The past year was all over the place for the stem cell and regenerative medicine field. As you can see in my scoring of my 21 predictions for 2021, quite a few things were surprises in terms of what did and didn’t happen.
Here’s an overview of the top stories of the year in our field.
COVID-19 and cell therapies: no there there yet
The pandemic is now far worse than most of us imagined a year ago. The next month or two are going to be awful as the omicron variant spreads quickly. How many of us, even with vaccination, will avoid catching it?
Throughout much of the pandemic some have held out hope that cellular therapies would be effective treatments for COVID-19. One big story of 2021 in our field is that there was no breakout clinical trial result on cells for the coronavirus.
I believe the idea was oversold, but there remains a chance that future clinical trial results could be solidly encouraging. However, with anti-viral pills coming onto the scene it seems to me that cellular therapies for COVID-19 face even higher hurdles for rationales and results, while potentially posing unique risks to patients. What risks? The main one is that cell infusions could do harm by overshooting and suppressing immune responses too much.
A broader challenge is that while anti-viral drugs target viruses specifically, infusions of cellular drugs such as MSCs may have broad effects all over the body and not all of them desired. Potential cell therapies for COVID also have to do better than steroids, which are now widely used for patients.
My student Mina Kim and I published a piece analyzing the potential impact of cell therapy trials for COVID-19 around the world and the results were discouraging over all. Almost all of these trials are not designed or powered to measure efficacy.
Stem cells for diabetes moving forward
Another big story of 2021 has been research on potential stem cell-based therapies for type I diabetes. Fortunately, this is more upbeat.
Vertex got a shot of good PR on its N=1 story of a patient infused with beta cells made from hESCs. His regulation of blood sugar was greatly improved for a time.
Talk of this being a cure was overblown, but at least this one patient did substantially improve for a time and that’s something.
With Vertex, ViaCyte, and others working on cell therapies for diabetes, I believe there’s real hope in the long run here.
Human embryos and embryo models in the lab
Research on organoids remained a hot topic in 2021. However, when it comes to growing 3D tissues from stem cells in the lab what has stirred the most discussion is related to human embryos. There are two fronts here.
Researchers are both growing actual human embryos in the lab as well as models of human embryos (sometimes called blastoids) that are now much closer to the real thing than ever before. This research raises some tough questions.
Eventually how will we know if the models are so good that they are essentially the real thing? If the models become the real thing then new ethical considerations kick in but may have already been violated.
With the ISSCR backing away from the 14-day rule on growing real human embryos in the lab, how will the field avoid someone recklessly or accidentally growing human embryos too long in the lab? New technology for growing mouse embryos much longer in the lab likely could be adapted for human embryos.
There are some useful things to be learned from carefully conducted research in this area, but it’s a risky area from an ethics and policy viewpoint. Missteps could easily spur harmful government interventions.
A tale of two federal lawsuits vs. stem cell clinics
A couple of years back the FDA filed suit against two stem cell clinic firms on opposite coasts that use adipose cell preps. There were significant developments in these court cases in 2021.
US Stem Cell
On the positive side, Florida-based US Stem Cell Clinic lost its appeal of the permanent injunction verdict against it.
A federal appeals court not only ruled for the FDA but also commented in their ruling on the other big case, which is ongoing here in California.
Cell Surgical Network
The federal district court case here in my state is against the stem cell clinic chain Cell Surgical Network, et al. We might call it the case that never ends given how many times it has been delayed by COVID and the judge, Jesus Bernal, still has not reached a final verdict months after the trial ended as of the writing of this post.
This thing is going to end eventually and probably sometime in January 2022, but it’s anybody’s guess what Bernal will decide. Whatever the decision it will likely be appealed.
Getting back to the federal appeals court and their ruling on the US Stem Cell appeal, they seemed to me to disagree with aspects of Bernal’s earlier decision against the FDA’s request for summary judgment in the California case.
More specifically, their statements seemed to argue that adipose cellular preps often used by clinics are in fact different from endogenous adipose tissue and so are drugs, an issue I see as at the heart of both cases.
Little FDA action on stem cell clinics after grace period ended
Whether you agreed with the FDA or not in its three-plus-year grace period for stem cell clinics and other sponsors to come into compliance on its regulations, you might have expected that after the so-called “period of enforcement discretion” ended in May 2021 that the FDA would do something new about those remaining non-compliant firms.
Maybe even just do something on a few of the most egregious ones?
What’s the point of even saying there’s a finite grace period to become compliant if after it ends nothing new happens for continued clinic offenders?
I’d guess and hope that the wheels are turning on some bigger actions behind the scenes that we just don’t know about yet, but we’ll see.
Some of us have speculated that the agency is waiting on Judge Bernal’s ruling here in California before taking any major steps. However, keep in mind that Bernal’s ruling may only have direct relevance for firms selling adipose cell products and there are hundreds or maybe even more than a thousand clinics that use other non-FDA-approved products. At least hundreds of those are both non-compliant and unlikely to be directly impacted by Bernal’s ruling, even if he does rule in favor of the defendants.
The FDA needs to be far more active moving forward on the noncompliant firms within the clinic industry overall and time is not on their side.
Another kind of unproven clinic? Duke & Cryo-cell risky plan
The push for an infusion clinic using an unproven cord cell product by cord banking firm Cryo-Cell via its partnership with a Duke pediatric cell therapy team has stirred controversy in 2021.
I wrote about it extensively here on The Niche and check out this helpful piece at Vice as well on this.
The FDA has been mum about this situation, but as I’ve written if the agency allows this Cryo-cell clinic to open it will set a bad precedent.
Who needs strong, consistent data to open a for-profit clinic according to the FDA? Apparently not everyone if this clinic does indeed open in the coming year or two. In my view Duke and Cryo-Cell just don’t have the data to back it up and it’s not even a close call.
What amps up the risks here is that the apparent for-profit clinic in question would focus on customers who are vulnerable children. Some unproven stem cell clinics both in the US and elsewhere also sell cord cells for autism and other pediatric conditions so there can be reverberations between the Duke/Cryo-cell efforts and these other firms, which is another risk.
The bigger picture issue is the extent to which the bedrock clinical trial process can be sidestepped to sell unproven cell therapies at a profit.
I have a feeling this issue is going to be a hot topic in 2022.
Stay tuned for my overall predictions for our arena for the coming year.