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Statement by FDA Commissioner Scott Gottlieb, M.D., and Biologics Center Director Peter Marks, M.D., Ph.D. on FDA’s continued efforts to stop stem cell clinics and manufacturers from marketing unapproved products that put patients at risk, while…

Stem cell therapies offer the potential to treat or even cure diseases or conditions for which few effective treatment options exist. Cell based therapies are one of the most promising new fields of science and medicine. These products hold tremendous potential for transformative and potentially curative treatments for some of the most devastating diseases and conditions challenging us today.

For several decades now, stem cells obtained from the bone marrow or peripheral blood have had an established role in providing valuable treatment for patients with certain cancers, like leukemia, and certain other serious diseases and conditions, such as aplastic anemia. But most forms of regenerative medicine are still in early stages of development and adult stem cells and stem cells from birthing tissues have not yet been shown to be safe and effective for use in the treatment of any other diseases or conditions.

Legitimate and well-intentioned investigators and firms are doing the essential groundwork to develop such products, fully in keeping with our statutes and regulations, examining their safety and potential benefits in a range of diseases and conditions, including cancers, heart failure, stroke and ALS (amyotrophic lateral sclerosis). But some actors are leveraging the widespread belief in the eventual promise of these products, flouting the statutes and our regulations, and deceiving patients by illegally manufacturing or selling purported therapies, and falsely promoting their benefits. This ultimately puts at risk the very patients they claim to want to help.

It’s important to recognize up front that there is a well-founded and longstanding rationale, established in statute and regulation, why certain stem cell therapies are subject to careful development requirements under the FDA’s existing regulations. There’s a false premise being asserted by some in the field that a product derived from a person’s own body and then manipulated and reinserted for another use different from the one it played in its original location is not subject to FDA regulation just because it originated from the person it was given back to. But stem cell products can create unique and serious risks depending on how they’re manipulated once they’re taken from the body and how they are used once they’re reinserted in the body. When a product undergoes more than minimal manipulation, its characteristics may be changed in a way that are novel, and its risks and potential benefits unique.

We’ve seen too many cases of sponsors claiming that cells aren’t subject to FDA regulation just because the cells originated from the same patient to whom the eventual manufactured product is being given. And we’ve seen too many cases of companies making unsubstantiated claims that these treatments prevent, treat, cure or mitigate disease where the products have sometimes led to serious patient harm.

Patient safety is our first priority. These violative actions create a direct risk to patients. They also create indirect risks by potentially encouraging them to forgo otherwise effective, available treatments, and opt instead for purported treatments that create risks and offer no demonstrated benefits. These kinds of false claims and violative activities also do a tremendous disservice to innovators who are working to legitimately develop safe and effective stem cell therapies by casting doubt across the entire field.

We’re now nearly halfway through the period during which the FDA 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 when the use of the product does not raise reported safety concerns or potential significant safety concerns, which we first announced in our November 2017 guidance on regulatory considerations for human cells, tissues, and cellular and tissue-based products. We’ve seen modest progress by the industry in coming into compliance, but much more work needs to be done. There’s no room for companies that place patients at risk through products that violate the statutes and our regulations, including by not having in place an IND or biologics license. As we come up on the end of this period during which the FDA intends to exercise enforcement discretion, we may take additional steps to delineate an efficient development path for promising products that pose lower risk to patients and that are being developed by sponsors who’ve engaged the regulatory process in a responsible manner by filing INDs. These would be cases where the sponsors have undertaken or are in the process of undertaking well-designed investigational studies with the intent of collecting information to more clearly identify the safety and benefits of their products.

But for sponsors whose stem cell products are regulated as drugs, devices, and/or biological products 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 step up our oversight. Over the past year, we have sent 45 manufacturers and health care providers regulatory correspondence, including warning letters, and we have two court cases pending. We’re committed to taking appropriate steps to address those that jeopardize the health of the people we are sworn to protect.

Today, we’re continuing these efforts. The agency issued a warning letter to Cord for Life, Inc., located in Altamonte Springs, Florida, for manufacturing unapproved umbilical cord blood products in violation of current good manufacturing practice (CGMP) requirements, including failing to validate processes to prevent bacterial contamination, raising potential significant safety concerns that put patients at risk. In addition, we issued 20 letters today to separate manufacturers and health care providers across the country who may be offering unapproved stem cell products, reiterating the FDA’s compliance and enforcement policy. We remain very concerned that countless clinics across the country continue to market violative stem cell products to patients, claiming that they don’t fall under the regulatory provisions for drugs and biologics. This is simply not true. The FDA has had final regulations in place for these products for well over a decade. We intend to take steps to help make sure that products are produced and marketed in compliance with statutes and our regulations. We’ll continue to conduct inspections, as appropriate, to help ensure such compliance. And we will not shy away from taking further steps when we see bad actors taking advantage of patients, and putting them at serious risk, for their own financial profit.

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 work closely with investigators and firms legitimately working in this field to do so in the most effective manner possible, while meeting the FDA’s standards for safety and efficacy as is expected with all drug and biological products. 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.

We acknowledge the challenges that product development can present for smaller entities such as academic institutions and group practices. With the goal of helping to facilitate the development of stem cell products, and to help small groups of collaborating investigators, the FDA continues to encourage sponsors to discuss novel clinical development approaches with the agency, such as the collaborative development model highlighted in our guidance Expedited Programs for Regenerative Medicine Therapies for Serious Conditions. In addition, during the next year, the agency will explore whether there are additional ways that it can assist legitimate developers of stem cell products to come into compliance with its regulations.

We remain committed to supporting patients’ access to safe and effective stem cell therapies, by ensuring that the process is as efficient as possible under applicable FDA regulations governing product investigation, manufacturing and approval. Although it’s our hope that product developers will voluntarily come into compliance with our regulations, it’s also our responsibility to take appropriate steps to address those unlawfully manufacturing or distributing products. This is especially true when it comes to products that may pose a significant risk to patients, including because of the way the products are being manipulated or used.

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

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.