In 2015, a 78-year-old Florida woman suddenly lost vision in both eyes after receiving a stem cell treatment for macular degeneration. The therapy she underwent at a clinic involved a unique type of eye injection that contained stem cells made from body fat. When her ophthalmologist later examined her, he saw bits of blood floating inside her eyes.
The case led the Food and Drug Administration (FDA) to file—and later win—a lawsuit against the company, which was subsequently prohibited from selling stem cell products without FDA approval. But the incident was not a one-off. Thousands of U.S. businesses sell unproven and often harmful stem cell products to consumers, claiming the products can treat a wide range of health problems—without having demonstrated to FDA that their concoctions are safe and effective. Furthermore, in a 2021 analysis, Pew identified 360 incidents of patients being harmed by stem cell therapies between 2004 and 2020; because clinics are not required to report adverse events, the numbers are likely much higher.
Products marketed as stem cell therapies may include cells extracted from a patient’s own body or from donated or purchased human biological material, such as amniotic fluid, placental tissue, or umbilical cord blood. Because they’re derived from such sources, they are often misperceived as medical practices, which states oversee, rather than medical products, which FDA regulates.
To clarify its authority, FDA updated its regulatory framework in 2017 and gave clinics three and a half years to comply with new medical product approval requirements. Unfortunately, rather than using the time to prepare for oversight, as a business columnist for the Los Angeles Times put it, “a torrent of shady operations poured into the field.” Indeed, between 2016 and 2021, the number of facilities offering unapproved stem cell treatments grew nearly fivefold from 570 to 2,754.
For more than five years, Pew’s health care products project has produced and supported research and analysis to help ensure the safety of stem cell interventions and other medical products. Its work has shed much-needed light on the stem cell market and helped regulators as they worked to protect patients and hold clinics accountable.
Pew research informs federal oversight
In 2019, Pew published research examining the potential impact of FDA’s regulatory framework. This work identified loopholes that could allow clinics to evade oversight, called on FDA to clearly communicate its decisions regarding the classification of human cell and tissue products, and encouraged the agency to balance innovation and patient safety in its review process. Since then, FDA has released multiple guidance documents for industry and health care professionals related to the proper administration, development, and evaluation of cell and gene therapies, as well as notices to clarify and reaffirm existing oversight policies.
In subsequent research published in 2020, Pew urged the agency to crack down on businesses selling illegal stem cell therapies. FDA now has sent more than 350 letters to manufacturers, clinics, and providers for offering unapproved regenerative medicine products, sought court injunctions against clinics that failed to comply with the law, and seized potentially dangerous products.
Pew also highlighted how the Federal Trade Commission could exercise its authority to regulate unfair, deceptive, or fraudulent business practices. This became especially important during the pandemic, as FTC warned businesses across the country that they were illegally marketing products to treat COVID-19 without “competent and reliable scientific evidence.”
Pew urges states to exercise their authority
Pew has also highlighted the role of states, some of which had already investigated and sued stem cell clinics. In 2018, for example, following an investigation by the North Dakota attorney general, a Bismarck stem cell clinic agreed to halt unapproved stem cell treatments and refund payments to patients. Similarly, after filing a lawsuit in 2019, New York secured a $5.1 million judgment against a clinic “for fraudulently and illegally advertising their stem cell procedures.” During the pandemic, Republican and Democratic attorneys general in several states, including Georgia, Arkansas, Iowa, and Nebraska, took similar legal actions.
Pew’s work also emphasized the need to empower patients with information. Legislators in Washington, California, Vermont, and Nebraska have all passed informed consent laws requiring clinics to tell patients when they are administering unapproved products.
Congress and FDA make structural changes to improve oversight
In its research, Pew stressed that FDA needs more resources to effectively oversee the growing stem cell market. Federal lawmakers addressed that issue in the Prescription Drug User Fee Act of 2022, with provisions that elevate and reorganize the FDA division in charge of reviewing cell and gene therapies. These structural changes—plus increased funding to hire more staff—will enable the agency to review more stem cell products, ensuring they are safe and effective before patients use them.
Despite the recent efforts of FDA, FTC, Congress, and various states, unapproved and illegal stem cell products continue to proliferate. This troubling boom—in addition to a recent legal setback in which a federal court ruled that the practices of a California stem cell firm are exempt from FDA regulations—highlights the need for broader, urgent enforcement.
FDA must move more quickly to fully implement its regulatory framework and significantly expand the scope of its enforcement activities against businesses offering unapproved and unproven stem cell treatments. It also should encourage patients and clinicians to report cases of harm resulting from these products. Finally, FDA does not have adequate resources to oversee the rapidly growing illegal stem cell industry on its own. For that reason, FTC and state policymakers also should crack down on clinics selling potentially harmful treatments. These steps would help protect patients’ health and foster the development of safe and effective regenerative products.
Kathy Talkington directs The Pew Charitable Trusts’ work on public health issues.