Unlawful stem cell products continue to harm people as FDA deadline looms
More than two years after the Food and Drug Administration updated its regulatory framework for regenerative medicine, hundreds of businesses continue to sell unapproved stem cell products and other drugs derived from human blood and tissue.
The framework clarified how the agency would oversee the growing market for these products, and it encouraged the development of new therapies while also aiming to restrain the proliferation of unproven — and often dangerous — stem cell treatments.
Businesses were given three years to come into compliance, a period that ends this November. But the FDA has signaled that many clinics and providers have yet to heed approval requirements for their products.
As of 2017, there were more than 700 clinics advertising stem cell products for a range of conditions, yet the agency has not received the volume of approval applications is expected. It’s increasingly clear that the FDA is going to need help from Congress to make sure regenerative therapy developers comply with the law.
Republican and Democratic lawmakers in Washington are aware of the problem: they’ve been appropriately concerned for several years about a large number of companies that sell unapproved stem cell products. With little to no evidence, providers often claim that these products treat countless medical problems from arthritis to autism and multiple sclerosis; in fact, many have harmed patients’ health and offered no proven therapeutic benefits.
Most recently, several people in Nebraska developed serious infections and sepsis, a potentially life-threatening complication, after receiving unapproved stem cell products. In other cases, patients have been blinded, developed tumors at the site of drug injection, and even died.
The companies that sell these products trade on the legitimate potential that cell- and gene-based therapies could one day help treat, and even cure, serious illnesses, traumatic injuries, and debilitating chronic conditions. The promise of this emerging field is real. But it takes years of careful testing to make sure that drugs are safe and effective for humans — that they will heal and not harm. So far, only a handful of cell-based therapies have been approved by FDA.
Nevertheless, marketing of unapproved products has continued. In 2018, for example, contaminated stem cell injections marketed by a California-based company, Liveyon LLC, sickened 12 people in three states. The Centers for Disease Control and Prevention reported that affected patients had painful bacterial infections, some of which spread to bones, as well as abscesses at injection sites and swollen spinal discs. Three people were hospitalized for more than a month.
FDA’s initial enforcement actions targeted the supplier of these tainted drugs. Meanwhile, Liveyon executives claimed that the company “was always planning on becoming its own manufacturer,” and the company subsequently began making and selling its own stem cell-based products. Still, the business failed to follow the law, according to a December 2019 warning letter from FDA, in which the agency admonished the company for not following manufacturing safety measures for drug and tissue products and for using inadequate procedures to screen blood donors for communicable diseases.
A week after the letter was issued, Liveyon suspended sales of its unapproved products and announced its intent to meet with FDA about conducting clinical trials.
This was an important public health victory, but there’s much more to be done.
The same day that FDA announced its concerns with Liveyon, the agency released similar letters sent to 20 manufacturers and health care providers who were allegedly marketing unapproved stem cell products and therapies. Among them were businesses claiming that their products could treat cancer, diabetes, asthma, and a host of other diseases.
The potential harm of unproven therapies to patients and their families goes beyond health. Backed by aggressive marketing tactics, clinics offering unproven treatments charge patients thousands of dollars — costs typically not covered by health insurance.
Lawmakers from both parties have encouraged FDA’s work. The House Appropriations Committee signaled its support last May in bipartisan report language accompanying an FDA funding bill, urging the agency to coordinate with the Federal Trade Commission to optimize enforcement and consumer education activities.
In June, after a federal court granted a permanent injunction against a Florida clinic offering unapproved stem cell products, Sen. Chuck Grassley (R-Iowa) praised the FDA and the Department of Justice for their efforts to “aggressively enforce the law.” And in July, the House Energy and Commerce Committee sent a letter to FDA expressing concern about growing number of stem cell clinics and the slow progress bringing them into compliance.
Members of Congress can provide still greater public support for FDA’s efforts as November’s critical enforcement deadline approaches. Timely congressional hearings can shine a spotlight on the promise of regenerative medicine, the pitfalls of rushing treatments straight to consumers without rigorous testing, and the dangers posed by treatments that are not yet proved to be safe, effective, or beneficial. These oversight activities, combined with adequate agency funding to enforce its regulations, can help save patients and families lured by the potential of unproven products, and can hold those who sell them to account in the public eye.
Allan Coukell directs The Pew Charitable Trusts’ health care programs.