A complaint filed in federal court indicates the vials were shipped from outside California but does not provide more detail on the origin. StemImmune also filed a patent application in February describing “the use of smallpox vaccine to induce an effective anti-tumor immune response.”
The type of vaccine that was seized is made from a virus similar to smallpox, but it does not contain smallpox virus and cannot give anyone smallpox, according to the Centers for Disease Control and Prevention.
Also Monday, the FDA announced it had sent a warning letter to a Florida stem cell clinic where a 2015 procedure led three women to go legally blind, according to a March study in the New England Journal of Medicine. The letter to U.S. Stem Cell Clinic said that the company was offering unapproved stem cell treatments and that an agency inspection uncovered violations of manufacturing standards, including potential sterility issues. The company also tried to stop FDA inspectors from talking with employees, the letter says.
“You should take prompt action to correct these deviations,” says the letter, which was sent Thursday. “Failure to promptly correct these deviations may result in regulatory action without further notice. Such actions include seizure and/or injunction.”
StemImmune did not respond immediately to a request for comment. In a statement, U.S. Stem Cell Clinic said it had given the FDA “unrestricted access” since 2014 and that it followed the agency’s rules. But it also suggested that regulations could prevent patients from getting treatments.
“The safety and health of our patients are our number one priority and the strict standards that we have in place follow the laws of the [FDA],” it said, adding: “We have helped thousands of patients harness their own healing potential. It would be a mistake to limit these therapies from patients who need them when we are adhering to top industry standards.”
The actions announced Monday are the latest salvo in a back-and-forth between regulators and the clinics that claim to be able to treat everything from paralysis to neurodegenerative diseases to erectile dysfunction. The clinics, which have proliferated around the country in recent years, have largely evaded regulation enforcement because they typically take a person’s own stem cells and inject them back into the same person, meaning the cells are considered “minimally manipulated.”
The FDA has tried at various times to regulate clinics offering unproven stem cell treatments more tightly and close down clinics that put patients at risk, but overall, their efforts have not amounted to much.
The FDA has held meetings about possibly regulating the therapies more like drugs, a move that has been backed by many academic stem cell experts who say that stem cell therapies, while holding great potential, are not ready to be used widely or outside regulated clinical trials. But clinic owners warn that the FDA could stamp out progress in the field and keep desperate patients from getting treatments.
“The government should not regulate our bodies. … I will always stand up for patient rights,” Kristin Comella, U.S. Stem Cell’s chief scientific officer, said at an FDA hearing months ago. (The FDA’s warning letter this month was addressed to Comella.)
Some experts have wondered whether the regulation-averse Trump administration would take further action against stem cell clinics. But in a statement Monday, Gottlieb said the FDA would be bolstering its enforcement as a way of “separating the promise from the unscrupulous hype” and that the agency would roll out a new “comprehensive policy framework” this fall to delineate the rules for stem cell treatments.
“There are a small number of unscrupulous actors who have seized on the clinical promise of regenerative medicine, while exploiting the uncertainty, in order to make deceptive, and sometimes corrupt, assurances to patients based on unproven and, in some cases, dangerously dubious products,” Gottlieb said. “These dishonest actors exploit the sincere reports of the significant clinical potential of properly developed products as a way of deceiving patients and preying on the optimism of patients facing bad illnesses. This put the entire field at risk.”
Experts who study stem cell clinics and the regulatory landscape generally praised the FDA’s moves, but said that it was unclear what the enforcement actions and Gottlieb’s statement meant for the field going forward.
“The most important thing is that the FDA is now publicly making a commitment to discipline what has become an out-of-control area of fraudulent medicine,” said R. Alta Charo, a bioethicist at the University of Wisconsin Law School.
One part of Gottlieb’s statement that stuck out to Charo, she said, was when he signaled some flexibility in terms of the areas that are subject to FDA regulation, with the commissioner writing that “the FDA must advance an efficient and least burdensome framework” and establish “a regulatory structure that does not become a barrier to beneficial new innovation.”
“It’s about a tone being set,” Charo said.
Leigh Turner, a University of Minnesota bioethicist, said that from one standpoint, the FDA’s enforcement actions targeted the low-hanging fruit of the bad actors in the industry. Presumably any administration would want to seize unauthorized smallpox vaccine supplies, and the Florida clinic was the subject of lawsuits and media coverage in addition to the high-profile academic paper.
“The larger question is going to be, what about the stem cell clinics … that are out there marketing therapies and charging people but don’t have a New England Journal of Medicine piece or lawsuits?” Turner asked. “Is it just going to be that after harm occurs that the FDA is going to do something? Or are we going to see a more systemic approach to this marketplace?”
The action against the California clinics was solely based on the vaccine, noted Turner, who has tracked the growth of clinics around the country. It did not mention all the other diseases and conditions that the clinics have been marketing their unapproved stem cell therapies for.