Statement from Norman E. "Ned" Sharpless MD, Acting Commissioner and Dr. Peter Marks M.D. PhD., Director - Center for Biologics Evaluation and Research (CBER)

The U.S. District Judge Ursula Ungaro of the Southern District of Florida issued an order that stops US Stem Cell Clinic, of Weston, Florida, and US Stem Cell, of Sunrise, Florida, and their Chief Scientific Officer Kristin Comella, Ph.D., from manufacturing or distributing any and all stromal vascular fraction (SVF) products, which are adipose (fat) tissue derived stem cell products, until they come into compliance with the law.

The U.S. Department of Justice, on FDA’s behalf, initiated this action against US Stem Cell Clinic and US Stem Cell and Comella in May 2018, seeking a permanent injunction after they failed to come into compliance with the law, including FDA regulations intended to protect patients from harm. On June 3, 2019, the Court granted the government’s motion for summary judgment after concluding that the defendants adulterated and misbranded their cellular products made from patients’ adipose tissue.

The action by Judge Ungaro is significant and sends a strong message to others manufacturing violative stem cell products. Court decisions like this reaffirm the FDA’s compliance and enforcement efforts in the ongoing fight to protect the public from individuals and clinics who mislead patients with unapproved and potentially harmful medical products. The agency will continue to take steps—such as issuing warning letters or initiating court cases—against those who endanger patients’ health with inadequate manufacturing conditions or by manufacturing and marketing products without the submission of appropriate applications containing safety and efficacy information to the FDA.

Patient safety is the foundation of our mission. We’re now nearly halfway through the period during which the agency intends to exercise enforcement discretion for certain regenerative medicine products with respect to the FDA’s investigational new drug application (IND) and premarket approval requirements – but only for those where the use of the product does not raise reported safety concerns or potentially significant safety concerns. The pace of progress of those offering these human cell and tissue-based products, including stem cell products, to come into compliance with the requirements has been slower than expected.

We know that there are clinics across the country that manufacture or market violative stem cell products to patients, claiming that they don’t fall under the regulatory provisions for drugs and biological products. The FDA has consistently stated that this is not true, and the result of this case proves that. There are many examples of companies deceiving patients with unsubstantiated claims about the potential for stem cell products to prevent, treat or cure serious diseases, and in those cases, we are committed to acting to protect patients. These actors are taking advantage of patients, many in vulnerable positions with chronic or terminal diseases, by leveraging the widespread belief in the eventual promise of these products, flouting the statutes and our regulations. This ultimately puts at risk the very patients that they claim to want to help, by either delaying treatment with legitimate and scientifically sound treatment or, worse, posing harm to patients, such as blindness, infection or possibly death.

For sponsors whose stem cell products are subject to premarket approval under the statutes and existing regulations, where the products create more significant potential risks because of the way that they’re manipulated or delivered, and who haven’t engaged the regulatory process to properly develop these products, we’ll continue our aggressive oversight, prioritizing resources to address those clinics, individuals and products that put patients most at risk. In fact, over the past year, we have sent 46 manufacturers and health care professionals regulatory correspondences, including warning letters, and one court case is ongoing.

But we also recognize that there is significant interest from many stakeholders and patients interested in seeing the safe development of legitimate, novel stem cell products. We remain firmly committed to advancing an efficient path for the development of safe, effective and innovative regenerative medicine products. There are many investigators and firms legitimately developing such products, fully in keeping with our statutes and regulations, examining their safety and potential benefits in treating a range of diseases and conditions, including cancers, heart failure, stroke and ALS (amyotrophic lateral sclerosis).

Our work to ensure compliance with the law and to protect patients does not take away from our firm commitment to advance an efficient path for the safe and effective development of novel regenerative medicine products and to help foster beneficial new innovations. We’ll continue to work closely with investigators and firms legitimately working in this field and will do so in the most effective manner possible, while meeting the FDA’s standards for safety and efficacy. Over the past year, the FDA has used a variety of tools to support such development, including providing advice through CBER’s INTERACT meeting process and through use of expedited programs, such as Regenerative Medicine Advanced Therapy designation, for those products that qualify, and our recently launched Tissue Reference Group Rapid Inquiry Program (TRIP) to also help manufacturers with informal, preliminary and non-binding assessments from the FDA regarding how specific cell and tissue-based products are regulated.

Stem cell products hold significant potential to improve human health. However, that potential will never be fully realized if careful scientific work and thoughtful clinical investigation supporting the safety and efficacy of these products are not conducted. The FDA is committed to helping advance the safe and effective development of novel stem cell products. We look forward to working with those who share our goal of bringing safe and effective products to market to benefit individuals in need.

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