Unproven stem cell treatments: Is the US Supreme Court sending a message?

Recently, the US Supreme Court declined to hear an appeal from stem cell clinics, which challenged the Food and Drug Administration (FDA)'s authority to regulate unproven stem cell treatments.

In California Stem Cell Treatment v United States, the Supreme Court refused to review the decision of the Court of Appeals for the Ninth Circuit, and denied the Goldwater Institute's motion to file a related brief for consideration. This decision means that the previous ruling will stand, affirming the FDA's regulatory oversight of stem-cell-based treatments (see BioNews 1260). This has important implications for stem cell clinics and their patients.

The FDA has become concerned about for-profit entities that advertise stem cell therapies as treatments for various diseases and medical conditions, without robust scientific evidence supporting the safety and efficacy of these medical procedures. In 2017, the agency developed governance for regulating regenerative medicine products and cellular therapies. Moreover, it expressed the intention to expand enforcement of rules relating to cellular therapies. The FDA has brought lawsuits to prevent unscrupulous stem cell businesses from administering stem cell products derived from adipose tissue to patients.

In this case brought by the FDA in 2022, the defendants were two California-licensed physicians. At two of its clinics in Beverly Hills and Rancho Mirage, the company offered stem cell treatments to patients seeking non-surgical alternatives for their degenerative disorders. The defendants had treated thousands of patients. The advertisement claimed that they possessed the technology to create a solution with the patients' stem cells, which could cure medical conditions ranging from Alzheimer's disease to erectile dysfunction.

As these procedures were not covered by medical insurance, the patients had to pay out of their own pocket. A single procedure costs US$8900, and a 12-treatment course costs US$41,500.

The defendants also operated a consortium of doctors intending to introduce regenerative medicine into their practices. The affiliates agreed to adopt the defendants' treatment protocol and pricing guidelines, share data, and purchase the defendant's tool ('Time Machine') for cell isolation.

In the California district court, Judge Jesus Bernal ruled that the defendant's stromal vascular fraction (SVF) surgical procedure and the expanded mesenchymal stem cells procedure were not drugs under the definition of the Federal Food, Drug, and Cosmetic Act (FDCA). These procedures were not, therefore, subject to the Act's sections on adulteration and misbranding. Additionally, the defendants' SVF procedure qualified for the same surgical procedure (SSP) exception.

Judge Bernal's decision caused a shock to the cell and gene therapy scene. The serious concern was that this ruling was inconsistent with legal precedents, such as the Florida district court decision, which was affirmed by the 11th Circuit.

The FDA filed an appeal to the Court of Appeals for the Ninth Circuit. Two leading global stem cell organisations – the International Society for Cell and Gene Therapy, and the International Society for Stem Cell Research – jointly filed a brief in support of the appeal.

In 2024, the Court of Appeals for the Ninth Circuit reversed the district court judgment in favour of the defendants. The three-judge panel held that the defendant's SVF consists of a drug under the FDCA based on the plain text of the statute. The panel rejected the defendants' contention that even if the SVF is treated as a drug, the same-day SVF treatment is exempt from FDA regulation under the SSP exception.

They acknowledged that the human cells, tissues and cellular and tissue-based products regulations lack clarity regarding the meaning of the exception. Thus, they would have to explore the context and structure and interpret the ambiguity in favour of the FDA.

'It's important to note that the Supreme Court's decision not to review a lower court decision is not a decision on the merits,' according to Hank Greely, professor of law at Stanford University. 'The Court could quite easily take up this same question coming in another case, particularly if it comes from a different lower appellate court.'

'On the other hand, the fact that it did not review this case does make it more likely that it will not choose to review a future case, especially as long as all the federal appellate courts to consider the question (two, so far) have reached the same conclusion,' he added.

While the Supreme Court's ruling affirms the FDA's stringent oversight of unproven stem cell treatments, it is unclear whether this success will lead to greater enforcement against unapproved stem cell procedures under the Trump administration. Nor is it clear whether the agency's enforcement efforts will change because of shifting priorities of the current administration, as well as risks of future administrative law challenges to the FDA regulations.