Cell therapies are a new and emergent entity in the biopharma space, and last year’s 21st Century Cures Act looked to address industry’s regulatory concerns by introducing the Regenerative Medicine Advanced Therapy (RMAT) designation offering personalised medicine makers an “efficient development program for, and expedite review of, [their] drug if the drug qualifies as a regenerative advanced therapy.”
But two separate enforcement actions in the past week have shown the abuse of such therapies within the US, and pushed the US Food and Drug Administration (FDA) to step up its regulation of the sector.
“Stem cell clinics that mislead vulnerable patients into believing they are being given safe, effective treatments that are in full compliance with the law are dangerously exploiting consumers and putting their health at risk,” FDA commissioner Scott Gottlieb said.
“As the FDA takes new steps to advance an efficient, modern approach to the regulation of cell based regenerative medicine, at the same time we will be stepping up our enforcement actions against clinics that abuse the trust of patients and, more important, endanger their health with unsanitary conditions or by purporting to have treatments which may not provide any benefit.”
Warning letter in Florida
US Stem Cell Clinic and its Chief Scientific Officer Kristin Comella received a warning letter for marketing its autologous stem cell products without FDA approval, as well as significantly deviating from current good manufacturing practice (cGMP) requirements in the manufacture of at least 256 lots.
The firm, located in Sunrise, Florida, claimed to offer stem cell treatments for a range of neurological, autoimmune, orthopaedic and degenerative diseases based on a patient’s own adipose tissue, which are processed into stromal vascular fraction (SVF) before being administered back into the patient.
However, the products are not the subject of an approved biologics license application (BLA) and have not demonstrated safety and efficacy. As such, the FDA said in a statement the company abused the trust of patients and put their health at risk and vowed to step up its vigilance against others offering unregulated stem cell treatment.
StemImmune and Smallpox
And yesterday the Agency announced it took action to stop the use of an unproven treatment developed by StemImmune Inc set to be administered to patients at the California Stem Cell Treatment Centers in Rancho Mirage and Beverly Hills, California.
Under the FDA’s orders, five vials containing 100 does of the non-commercially available Vaccinia Virus Vaccine (Live) were seized by the US Marshals Service. The vaccine was intended for combination with stromal vascular fraction to create an unapproved stem cell product intended to be directly injected into cancer patients’ tumours.
The Agency said it investigating how StemImmune sourced the vaccine, which is reserved only for people at high risk for smallpox and is not commercially available.
Gottlieb also attacked the “audacious but ultimately hollow claims made by these kinds of unscrupulous clinics or others selling so-called cures.”
He added: “The FDA will not allow deceitful actors to take advantage of vulnerable patients by purporting to have treatments or cures for serious diseases without any proof that they actually work. I especially won’t allow cases such as this one to go unchallenged, where we have good medical reasons to believe these purported treatments can actually harm patients and make their conditions worse.”
Furthermore, he pledged to “vigorously investigate these kinds of unscrupulous clinics using the full range of our tools, be it regulatory enforcement or criminal investigations.”