Unapproved Stem Cell Therapies Targeted in FDA Crackdown

FDA commissioner Scott Gottlieb, MD, announced the agency would take more forceful steps to prevent unregulated stem cell therapies from endangering patients. One of its actions included the seizure of smallpox vaccines obtained by a stem cell treatment company as part of an untested cancer therapy.

A statement from Gottlieb outlines a 2-pronged plan of intensified enforcement efforts and more efficient regulatory policies, meant to keep dangerous products out of patients’ hands while enabling innovation by stem cell researchers working within the law. The actions are necessary as the promising field of regenerative medicine has spawned “a small number of unscrupulous actors” hoping to take advantage of the uncharted clinical territory for profits, he explained.

“Products that are reliably and carefully developed will be harder to advance if bad actors are able to make hollow claims and market unsafe science,” the statement read. “In such an environment a select few, often motivated by greed without regard to responsible patient care, are able to promote unproven, clearly illegal, and often expensive treatments that offer little hope, and, even worse, may pose significant risks to the health and safety of vulnerable patients.”

It highlighted 2 enforcement activities undertaken in recent days to exemplify the kind of stepped-up oversight the agency intends to practice, but acknowledged that there are many more remaining sellers of unproven therapies to confront. Gottlieb ordered the creation of a new working group to pursue and prosecute in these instances.

One example of enforcement was a warning letter sent to US Stem Cell Clinic regarding its “significant deviations” from manufacturing practice requirements, which could contaminate its products. The letter also alleged that the company attempted to bar FDA inspectors from investigating the facility, which violates federal law. US Stem Cell Clinic now has 15 days to send a letter to the FDA outlining how it plans to correct the deviations.

The second enforcement activity involved the seizure of 5 vials of the vaccinia virus vaccine from StemImmune Inc after inspectors confirmed the vaccine was used in an unapproved stem cell product. Vaccinia virus vaccine is used to inoculate against smallpox, and while it does not contain any actual smallpox virus, it is highly regulated by authorities, so the FDA will investigate how StemImmune managed to obtain it. In a press release announcing the seizure, Gottlieb explained that the possibly dangerous stem cell “therapy” was being used in patients with cancer.

“Speaking as a cancer survivor, I know all too well the fear and anxiety the diagnosis of cancer can have on a patient and their loved ones and how tempting it can be to believe the audacious but ultimately hollow claims made by these kinds of unscrupulous clinics or others selling so-called cures,” said Gottlieb.

The FDA plans to create a new framework by this fall that will clarify its authority and the regulatory guidance surrounding stem cell products, with the aim of encouraging stem cell innovation by developers who follow the law and act transparently. Gottlieb’s announcement emphasizes that the agency does not want the disreputable stem cell peddlers to “poison the well” and ruin the public’s trust in regenerative medicine altogether.

This crackdown on unregulated stem cell therapies comes just months after a similar FDA initiative that warned consumers to avoid fraudulent cancer “treatments” and issued warning letters to 14 companies selling these unapproved products, which included pills, lotions, drops, and syrups.