The US Food and Drug Administration (FDA) on Wednesday warned stem cell product supplier American CryoStem Corporation for marketing an unapproved product after the agency inspected the company's Eatontown, New Jersey site in July.
The warning letter comes just months after FDA announced a new framework for regulating regenerative medicines that included a 36-month period of enforcement discretion by the agency, though at the time, an FDA spokesperson told Focus the agency would not exercise enforcement discretion for products "that pose a significant safety concern."
Despite the mention of enforcement discretion in the agency's guidance, FDA Commissioner Scott Gottlieb on Thursday said the agency will "be stepping up our enforcement activities against those who manufacture and market products in ways that put patients at risk."
Leigh Turner, associate professor at the University of Minnesota Center for Bioethics, told Focus that while it's difficult to make out an overall trend in FDA's enforcement activities in the wake of the new framework, his sense is that the agency will continue to increase enforcement efforts.
Turner also said that going after suppliers of unproven stem cell therapies is a good use of FDA's limited resources, and that action against a publicly traded stem cell supplier will have more visibility than action against individual stem cell clinics.
"I think this is one that when you look at federal regulations, when you look at the final guidances that FDA put out, this was a business that was not complying," Turner said.
According to FDA, American CryoStem's Atcell product uses adipose tissue harvested from patients and processed into stromal vascular fraction (SVF) before being expanded through cell culture and sent back to physicians to be administered. FDA says these processes go beyond "minimal manipulation" and thus do not meet the regulations under Section 361 of the Public Health Service Act.
FDA also says that Atcell fails to meet requirements for homologous use, as American CryoStem markets the product to treat a number of conditions, including Parkinson's disease and multiple sclerosis, where its use would go beyond the basic functions of adipose tissue.
"Indeed, American CryoStem's Chief Operating Officer, Anthony Dudzinski recently acknowledged that American CryoStem's 'cellular expansion' therapies (like Atcell) are 'breakthrough technologies which are about sending cells the same way you send any other drug to a doctor,'" FDA writes.
As such, FDA says that Atcell fails to meet exemptions for minimal manipulation and homologous use and would require approval under a biologics license application (BLA) before being marketed.
FDA also says it found "significant deviations from current good manufacturing practice (cGMP)" that raise serious safety concerns, including the use of unvalidated manufacturing processes, inadequately controlled environments and a lack of control of the components used in manufacturing.
These issues, FDA says, create a significant risk that Atcell could be contaminated or have other serious quality defects.
In its response to FDA's inspection observations, American CryoStem said that Atcell is distributed for "research use only" and is being studied in an institutional review board (IRB) approved study. However, FDA says that an investigational new drug (IND) application is required before beginning study in humans.
Warning Letter, FDA