The FDA has cautioned Genetech, Inc. for processing umbilical cord blood into unapproved human stem cell products that may have contributed to the sickening of 12 people.
Editors Note: A previous version of this article identified Genetech as Genentech. Updated 1/3/2019 at 8:28 AM EST
Stem cells save lives.
They may also be a source of infection.
The US Food and Drug Administration (FDA) has issued a warning to pharmaceutical company Genetech, Inc. after unapproved umbilical cord blood-derived stem cell products marketed by the company and distributed by Liveyon were found to have caused 12 cases of bloodstream and/or joint infections in patients who had received them via injection or infusion in outpatient clinics in Texas (7 cases), Florida (4 cases), and Arizona (1 case). All 12 patients affected were hospitalized—1 for nearly 2 months; as yet, no fatalities have been linked with the stem cell products.
“The company’s failure to put in place appropriate safeguards may have led to serious blood infections in patients,” FDA Commissioner Scott Gottlieb, MD, said in a statement announcing the warning. “We remain committed to supporting the development of safe and effective cell-based regenerative medicine and advancing our comprehensive regenerative medicine policy framework.”
In addition to the specific warning to Genetech, the FDA has issued letters reiterating its compliance and enforcement policies to other manufacturers and health care providers who may be offering stem cell treatments. Dr. Gottlieb noted that to date, though, the agency has been “discouraged by the overall lack of manufacturers” who are willing to work with it in improving oversight and quality control in stem cell processing.
“The letters…are a reminder that there’s a clear line between appropriate development of these products and practices that sidestep important regulatory controls needed to protect patients,” he added. “We’ll be increasing our oversight related to cell-based regenerative medicine as part of our comprehensive plan to promote beneficial innovation while protecting patients.”
In a report on the FDA warning published on December 21, 2018, The Washington Post noted that the US stem cell industry has grown significantly in recent years, with hundreds of clinics nationally selling the products for the treatment of conditions ranging from Parkinson’s disease to multiple sclerosis. Currently, the only FDA-approved indication for umbilical cord blood-derived stem cells is hematopoietic or immunologic reconstitution.
In a “Notes from the Field” brief also published on December 21, 2018, in the US Centers for Disease Control and Prevention’s (CDC) Morbidity and Mortality Weekly Report (MMWR), investigators from the agency’s Division of Healthcare Quality Promotion in the National Center for Emerging and Zoonotic Infectious Diseases reported that the identified bloodstream infections linked with the stem cell products included Enterobacter cloacae and Citrobacter freundii, while the joint infections found were Escherichia coli, Enterococcus faecalis, and Proteus mirabilis. The vast majority of cases were reported after the CDC issued a nationwide call for reports of culture-confirmed infections in patients who had received the Genetech/Liveyon product in October. Liveyon has since ceased distribution of the stem cell products and has issued a recall for those already in circulation.
“CDC tested unopened vials obtained from the Texas and Florida clinics where the initial patients had received the product,” the authors of the MMWR article wrote. “The 6 vials from Texas had the same cord-blood donor and processing date as those that had been administered to the patients with infections. The 4 vials from Florida were from different donors and processing dates than were the vials from Texas.”
According to the authors of the brief, investigations into additional active cases, laboratory testing of affected stem cell products, onsite assessments of health care facility infection control, and manufacturing practices (including distribution) are ongoing.
“Initial investigation suggests that bacterial contamination occurred before distribution,” they wrote. “Umbilical cord blood cannot be decontaminated after collection because there are currently no validated processes for sterilization, so manufacture of derived products must be highly controlled to prevent distribution of contaminated products. Regardless of when contamination occurred, this investigation highlights the serious potential risks to patients of stem cell therapies administered for unapproved and unproven uses.”