Hundreds of U.S. stem cell clinics selling experimental therapies without an FDA permit can expect to face the agency’s enforcement arm and potential action from the federal government.
The Food and Drug Administration ended a three-and-a-half year grace period Monday designed to allow these clinics to come into compliance with its 2017 regenerative medicine policy. The agency has worked with federal law enforcement agencies to shut down the most egregious cases, including one that made several women blind.
Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, has made clear for years that the agency will be stepping up its enforcement over these unproven treatments once the discretion period ends. It was supposed to end last November, but the FDA provided a six-month extension due to the pandemic.
“It’s a high priority that we get the message out there,” that these products need “investigational new drug applications, or should have a biologic license application submitted, or they should not be marketed,” Marks said at the Food and Drug Law Institute’s annual meeting May 20.
Clinics that sell these treatments say they are surgical procedures that don’t require FDA regulation. The agency counters that these companies are making unproven and potentially harmful claims about products that fall under its regulations for human cells, tissues, and cellular and tissue-based products, or HCT/Ps.
The agency doesn’t oversee what it defines as minimal manipulation of HCT/Ps, but that’s a designation for procedures such as bone marrow transplants, which have been used in medical care for decades, to proceed without FDA oversight. The clinics offering the new and unproven treatments typically take someone’s fat cells and reprocess them into stem cells, with claims that these cells can treat a range of diseases from Parkinson’s to heart disease and even Covid-19.
“All cell therapies should be proven safe and effective in well-regulated clinical trials before being marketed to patients. In recent years, increasing numbers of patients have been harmed by such therapies,” Sean Morrison ISSCR public policy committee chair and director of the Children’s Medical Center Research Institute at UT Southwestern, said in a statement to Bloomberg Law. “We hope increased FDA enforcement will reduce the number unscrupulous clinics that take advantage of desperate patients.”
The International Society for Stem Cell Research said it welcomed increased FDA enforcement of its regulations against the marketing of unproven cell therapies.
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“It’s really time for FDA to start aggressively enforcing the law to protect patients from these products, which are risky,” Liz Richardson, project director of health-care products for The Pew Charitable Trusts, said in an interview. “They often cost people, not just in terms of their health, but in terms of their money, because a lot of these treatments are paid for out of pocket.”
These clinics have harmed hundreds of patients since they’ve proliferated, Pew found in a report released Tuesday on its health projects website. Pew identified 360 people harmed between 2004 and September 2020 by stem cell injections and other regenerative therapies. The data came from adverse events reports from medical literature, submissions to the FDA’s adverse event reporting system, government publications, news articles, and online consumer reviews of stem cell businesses such as Google and Yelp.
Pew identified at least 21 patient deaths, as well as injuries that included life-threatening infections, blindness, cardiac arrest, and the growth of tumors and lesions.
Those incidences are probably much lower than the level of harm that’s actually happening, Richardson said. “We know that adverse events are underreported for any medical products, including those that are FDA regulated, and there’s mandatory reporting involved. But for this, these products that are unapproved and unproven, the rate of underreporting is probably much higher.”
While Richardson said she wants the FDA to be more aggressive in going after these clinics, she acknowledged there are limits on the agency’s ability to pursue hundreds of clinics at once. Marks’s center is small and also regulates vaccines, monoclonal antibodies, convalescent plasma, and blood, and there are about 900 active investigational new drug applications for cell and gene therapies.
Pew will be tracking the agency’s enforcement effort over the next several months.
“It’s important to acknowledge that there are limitations in what the agency can do, certainly, given their chronic underfunding. That’s a reality,” Richardson said. “It probably will be a slow ramp-up, given just the fact that we are still in the middle of a pandemic.”
The Federal Trade Commission also has authority over businesses making illegal claims, and state medical boards and attorneys general also can make strides in protecting patients from potentially dangerous products, Richardson said. “It’s not just the FDA’s job. There’s a role for other regulators in this space.”