FDA Seeks Injunctions in Fight Against $2.4bn Unapproved Stem Cell Market

Industry group International Society Cell and Gene Therapy (ISCT) has supported regulatory actions aimed at permanently closing two unapproved US stem cell clinics.

The US Food and Drug Administration (FDA) is seeking a permanent injunction against Sunrise, Florida-based US Stem Cell Clinic LLC and Californian firm California Stem Cell Treatment Center Inc for marketing stem cell products without approval.

The actions are the latest in a crackdown by the regulator on unproven cellular therapies (UCT) and complement a suite of new guidance documents issued last November aimed at detailing a science-based process for regenerative medicines.

“Cell-based regenerative medicine holds significant medical opportunity, but we’ve also seen some bad actors leverage the scientific promise of this field to peddle unapproved treatments that put patients’ health at risk,” FDA commissioner Scott Gottlieb said.

“The FDA will continue to take enforcement actions against clinics that abuse the trust of patients and endanger their health with inadequate manufacturing conditions or by purporting to have treatments that are being manufactured and used in ways that make them drugs under the existing law but have not been proven safe or effective for any use.”

US$2.4 Billion Unproven Stem Cells Industry

ISCT – a group made up of clinicians, researchers, regulatory specialists, technologists and industry partners in the cell and gene therapy sector – welcomed the injunctions and proposed further actions to clampdown on unproven cellular therapies (UCTs).

“It is vital that regulators, as recently demonstrated by the FDA, continue to identify and shut down clinics conducting unproven and potentially harmful cellular interventions,” said ISCT chair Massimo Dominici.

“However, all representatives of the ISCT Presidential Task Force agree that the job cannot be left to regulators only. There is no magic bullet for UCT with complex challenges. Patients affected by diseases with a high-unmet medical need have few options. It is up to the cell therapy community to collectively drive more therapies through approval, reduce costs through innovative development and manufacturing strategies and educate the public and patients about the dangers of unproven cellular therapy.”

According to ISCT, the size of the UCT market is currently US$2.4 billion (€2 billion) and involves around 60,000 patients annually, who pay as much as $40,000 per treatment.

US Stem Cell Clinic

US Stem Cell Clinic (USCC) is focused on the “discovery, development and commercialization of cell based therapeutics that prevent, treat or cure disease by repairing and replacing damaged or aged tissue, cells and organs and restoring their normal function,” its website claims.

But an FDA warning sent in August 2017 accused the firm of processing adipose tissue into stromal vascular fraction and administering the product both intravenously or directly into the spinal cord of patients to treat a variety of serious diseases, without regulatory review or approval. The FDA also found significant deviations from current good manufacturing practice (cGMP) requirements during a visit to the clinic in May 2017.

The clinic responded to the warning, with chief scientific officer Kristin Comella claiming the criticisms the FDA made relate to the manufacture of drugs, and “not something that a medical clinic would be required to follow.”

She continued: “USCC is not manufacturing a drug.  That being said, in an abundance of caution, US Stem Cell Clinic will immediately switch to 510(k) approved systems to process tissue in clinic such as bone marrow systems and/or fat systems.”

Also in August, the FDA took action to stop the use of an unproven treatment developed by StemImmune Inc being administered to patients at the California Stem Cell Treatment Centers in Rancho Mirage and Beverly Hills, California.

Five vials containing 100 does of the non-commercially available Vaccinia Virus Vaccine (Live) were seized by the US Marshals Service, under the FDA’s orders. The vaccine had been intended to be used to create an unapproved stem cell product intended to be injected into cancer patients’ tumors.