The Food and Drug Administration just put dubious stem cell clinics on notice.

The agency announced plans on Monday for new policies and enforcement efforts to stamp out what it called “unscrupulous actors” peddling unproven, potentially dangerous, and often expensive stem cell therapies—including a bizarre and troubling instance involving smallpox vaccine.

As an initial demonstration of its harder stance, the agency today posted information on two enforcement efforts. One was a warning letter to a Florida stem cell clinic that had posed as legitimate clinical research and ended up blinding three patients after injecting stem cells directly into their eyeballs . The other was a concerning announcement that the agency had seized five vials of smallpox vaccine from stem cell clinics in California.

The clinics, run by StemImmune Inc., were said to be mixing the dangerous vaccine with stem cells for an unproven, unapproved, and potentially harmful cancer treatment that was injected intravenously into patients or directly into their tumors. Though the injection of stem cells alone lacks safety and efficacy data, the vaccine is known to be dangerous. The vaccine contains a live poxvirus, similar but less harmful than smallpox. When it’s injected into patients with weakened immune systems—such as many cancer patients—the vaccine can cause life-threatening side effects, such as swelling of the heart.

In today’s announcement, the agency noted that the vaccine, Vaccinia Virus Vaccine (Live), is not commercially available. It is reserved for people at high risk of being infected by the otherwise eradicated virus, such as researchers or military personnel. “The FDA has serious concerns about how StemImmune obtained the product for use as part of an unapproved and potentially dangerous treatment,” the agency said. The FDA added that it is actively investigating the vials’ origins.

Disquieting dose

At least some of the vials came from the Centers for Disease Control and Prevention, according to CDC spokesperson Thomas Skinner. In an interview with Ars, Skinner explained that private and public institutions around the country can request the vaccine and that the agency has rules and regulations governing how it’s dispensed. According to Skinner, StemImmune had told the CDC it needed the vaccine to protect some of its own researchers.

But in the FDA’s announcement, the agency reported that it had confirmed that StemImmune was actually using the vaccine in cancer patients. The FDA said that, of the five vials it seized, four were intact and one was partially used. The agency is investigating where the other vials came from and how StemImmune got them.

In an e-mail to Ars, StemImmune did not respond to questions about the FDA’s safety concerns or the origins of the vaccine vials. The company did offer this statement:

StemImmune, a biopharmaceutical company engaged in cutting-edge R&D of adult human stem cell-based therapies for the treatment of cancer, is fully cooperating with the FDA about the development of its stem cell-based investigational cancer therapy. We look forward to continuing our dialogue with the FDA as we seek to bring this important cancer therapy to cancer patients.

As for the Florida stem cell clinic, US Stem Cell, the FDA warned that it was marketing products without FDA approval. The clinic also had “significant deviations from current good manufacturing practice requirements, including some that could impact the sterility of their products, putting patients at risk,” according to the FDA.

In a statement e-mailed to Ars, US Stem Cell responded:

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 Food and Drug Administration. Since November 2014, we have offered the FDA unrestricted access to our facilities and our processes and will continue to work with them as it pertains to clinical and industry improvements. The FDA has stated that they will have specific stem cell guidelines by the 21st Century Cures Act deadline of December 13, 2017, and we intend to follow those standards as well.

Low-hanging fruit?

Questionable and harmful clinics like those run by StemImmune and US Stem Cell have proliferated in the US, according to researchers. A study last year found nearly 600 clinics had popped up across the country , claiming to treat everything from autism to Parkinson’s disease, mild joint pain, mental health conditions, and cancers.

In today’s announcement, FDA Commissioner Scott Gottlieb spoke firmly about cracking down on those clinics and their treatments:

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... 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.

But Leigh Turner, a bioethicist at the University of Minnesota who has extensively researched these stem cell clinics (and published last year’s study enumerating the clinics), is hesitant to say this is the beginning of the end for the harmful treatments. “In some respects, I feel encouraged by today’s developments,” he told Ars when reached by phone. The enforcement efforts and plans for new policies and working group are positive signs.

But, there have been “bold statements” before about crackdowns and nothing much happened afterward, he said. “And so, one question from me is—assuming [the FDA will] do something—are we going to see a more systemic, comprehensive, organized kind of approach to what looks like a serious problem in the market place? Or are we going to see a more ‘low-hanging fruit’ approach,” in which the FDA goes after egregious cases following lots of complaints, media investigations, reports of harms, or lawsuits, etc.

“It’s good to see the FDA do something,” Turner said, but not when it’s “on the backside of people being badly injured.”