Today I received a copy of the so-called “483 report” of that audit. It is basically a summary of problematic issues that the FDA says need to addressed.
The FDA report is cause for some significant concern about Celltex in my opinion.
Further, the report and the concerns it raises are also a big challenge for the adult stem cell industry more generally. In fact, I view it as a major setback to the adult stem cell for-profit industry as a whole.
Because Celltex has been in the limelight so much of late, in part because they were involved in Texas Governor Perry’s stem cell transplants, that it has become somewhat of a poster child for the adult stem cell industry.
You can read the partially redacted 9-page FDA report on Celltex here (PDF). An image of the first page is below.
It is unclear why the FDA redacted some portions, but it could be to preserve proprietary information.
I received the report from Dr. Leigh Turner (hat tip) of University of Minnesota who bravely was the one who originally requested that the FDA look into Celltex and was later threatened with litigation.
Just recently, Dr. Turner put in an FOI request to the FDA for the report on their audit of Celltex after reading my blog post indicating that Celltex had been audited. This is how the report has now been made public.
The FDA audit makes 14 observations about Celltex in their report that are collectively broken down into 79 individual bullet points. FYI, HCT/Ps are defined by the FDA as “Human Cells, Tissues, and Cellular and Tissue-Based Products”. Don’t let acronyms scare you off. I summarize my thoughts on the report below in plain to understand English.
Note that a preliminary 483 report is just that–preliminary. That means the FDA later can and often does provide additional comments and concerns to companies such as Celltex that get audited and certainly can take further actions if it deems appropriate in the future.
What does the FDA 483 report on Celltex mean?
Amongst other things, the FDA seems to be arguing (rightly or wrongly) in the audit report that the Celltex end product that goes into patients via other doctors and clinics cannot be assured to
- (1) contain living cells of a specific type and number
- (2) be sterile, and
- (3) be properly labeled.
The FDA also seemed concerned about a lack of documentation and validation as well as some aspects of how the Celltex facility is designed and run.
All are serious issues in my way of thinking and I’m not one to ever say the FDA is wrong.
The tube labeling concern is more important than you might think on first read for Celltex. Why? This is particularly important for Celltex because they have made such a point that they are strictly the producers of the stem cell product while other doctors and clinics are the users meaning the ones transplanting the product into the patients. Since the cells in tubes are handed off from one entity to another, the labeling of the tubes containing the cells is incredibly important.
Fortunately, tube labeling is easily solved. Some of the other issues may be far more challenging and expensive for Celltex to address to the FDA’s liking, but I hope they fully comply with the FDA’s instructions.
Dr. Turner is harsher in his view of Celltex and the meaning of this report.
Today he told me:
Since February, many individuals have expressed both concern and outrage in response to Celltex’s involvement in processing mesenchymal stem cells, banking stem cells, and making these cells accessible to individuals with such illnesses as multiple sclerosis and Parkinson’s disease. Among various issues raised by bioethicists, scientists, journalists, and other individuals who have investigated Celltex’s operation in Texas are fundamental questions about whether the stem cells Celltex provides to patients are safe and effective. Celltex has failed to make any attempt to demonstrate safety and efficacy and yet it charges patients with serious illnesses $20,000 to $30,000 for stem cell “infusions.” There is no reason to assume that these procedures are safe and effective. To the contrary, by providing local physicians with clinically unproven stem cell interventions Celltex is putting patients at risk of harm. From a regulatory perspective, all signs indicate that the stem cells processed and banked by Celltex are more than minimally manipulated and are therefore classified by federal regulations as biological drugs. This means that Celltex should have submitted an Investigational New Drug application to the FDA, provided FDA with extensive documentation in support of conducting trials involving human participants, and before proceeding confirmed that FDA did not place a clinical hold on their IND application. These steps are routine for credible scientists determined to establish whether stem cell interventions are safe and effective. Now, to familiar array of objections to Celltex’s business operation, FDA inspectors reveal a long list of egregious failures to engage in good manufacturing practices. Before today, discussion centered upon whether Celltex was violating federal regulations. Now that the FDA inspection report is available we should also ask why Celltex executives and employees in running their clinic appear to be violating both federal regulations and basic protections for patients.
I will find it fascinating to see what Governor Perry’s reaction is to this report. I myself met with Governor Perry in person two weeks ago today, a meeting I found productive and encouraging. I found Perry to be engaging and open to new ideas.
The meeting was stimulated in part by an open letter I wrote to the Governor on how I sincerely believed that he and Texas could be the leaders in stem cell research. One recommendation I made was for Perry to distance himself from Celltex. I’ll be curious to see if he does that in the aftermath of this report.
I still believe that Texas has a very bright future as a leader in stem cell research and clinical translation.
Interestingly, not even mentioned (yet) by the FDA in the report was the issue of whether the stem cells in the product made by Celltex are “more than minimally manipulated”, a crucial issue likely to come up in the coming months and years (for more background on the key issue see my previous post on minimal manipulation).
Why do I say “in the coming months and years”?
Because this is only the beginning of what is likely to be a long-term relationship between the FDA and Celltex.