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The U.S. Food and Drug Administration (FDA or Agency) has become increasingly active in stem cell and regenerative medicine product regulation within the past year previously announcing that it would release a modified framework for the regulation of these products in September. On August 28, FDA issued two news releases about recent enforcement actions taken against Florida and California stem cell companies and clinics, as well as a statement from FDA Commissioner Dr. Scott Gottlieb. Dr. Gottlieb noted the promise of adult stem cells as viable therapies with the appropriate testing and scientific evidence, and vowed to work with industry. He also, however, stated that the Agency would increase its enforcement activities against “unscrupulous actors” in this field.

Recent Enforcement Actions

On August 24, FDA issued a warning letter to U.S. Stem Cell Clinic, LLC in Sunrise, Florida. for violations of the Federal Food, Drug, and Cosmetic Act (FFDCA) – namely, that the clinic (1) produced and administered unapproved stem cell treatments to patients; (2) violated current good manufacturing practices (cGMPs) and current good tissue practices (cGTPs); and (3) impeded FDA’s inspection of the clinic. This same clinic was the subject of widespread stories in the popular press after publication of a New England Journal of Medicine article in March 2017 that reported varying degrees of vision loss in three patients who were treated with stem cells at the clinic. One day after FDA’s news release about the warning letter, the clinic pushed back at the Agency by posting a public response and video.

On August 25, the U.S. Marshals Service seized non-commercial vials of live Vaccinia virus vaccine from StemImmune Inc. of San Diego, California. StemImmune allegedly used this vaccine, intended for very limited use against smallpox, to create an unapproved stem cell product by mixing the vaccine with stem cells and administering the product to cancer patients at the California Stem Cell Treatment Centers in Rancho Mirage and Beverly Hills, California. The U.S. Department of Justice filed the seizure complaint and warrant on behalf of FDA in the U.S. District Court for the Central District of California (Southern Division).

FDA Proposals

These two recent events prompted Commissioner Gottlieb to issue a statement on August 28 about the future of regenerative medicine regulation at FDA. Dr. Gottlieb acknowledged the “small number of unscrupulous actors” that risk tarnishing the regenerative medicine field as a whole, as well as the “sincere reports of the significant clinical potential of properly developed products.” While promising additional details in the coming months, Dr. Gottlieb announced the following:

  • FDA will launch a new working group to use “whatever legally enforceable means are necessary” to protect the public from companies and clinics that do not abide by FDA regulations and therefore put the public health at risk.
  • FDA will release a new framework this fall to establish when a regenerative medicine product is subject to FDA’s jurisdiction, and when such a product can be used by a physician as part of his or her practice of medicine without FDA oversight. This framework will be communicated through a series of guidance documents based upon previous public input on regenerative medicine therapies.
  • FDA will issue a compliance policy to provide “a very reasonable period of time” for companies currently producing stem cell treatments to approach FDA to determine whether an FDA-approved application for the treatment is required.
  • FDA will create a “novel approach” for stem cell approval to allow “very small product developers to gain all the benefits of FDA approval through a process that is minimally burdensome and less costly.”
  • Finally, FDA will continue to implement the Regenerative Medicine Advanced Therapy (RMAT) designation, established in Section 3033 of the 21st Century Cures Act, that provides expedited development and approval pathways for regenerative medicine therapies.

What Companies Should Do

Although FDA has not yet released the full scope of its plans, companies, clinics and physicians currently developing or using stem cell products must recognize that the Agency is committed to taking enforcement actions when necessary to protect the public health. Therefore:

  • Assess your current activities and stem cell-based products to determine if they are subject to FDA oversight and, if so, to what degree. Use the current cell and tissue regulations as the basis for your assessment. Even though FDA has promised changes, we do not expect the Agency to completely upend its current regulatory framework; rather, FDA will likely introduce modifications while keeping much of the existing framework intact.
  • Review your manufacturing processes for compliance with cGMPs and cGTPs.
  • Review any materials that define your product and processes, including any promotional statements and website content. As is often stated about product promotion, “You are what you claim.” Words do matter.
  • Stay on top of new statements, warning letters and enforcement actions issued by FDA to help you continue to navigate the current – and changing – landscape.