinics where the smallpox vaccine preparation was allegedly given to patients, told me. StemImmune, the San Diego biotech firm that allegedly developed the concoction, said in a statement that it's "fully cooperating with the FDA about the development and use of its stem cell-based investigational cancer therapy" and is "working to understand and address the questions raised by the FDA."
The FDA's sights are trained on a web of shadowy entities that mimic the look and feel of legitimate research bodies, including research firms, medical clinics and ostensibly nonprofit review boards. This industry has even turned a service of the U.S. government into an unwitting accomplice.
That service is ClinicalTrials.gov, a public database of clinical studies of drugs and medical treatments using human subjects. As Turner documented in a paper in July, the website, which is sponsored by the National Institutes of Health, effectively does nothing to screen out unscrupulous, unethical, or fake research projects. They're listed next to legitimate human studies of new drugs and devices vetted by the FDA, which requires evidence of successful laboratory and animal trials before it gives the go-ahead to human testing. But Turner argues that a trial's mere appearance on the website could mislead patients into thinking it's scientifically valid and approved by government bodies.
Among other trials, Turner examined five listed by StemGenex, a La Jolla clinic we've reported on previously. The clinic says its goal is to provide patients with "access to safe and effective stem cell therapies" for conditions including Parkinson's, multiple sclerosis, rheumatoid arthritis, osteoarthritis and chronic obstructive pulmonary disease. It does so by removing fat cells from a patient by liposuction, purportedly extracting stem cells from the fat, and reinjecting the cells into the patient. Experts say there's no scientific evidence that this procedure will treat the conditions.
"StemGenex uses the five studies it has registered with ClinicalTrials.gov as a marketing tool intended to confer legitimacy on its business practices," Turner wrote. StemGenex didn't reply to a request for comment on Turner's assertion. But the firm implies to patients that registration of its trials on ClinicalTrials.gov is a sign of StemGenex's credibility. "By providing access to registered clinical studies through The National Institute of Health," the firm's website says about a multiple sclerosis study listed on the government site, "we are providing patients with the ability to choose a stem cell treatment center with the highest standard of care."
The NIH says it has taken steps to warn the public against assuming that any trial listed on its website is legitimate. "We added a prominent disclaimer on the CT.gov homepage in March 2017," the agency told me by email, "stating that 'Listing of a study on this site does not reflect endorsement by the National Institutes of Health.'" But it was silent in response to a direct question about Turner's point that its failure to screen listings for even minimal legitimacy could easily allow unscrupulous registrants to mislead patients.
As the nation's sentinel of the safety and efficacy of drugs and medical devices, it was incumbent on the FDA to act long before now. The clinics marketing treatments directly to consumers have capitalized on the public's impression that stem cells are some sort of medical miracle. Dr. Mehmet Oz warned his vast television audience about this misconception in February, when he aired a lengthy undercover investigation of stem cell clinics and called for government regulation.
These treatments are not only a waste of money, but potentially dangerous. In March, the New England Journal of Medicine reported on the cases of three patients who suffered permanent partial or complete blindness after liposuction-derived stem cells were injected into their eyeballs, purportedly to treat macular degeneration. According to the medical journal, that treatment was performed at the Florida facility operated by U.S. Stem Cell, which received the FDA warning letter Monday, under its previous name Bioheart.
This industry's concept of medical treatment may pose a greater threat to public health because it strives to turn the scientific method upside down. StemGenex, in its response to a lawsuit over its marketing brought by several patients, asserts that the plaintiffs "cannot prove" that its "representations regarding the efficacy of its stem cell treatments are actually false." The former patients, it says, "do not cite to a single scientific study that disproves [StemGenex's] advertised claims."
The usual goal of medical research is to prove something works, not to profit from the absence of proof that it fails. The FDA may finally force purveyors of stem cell snake oil to do things right. If that means driving some of them out of business, they won't be missed.