The FDA and its CBER branch have been doing a good job overall in the last 3+ years to tackle the unproven stem cell clinic problem, but lately on the stem cells for COVID-19 front there is reason for real concern.
I believe the agency is taking on major risk overall by clearing a vast number of cell therapy trials for COVID-19 mostly without much data, but the clearance of a few programs by for-profit stem cell clinics is especially problematic in my view.
FDA on the ball more generally on clinics
For background and on the positive, the FDA is taking the problem of the stem cell clinic industry very seriously and taking action on clinic firms that are injecting patients with unapproved stem cell drugs.
It has sent off dozens of untitled and warning letters to clinic firms pitching unproven stem cells and other regenerative biologics like exosomes. The agency’s biologics branch called CBER is really on the ball on this these days. Its Director Peter Marks deserves big kudos for the serious commitment and marathon work here over many years to make things happen on this problem. Dr. Marks was the subject of an interesting piece in the WaPo by Laurie McGinley recently: Meet the most important federal official you probably don’t know — the man who holds the fate of the coronavirus vaccine in his hands.
Beyond the letters, the agency has done more on the clinic industry as well, especially as compared to during the Obama years. For instance, the FDA has also filed suit against two firms selling unapproved fat stem cells, one in California and one in Florida, and prevailed in the latter case already getting a permanent injunction. The California federal district court case is still pending. It is a major undertaking for the FDA to file suit via the DOJ so these suits are a big deal in a positive way.
Clinic firm Giostar’s compassionate use clearance from FDA
However, it’s more of a mixed bag when it comes to COVID-19. While the agency has rightly taken some firms to task for unsupported claims about treating COVID-19 with stem cells, it has also green lighted others to inject patients.
One of the most concerning stem cells for COVID-19 clearance I’ve seen reported so far involves Giostar. This headline and article from a media firm called India West about GIOSTAR, “Indian American-founded GIOSTAR Announces FDA Approval for COVID-19 Clinical Trial with Stem Cells” raises some red flags.
What’s the problem?
To start with, in my opinion GIOSTAR fits the bill of being an unproven stem cell clinic chain. It has been marketing unproven stem cell injections for years around the world for a variety of health conditions.
The firm makes some highly questionable claims to biomedical legitimacy, such as this on the Chicago clinic’s website:
“GIOSTAR is comprised of a world-renowned international group of researchers, doctors, and scientists in the field of stem cell biology, cancer, and gene therapy. The team has won several awards, conducted pioneering studies, and published extensive research in leading medical journals. Further, the GIOSTAR team has helped develop stem cell research programs for prestigious institutions in the United States, such as the University of California San Diego (UCSD), Salk Research Institute in San Diego, University of California Irvine (UCI), Burnham Institute of Medical Research in San Diego, and the University of California Los Angeles (UCLA).”
But is this accurate?
Past problematic claims from Giostar
Michael Hiltzik over at the LA Times caught the firm on some of these claims just last year in 2019. Here’s one particularly striking passage about the UCLA-related and other academic claims of GIOSTAR leader Anand Srivastava (pictured above; note that Patel here refers to Giostar Chief Executive Deven Patel):
“Now let’s turn to Srivastava’s credentials, which are the bedrock of Giostar’s assertion of stem-cell treatment leadership. Patel acknowledged in an email that some of Srivastava’s descriptions of his prior academic appointments have been exaggerated. They include his claims on the company website to have been an associate professor in the department of cellular and molecular biology at UCLA’s medical school, an associate professor at UC Irvine medical school, and an assistant professor at UC San Diego medical school.
According to the schools, Srivastava held none of those posts, which almost certainly would have been tenure-track appointments. UCLA says its medical school does not have a department of cellular and molecular biology.”
To me that kind of stuff does not build trust in the firm, yet it does appear the FDA cleared their COVID-19 work in some way. Does the company has some compelling data that convinced the FDA? As the West India article stated, does the firm have an “approved clinical trial”? It doesn’t quite seem like a trial.
How much data should be required?
The India West article provides some details on the compassionate use clearance:
“San Diego, California-based Global Institute of Stem Cell Therapy and Research, a leader in stem cell research, has announced that they have received an approval for a COVID-19 clinical trial, led by their Indian American medical director Dr. Prabhat Soni. GIOSTAR will conduct the trial using stem cells to treat COVID-19 patients under the approval of the United States Food and Drug Administration “expanded access for compassionate use” program, said a PRNewswire report. The institute is exploring a promising alternative approach to the devastating disorder, which leverages the anti-inflammatory properties of mesenchymal stem cells (MSCs). According to Soni, the investigation is based upon two decades of stem cell research by GIOSTAR’s Indian American co-founder, chairman and chief scientific officer Dr. Anand Srivastava.”
It’s not clear to me how much rigorous, relevant data they have.
More generally, how much data and of what quality are needed for clearance to infuse patients with stem cells for COVID-19 these days?
It seems like things are very different now than in the past for non-COVID-related health conditions. For example, for other kinds of cell therapy efforts the FDA might require rigorous, directly relevant pre-clinical data collected over a few years. A pandemic may justify speeding things up via a lower data requirement, but how low is OK?
The FDA recently cleared an IND by the long-time stem cell clinic firm Celltex in Texas for fat MSCs for COVID-19. Celltex has a long history with the FDA. Years ago it moved its clinic doing stem cell injections down to Mexico. You can see a YouTube video of a Celltex TV commercial above.
There seems to be a focus in the new Celltex COVID-19 program at least in part on using stem cells as a preventative measure for COVID-19, which in my view is even less likely to be proven helpful than employing the cells for treatment. Routine injections of stem cells for purported immune modulation for COVID-19 protection or other reasons could end up being very profitable though.
Politics is clearly at play in the agency’s evaluation of cell therapies for COVID-19. It feels like the Trump administration and its allies have pressured the FDA to move as many COVID efforts forward as possible.
Celltex customer, former board member, and long-time advocate, Rick Perry, recently went on Fox News to plug the clinic firm. Perry was also the DOE Secretary for a time for the current administration. Earlier, Trump attorney Rudy Giuliani hosted Celularity CEO Bob Hariri on his show, which felt to me somewhat like an infomercial. Both Celltex and Celularity got their IND clearances from the FDA. How much of a role did politics play in these cases?
Can we expect the FDA to continue clearing so many applications for experimental cell therapies for COVID studies including by other stem cell clinics and related regenerative firms?
Now apparently under pressure from Trump, Hahn just approved an EUA for convalescent plasma for COVID-19 after earlier the agency seemed to indicate it wasn’t that upbeat about this approach. Check out this new, frank conversation between STAT’s Adam Feuerstein and Matt Herper on Hahn’s approval of convalescent plasma and politics at the FDA.
Overall, I see major risk from the FDA letting so many stem cell-related trials and compassionate use programs to proceed including especially by clinics. In the bigger picture, the perception that politics is now potentially playing a sizable role in FDA decision making also risks undermining faith in their decisions. That trust is particularly important during the COVID-19 pandemic with potential new vaccines being considered in the next few months.