Toward the end of each year, I make stem cell & regenerative medicine predictions for the coming year. It’s a fun tradition here on The Niche that goes way back. The stem cell field has changed dramatically as reflected in each year’s predictions. Here’s a list of past predictions with my grades of how they did: 2022, 2021, 2020, 2019, 2018, 2017, 2016, 2015, 2014, 2013, 2012, and 2011.
To make it more fun this year I’m going to include some riskier predictions. These may be less likely to be accurate, but they’re more interesting and potentially impactful.
FDA & stem cell clinic predictions
1. The FDA wins its unproven stem cell clinic appeal of the verdict in the Cell Surgical Network lawsuit. As many of you know, the FDA lost the big lawsuit against the adipose stem cell clinic chain Cell Surgical Network in 2022. They appealed to the Ninth Circuit Court, which I’m thinking is most likely to side with the FDA. In that scenario, there would be an injunction on the clinic chain. Further, more broadly adipose cell preps like SVF would be solidly defined as drugs requiring premarket approval. On the other hand, if I’m wrong and the stem cell clinic chain prevails again in the appeal, that will turn clinic oversight on its head.
2. Anemic clinic oversight continues at the agency overall. Despite the likely win in that appeal mentioned above, the FDA still does surprisingly little about the overall unproven stem cell clinic problem in 2023. I hope I’m totally wrong on this. However, so far it seems one might not be able to underestimate how little the FDA will do in any given year on clinics. Tens of thousands of Americans are at risk each year from these unapproved drugs. It was disappointing to see this recent, very long interview with Commissioner Califf on goals looking ahead without one word about the massive unproven stem cell clinic problem. Is it no longer a priority?
Where the FDA is most active in 2023 is in the perinatal space, which has been its focus of oversight space for the last few years. We could see a few more warning letters there.
3. Unproven exosomes. Unproven exosomes make more big news. Regenerative clinics and suppliers will continue to sell iffy exosomes. Will the FDA do more on exosomes? Who knows?
4. ExoFlo, et al. Speaking of exosomes, there will be some development on the firm Direct Biologics and its ExoFlo product. I’m still unsure how they got to an FDA-cleared Phase III trial. Tons of great unpublished data?
Another exosome drug manufacturer, Kimera Labs, could make news too somehow, whether good or bad I don’t know. They’ve been seeking an IND on exosomes for a long time. We may also see the lawsuits between various exosome firms make news.
Embryos and stem cell models
5. Crossing the line with human embryos. Someone grows human embryos past the previous 14-day rule limit. This could be Jacob Hanna and his firm Renewal Bio. It’s a very risky idea to go much further than two weeks. A variety of research including work in Hanna’s lab growing mouse embryos for long periods in the lab has set some of the foundation for trying the same with human embryos.
6. Embryo models. Stem cell-derived human embryo model research continues to be hot. The long-term culture of realistic human embryo models could cause controversy too. It’s also possible on that last point that human embryo models that are too similar to the real thing could be grown for far too long by someone.
Why do these predictions #5-6 matter in terms of predicted missteps here? One of the big risks is that images of human fetus-like structures will be used against the stem cell field more generally, hurting even non-controversial research in a big way.
CRISPR & gene therapy
7. CAR-T continues good news. The CAR-T field has revolutionized outcomes from some cancer patients. I see the positive trend continuing. It’s one of the most exciting areas in the field. One interesting side note here is that the FDA seems to consider CAR-T cells to be regenerative medicine but most scientists don’t seem to see it that way.
8. More good news here as the gene-editing clinical work on sickle cell disease continues to be promising. It’s been so exciting to watch this work unfold over the years.
9. He Jiankui in the news. Will He Jiankui stay out of trouble? It’s perhaps 50-50 on that in coming years. We might get the first substantive update on the health or genomes of the two-to-three CRISPR babies. His news in 2022 of getting back into science wasn’t entirely reassuring. Expect more on him and probably from him in 2023 as he tries to bounce back.
10. Is someone else going germline? At least one researcher publicly advocates germline human gene editing. It could be more than advocating. We might hear about plans to “do it the right way.” I’m not sure what that would be mean. There are almost unresolvable safety and practical concerns.
11. Cell and/or gene therapy safety concern. At least one or more safety issues come up again in the gene therapy arena like last year. It’s almost inevitable in a rapidly expanded area of clinical research to have setbacks. We need transparency on both positive and negative developments.
