The US Food and Drug Administration (FDA) has audited Celltex Therapeutics Corporation, a stem-cell treatment purveyor based in Sugarland, Texas. The results aren’t pretty, and this is only the beginning.
The company has been providing unproven and unapproved stem-cell treatments to patients suffering various disorders. Nature has previously suggested that an audit was necessary. Leigh Turner, a bioethicist at the University of Minnesota in Minneapolis, wrote directly to the FDA to ask for one.
Two investigators from the FDA visited Celltex over 16–27 April. They made 14 general observations about the ways in which Celltex cell-manufacturing procedures are not up to scratch, including failure to ensure the cells are uniform, viable and sterile. These failings are rooted in procedural failures, such as not monitoring the temperature or humidity of the processing rooms and mislabelling or failing to label containers. One bottle had contradicting labels in English and Korean — probably indicating a quality-control problem in integrating reagents from RNL Bio, based in Seoul. RNL Bio, which offers its own controversial stem-cell treatments and has been investigated after the death of two patients, licenses its stem-cell manufacturing technology to Celltex. Altogether, the FDA found 79 deficiencies.
That is alarming considering that Celltex has been injecting cells into patients. Even if stem cells are produced through proper procedures, there are uncertainties over the safety of injecting them into people. The poor manufacturing record shown in the FDA document indicates that patients may have been subjected to much greater danger than necessary.
The FDA observations come in a Form FDA 483 document. It is expected that, within 15 business days, recipients respond point by point, indicating actions that have been or will be taken to address each problem. Responding is not compulsory, but failure to do so could lead to more stringent actions by the FDA, including the possibility of shutting down operations.
The observations left open the question of whether Celltex will be censured for treating patients without approval, as Regenerative Sciences, based in Broomfield, Colorado, was in 2010. Unlike RNL Bio, which, in recognition of the fact that such stem-cell treatments are not allowed in the United States or its home country of South Korea, sends patients to Japan or China for the actual injection, Celltex has been paying doctors in Texas to inject its cells into people. (The company chief executive, David Eller, has denied that the company treats patients, but a doctor who injects Celltex cells into patients says that Celltex pays him and others US$500 per injection.) For the time being, the FDA seems to be approaching Celltex just as a stem-cell manufacturer — the observation form calls it a “biological drug manufacturer”. But the clinical use is a separate problem. Another shoe is yet to drop.
UPDATE 28 June 2012:
Celltex has now responded to the flurry of news stories about the FDA’s audit of its facilities. In a press release it blamed the negative audit on language difficulties arising from its partnership with RNL Bio, the Seoul-based stem cell company that had licensed its technology to Celltex. It says:
Celltex’s laboratory is currently operated by its licensing partner RNL Bio (dba Human Biostar), with lab technicians and scientists from RNL’s Seoul, Korea headquarters. The main issues in the FDA observations arose from a language barrier. RNL scientists extensively document procedures, including validations, but they are recorded in Korean and were not able to be provided in English to FDA during its visit. Since the FDA’s visit, the RNL procedures and other documents have been translated to English by an independent, professional translation service, and supplied to the agency.
The company is also at pains to point out that:
Some media reports and social media chatter suggest that Celltex is somehow acting illegally or providing unapproved treatments. These statements are inaccurate.
We have resolved many of the FDA observations, and we are working to address the remainder.
Yet it directly contradicts a key finding of the FDA’s audit — that Celltex’s procedures were insufficient to ensure the provenance and sterility of the cells its produces — saying:
Celltex ensures that all of the cells it provides to physicians for therapeutic use are sterile, viable, intact mesenchymal stem cells.
In the press release, Celltex also sticks to the idea that it is only a distributor of stem cells and, as such:
Celltex’s process for reproducing adult mesenchymal stem cells is legal, and there is no requirement that the cells be approved or licensed.
But Celltex also pays physicians to inject patients with the cells (see ‘Stem-cell therapy takes off in Texas‘). The clinical use of such cells does require FDA approval.