FDA Acts to Stop Use of Unapproved Regenerative Medicine Products

The Food and Drug Administration has taken action in recent months in two instances to protect consumers from the potential dangers of unproven stem cell and blood plasma products marketed to treat a host of conditions.

In November, the agency sent a warning letter to a California business that was selling stem cell products derived from umbilical cord blood for administration to people with arthritis and other conditions, although the treatments had not been approved by FDA for this use. The letter—an important step that could lead to enforcement actions such as product seizures and injunctions—came after at least 12 people in Florida, Texas, and Arizona were hospitalized following infusions or injections of cord blood cells that caused bloodstream and joint infections as well as abscesses along the spine and skull.

When FDA inspectors visited the facility that processed the cord blood products, they cited at least 19 concerns with its manufacturing process. They included failures to appropriately screen blood donors for diseases such as HIV, hepatitis B, or hepatitis C, and a failure to adequately clean and disinfect rooms and equipment.

Then in February, the agency issued a statement cautioning consumers and providers about businesses transfusing blood plasma from young donors into older patients, based on the unproven theory that the transfusions could treat or prevent conditions ranging from normal aging to serious illnesses such as Parkinson’s, multiple sclerosis, Alzheimer’s, heart disease, and post-traumatic stress disorder. In a clinical trial conducted by one such business, people ages 35-92 paid $8,000 to receive 1.5 liters (about 6 cups) of plasma from donors ages 16-25, but results from the trial were not published.

The agency’s warning noted that plasma transfusions confer no known benefits for the conditions they were being marketed to treat or prevent, but do pose well-established risks, including allergic reactions, acute lung injury, or transmission of infectious diseases.

These are among the examples of businesses taking advantage of public excitement over the promise of regenerative medicine—a broad field that includes stem cells, bioengineered tissues, and gene therapies—to peddle risky and often expensive interventions, typically not covered by insurance and with no proven benefits.

Some regenerative therapies have substantial clinical potential, and many trials are underway to translate that into real treatments. In recognition of this, FDA has sought to balance the need for ensuring product safety and effectiveness with the desire to encourage innovation in this nascent field.

In November 2017, the agency released a regulatory framework for regenerative medicine that sought to clarify the distinctions between products that are subject to FDA’s full drug approval requirements and those that are not. The agency also streamlined the review process for new therapies and reduced some of the regulatory requirements on product developers.

FDA has also pledged to step up enforcement activities against developers marketing unapproved products. Its recent actions indicate that the agency is, appropriately, prioritizing situations where threats to patient health and safety are grave and where businesses are operating at such a large scale that they potentially expose many patients to risk. For example, because these plasma and cord blood interventions required blood from donors, they posed a higher risk of infection or allergic reaction than other interventions marketed as stem cell treatments, which are often sourced from a patient’s own bone marrow or fat.

An estimated 716 clinics nationwide offer unapproved stem cell interventions, a reality that reinforces the need for ongoing regulatory actions by FDA and other oversight bodies charged with protecting public health or protecting consumers from false and misleading advertisements. FDA officials have made clear that they expect more such actions this year and beyond.

The day the agency issued its warning to the California company, officials said they had sent letters to 20 other clinics “to reiterate the FDA’s compliance and enforcement policy to other manufacturers and health care providers who may be offering stem cell treatments.” These actions send a message that regulators will not ignore those offering unproven and risky regenerative interventions to patients.

Liz Richardson directs The Pew Charitable Trusts’ health care products project.