12. Another rough year for most regenerative biotechs. Which firms will continue to struggle? Athersys seems on the edge as its stock was down 97% in 2022 as of the writing of this post. Mesoblast also urgently needs some good news, possibly from the FDA on GvHD. The FDA doesn’t seem inclined that way so far though. Most likely 2023 will be another bad year for Athersys, Mesoblast, and BrainStorm Therapeutics.
This is another one of those predictions where I hope I’m wrong. Many biotechs in this space got a major COVID bounce in the middle of the worst of the pandemic, but that has largely given way to big losses.
At the same time, in 2023 there will be some good news for a few specific biotechs. For example, I think CRISPR Therapeutics will end 2023 higher than its value now on December 27, 2022.
You can see the 24 stem cell stocks that I follow. I may update that list. I don’t currently directly invest in any biotechs and this post is not financial advice.
13. More RMATs. The number of FDA RMATs goes up at least 10% more. It’s hard to tell so far if anything has come of all these RMATs. Too early to say? What do you think?
14. Vertex/diabetes. Vertex has made big headlines and gotten some very favorable diabetes-related coverage including from the NYT. It recently acquired two major competitors, Semma and ViaCyte, leaving it in the driver’s seat on cell therapies for diabetes. Often though after a big firm acquires smaller ones, there is a period of transition. During those periods not much happens related to the acquired firms’ pipelines. I hope with Vertex that we don’t see what happened after Atellas bought ACT/Ocata, which was basically nothing for many years. Leaning positive, I think we’ll hear some concrete plans and news from Vertex in this space in 2023 related to diabetes clinical trial work. More broadly, Vertex seems like a strong biotech with a proven track record.
15. Cord blood banking/therapy downturn continues. Firms like Cryo-Cell and ViaCord don’t have a good 2023. This industry continues to struggle more generally as it looks for new ways to make money. What about MSC and tissue banking? Overall, I believe public cord blood banks are the best way to go and have the most benefit to society. I would be very surprised if the FDA allowed Cryo-Cell to open its planned infusion clinic in 2023. The clinic would infuse kids with unproven cord cells and charge for it. On the other hand, in the past, the FDA seemed to have a puzzling soft spot for the Duke Autism Center, which has a deal with Cryo-Cell, and continues to charge for unproven cord blood cell infusions for autism and other conditions under expanded access.
16. The idea of stem cells or other cells for COVID still mostly languishes. Other approaches besides cell therapies continue to seem more promising in 2023. There may be some small studies arguing for benefits of cell therapies, but they are unlikely to be convincing.
17. Organoids. More interesting developments on organoids. We’ve already seen some brain ones that sprout early eye-like structures, venom-producing organoids, teary organoids, CSF-producing organoids, and more.
18. De-extinction is hairy even in the planning stages. The efforts to bring back woolly mammoths generate more tough questions. Those pushing it don’t have good answers. In late 2022 an article on headaches that the de-extinction fans are facing even now made some good points. On the potential complicated issue of ivory and cloned woolly mammoths, one idea was to make tuskless mammoths. That seems like an awful idea. What’s next, CRISPR-made bald mammoths so they can deal with climate change better? I can’t help but feel like the de-extinction efforts here are more about human ambitions than positive goals.
19. Human cloning is in the news. In late 2022, a myth started floating around that covid tests will be used to clone people, which is total baloney. Human cloning will be in the news in 2023.
Changes and setbacks
20. Tessier-Lavigne is no longer Stanford President by the end of 2023. Many of his past publications have received close attention and have issues. It’s unclear if he had any meaningful direct role in any of this, but it’s likely even so that he won’t stay on at Stanford.
21. Mixed news on iPSC trials in Japan. It’ll be a mixed bag. For example, I think some trials will have positive preliminary news but the government will decrease funding moving forward. Overall, I’m still very upbeat about these trials in Japan.
22. Elon Musk’s Neuralink has its brain frozen. There are clear signs that all is not well at Neuralink. Maybe Musk’s own personal neural network (brain) is spread too thin what with all his brainpower going to flailing at Twitter. Tesla stock is suffering. Neuralink has been hyped for sure. I don’t see strong neuroscience leadership there. I’m going to predict that Neuralink will get a warning letter from the FDA or be effectively stopped in its tracks in 2023. There’s major bad news of some kind.
23. FDA leadership change. A major leader at the agency leaves.