On Monday, the U.S. Food and Drug Administration (FDA) announced that the agency is targeting clinics that offer unproven stem cell therapies, calling such offices “unscrupulous clinics” selling “so-called cures.” The FDA seized materials from one clinic in California, and sent a warning letter to another in Florida.
“The FDA will not allow deceitful actors to take advantage of vulnerable patients by purporting to have treatments or cures for serious diseases without any proof that they actually work,” said FDA Commissioner Dr. Scott Gottlieb in a statement.
The agency announced that on Friday, Aug. 25th, U.S. Marshals seized five vials of a vaccine that is intended for people at a high risk for smallpox (for example, people in the military) from StemImmune Inc. in San Diego, California. The FDA says it learned that StemImmune was using the vaccines as well as stem cells from body fat to create an unapproved stem cell therapy. On its website, StemImmune says “The patient’s own (autologous, adult) stem cells, armed with potent anti-cancer payloads, function like a “Trojan Horse,” homing to tumors and cancer cells, undetected by the immune system.” The stem cell treatment was injected into the tumors of cancer patients at the California Stem Cell Treatment Centers in Rancho Mirage and Beverly Hills, California.
The FDA also sent a warning later to U.S. Stem Cell Clinic in Sunrise, Florida. The company recently came under public scrutiny when a March report revealed that three people had severe damage to their vision — one woman went blind—after they were given shots of what the company said were stem cells into their eyes during a study sponsored by the clinic. The FDA says that an inspection of U.S. Stem Cell Clinic revealed that the clinic was using stem cells to treat diseases like Parkinson’s, amyotrophic lateral sclerosis (ALS), chronic obstructive pulmonary disease (COPD), heart disease and pulmonary fibrosis. According to the FDA, there are currently only a limited number of stem cell therapies approved by the agency—including ones involving bone marrow, for bone marrow transplants in cancer care, and cord blood for specific blood-related disorders. There are no approved stem cell treatments for other diseases.
The FDA says U.S. Stem Cell Clinic also attempted to interfere with the FDA’s most recent inspection by refusing to allow FDA investigators to enter without an appointment, and denied the agency access to its employees. “Refusing to permit entry or FDA inspection is a violation of federal law,” the FDA says.
Action by the FDA on clinics promoting unproven stem cell therapies is “a long time coming,” says Sean Morrison, former president of the International Society for Stem Cell Research (ISSCR) and director of the Children’s Research Institute at UT Southwestern. “Clinics are preying on the hopes of desperate patients claiming they can cure all manner of diseases with stem cells that have not been tested in clinical trials, and in some cases, are flat out impossible.”
In the past, medical experts were concerned over Americans traveling to countries with less medical regulation for stem cell therapies, but Morrison says such clinics have been popping up stateside over the last five years. “It’s not a few companies in the U.S. making claims about therapies with stem cells,” says Morrison. “It’s scores of companies. The problem has exploded in the U.S.”
Morrison blames the lack of FDA crackdown in the past for the growing problem. “At some point people made the calculation that the FDA didn’t seem to be enforcing these laws,” he says. “The margins are huge. They charge people tens of thousands of dollars.”
Since stem cell therapy is still an active and legitimate area of scientific research, it can be hard for Americans to figure out what is safe and effective and what is not. Even when it comes to clinical trials, the scientific soundness is murky. A July 2017 paper reported that 18 U.S. companies have registered “patient-sponsored” stem cell studies on ClinicalTrials.gov. That means that the patients receiving the treatment paid for them, which isn’t the case in more legitimate studies. None of these were gold standard studies: meaning the people were not randomly assigned to receive the treatment or not, so the participants knew they were receiving the therapy — that could bias the results. Only seven of the studies disclosed upfront that patients had to pay to join the study, and none revealed that the costs ranged from $5,000 to $15,000 a treatment, Wired reports.
While Morrison says he’s glad the FDA has taken action, he says it’s not enough—at least not yet. “The FDA has to show that there is really a sustained commitment to enforcement,” he says. “When the FDA wasn’t bringing actions against these companies, I think people thought this meant that it was a gray area and that they could get away with it.”
Undoing that damage could be a long process, and one that Morrison says needs consistent attention by the agency. In a letter released on Monday, FDA commissioner Gottlieb said the agency is stepping up enforcement of stem cell therapies and regenerative medicine. “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,” said Gottlieb. Whether those efforts have an impact remains to be seen.