Stem cell therapy, also known as regenerative medicine, has been around for decades, but in recent years, the use of and interest in stem cell therapy has increased exponentially. The dramatic utilization of stem cell therapy, and the increasing government spend related to these novel techniques, have now caught the eye of federal regulators and prosecutors. In this client alert, we profile some brief context of stem cell therapy, the government’s regulations governing these techniques, and some of the best practices for those interested in this emerging space.
Background on Stem Cell Therapy
Stem cells are “cells from which all other cells with specialized functions are generated” (i.e., the body’s “raw materials”). Stem cells may duplicate themselves to create more stem cells or they may generate cells with a specific function like blood or brain cells.
Stem cell therapy is used to repair or replace damaged tissue or cells within the body. Many in the medical community are hopeful that stem cell therapy can be used to treat a wide array of conditions and diseases from multiple sclerosis to vision loss to traumatic spinal cord injuries to Lou Gehrig’s disease — just to name a few.
The FDA’s Role and Concerns
The Food and Drug Administration (FDA) oversees and regulates stem cell therapy treatments. While the FDA has acknowledged that stem cell therapy has the “potential to treat diseases or conditions for which few treatments exist,” there are still only a few treatments that have actually been approved by the FDA. Many treatments are still only in early investigatory stages.
The FDA has recognized the massive potential that stem cell therapy has in allowing patients treatments for various conditions. Consequently, in 2017, the FDA issued guidance indicating its intent to “exercise enforcement discretion” as a means “to support and expedite the development of regenerative medicine products.” This “enforcement discretion” period was to allow innovators time to determine whether “to submit an Investigational New Drug (IND) or marketing application and, if such an application is needed, to prepare and submit the application as appropriate.” The FDA, however, has made clear its enforcement discretion policy only applies to “products that do not raise potential significant safety concerns.” What the FDA considers “significant” is debatable, creating uncertainty and ambiguity for those who might be relying on the FDA’s enforcement discretion period.
Initially, the FDA stated that its enforcement discretion period would last through November 2020. But in July 2020, the FDA extended its enforcement discretion period through May 2021 — a fast-arriving date. It remains unclear whether the FDA intends to extend the time period of its enforcement discretion any further, but either way, stem cell therapy providers would be well-served by planning for and expecting enforcement efforts to ramp up in the near future.
In 2019, the FDA went to great lengths to warn consumers of the potential fraud that may arise from what it called stem cell therapy “hype,” and encouraged consumers to make sure any stem cell therapy treatments were either approved or being studied as an IND. The FDA’s concerns have led to multiple enforcement actions, including one just last month. On February 1, 2021, for example, the government announced the indictment of Ashton Derges, a healthcare provider in Missouri, who marketed “stem cell shots” as a successful treatment for various conditions, including COVID-19. According to the indictment, Derges was paid nearly $200,000 by patients for the “stem cell shots,” none of which actually contained stem cells at all. While this alleged fraud was not particularly sophisticated, it nonetheless marked a significant development: the government’s first criminal prosecution of those touting stem cell therapies.
But blatant fraud is not the only type of stem cell therapy case the government has expressed interest in investigating. A primary concern of the government is the marketing and use of unproven stem cell treatments as miracle cures. A good case study of the risks associated with aggressive marketing of stem cell therapy is a case out of Florida involving US Stem Cell Clinic Inc. The clinic was marketing stem cell therapy to treat conditions and diseases such as Parkinson’s disease, stroke, and brain injuries — none of which were approved by the FDA. And, much of the marketing that US Stem Cell Clinic used promised almost miraculous results. As a result, last year, the FDA successfully permanently enjoined the US Stem Cell Clinic from selling or providing those stem cell therapy treatments. Notably, this case was pursued by the FDA despite the FDA explicitly stating its intent to be lenient with emerging stem cell therapy treatments.
Stem cell therapy is a groundbreaking medical tool with great possibilities to treat a plethora of diseases and conditions. As the industry continues to expand, so will the government’s interest. Our firm continues to see an uptick in cases involving stem cell therapy treatments. And we have successfully assisted clients in avoiding unnecessary scrutiny by the FDA and other government regulators.
If you are in the stem cell therapy industry or are considering offering stem cell therapy treatments, we recommend that you:
- Carefully monitor the emerging FDA guidance. The guidance on this topic is changing as quickly as the medicine, and it is important to remain abreast of the FDA’s latest stance on stem cell therapies. If you have questions, consult with a lawyer — or call us — and we will be happy to walk you through the latest guidance.
- Be mindful of marketing materials. The FDA and DOJ have focused on companies with ultra-aggressive marketing materials as the prime targets. Carefully ensure that every representation is backed by verifiable findings.
- Review payment transactions. The DOJ is applying its traditional prosecutorial theories to emerging therapies. And, an easy way to get tripped up in the crosshairs of the government is by running afoul of the Anti-Kickback Statute, a statute which penalizes payment for referrals of healthcare business. Therefore, providers should ensure that all payments reflect actual services rendered and that these payments comport with the broad mandates of the Anti-Kickback Statute.
- Finally, providers should ensure that they proactively consult counsel. Many of the theories of liability the government pursues are intent based. And, accordingly, providers that consult with legal counsel often can avail themselves of defenses that others cannot. As the adage suggests, an ounce of prevention is worth a pound of cure.
Attorneys Jason P. Mehta, Matthew W. Bedan, and Giovanni P. Giarratana are members of Bradley’s Government Enforcement and Investigations Practice Group in Tampa, Florida. They have extensive experience assisting clients in navigating government investigations and enforcement actions and offer practical, proactive compliance strategies to avoid them.