Grading my 2022 regenerative medicine predictions

It’s that time of year again when I grade my previous year’s predictions for the stem cell & regenerative medicine space.

Overall, I did better than a typical year. I give the 2022 predictions an A- overall.

This exercise can be kind of fun but also humbling. For example, I tend to be too optimistic about how things will go on most things related to the FDA.

You can also check out my 2021 stem cell predictions before seeing my grading of this year’s prognostications. You can even check out how I did on my annual predictions from almost a decade ago in 2013. Reading those is like a time warp.

Stay tuned for my 2023 crystal ball reading.

Grading my regenerative medicine predictions for 2022.

For 2022 I divided the predictions into categories.

Below I list my predictions and my grades for how they fared.

COVID-19 and cell therapies

1. Cell therapy for COVID yields at best a mixed bag of results. Given the mostly weak study designs testing various cells (including especially MSCs) for COVID-19, I predict there will not be an FDA approval in this space in 2022. There may be some trial news that seems positive but on the whole there also won’t be a clear picture in this area.

In the meantime, the push to develop anti-viral drugs against COVID will continue, which may also generate a mixed bag of news but overall be more promising than cellular approaches.

Grade: A. There has been little concrete progress on the idea of cell therapies for COVID-19. Other non-cellular approaches continue to look much more promising. Last year my student Mina Kim and I published a paper showing that most cell therapy trials in this space aren’t designed to be up to the task of producing clear data.

Unproven cell “therapy” clinic predictions

2. FDA goes big(ger) on clinics. The FDA takes some kind of unprecedented action against unproven stem cell clinics this year. Such action could be 6 or more warning letters, or more than 2 new injunctions, or large-scale fines. The prediction is that something happens here where we go, “that’s new” or “that’s big!”

Grade: A. As an update, Jimmy Lee Taylor was sentenced to 202 years in prison in December so this prediction was correct.

3. First recent criminal charges for some kind of cell clinic-related firm. Related to the above prediction but more specifically, I think 2022 could be the first year in a long time that we see some kind of federal or state level criminal charges related to stem cells or cell clinics or suppliers. There have been past cases with criminal charges, but not for a long time and they generally did not seem very high impact. Some non-FDA compliant clinic-related firms (most likely in the troubled perinatal stem cell space) have been so reckless in recent years that charges could be appropriate for the most extreme cases. A lot of people have gotten hurt or ripped off.

Grade: C. There wasn’t anything majorly new here, but Patricia Derges was convicted of fraud in her stem cell case this year. State AG’s also took non-criminal actions. Fo instance, in Washington State the AG effectively shut down a stem cell clinic this year and won a large settlement.

4. U.S. vs. Cell Surgical Network court case finally ends but leads to an appeal. In this case the FDA is seeking a permanent injunction against a group of adipose cell clinics. The judge may issue a mixed verdict here in California, giving the defendants and the FDA each something. If Judge Bernal gives the defendants at least a partial victory then that could severely muddle things given the very different verdict in the US Stem Cell case in Florida federal district court. Plus, US Stem Cell lost its appeal of the verdict with a federal appeals court, which mentioned the California case in its ruling. This all sets the stage for an appeal of the California case.

Grade: A. The FDA lost the Cell Surgical Network lawsuit but appealed. I’m not sure why, but it took the Feds forever to even file the appeal too. Hopefully the Ninth Circuit Court will be logical here in ruling on this appeal.

5. Cryo-Cell doesn’t have a great year. This publicly-traded cord cell banking firm (CCEL) is trying to also become a cell therapy firm. The problem in my view is that they are doing this on a foundation of an unproven cord product via a mega-deal with a Duke pediatric neurological cord cell therapy team. While the FDA may let them open their planned unproven cell clinic based on compassionate use, there’s a real chance that it may never come to fruition. I predict the stock will be down for 2022 vs. the close of 2021, and/or there are other negative developments for Cryo-Cell or their partners the Duke team.

Note that what they are doing already via compassionate use and the planned clinic looks inherently different at a regulatory level than what we typically think of for unproven clinics as Duke has FDA clearance for the compassionate use, but I believe at the other level of biomedical science this is still very much unproven.

Grade: A. It was an awful year for Cryo-cell. Their stock collapsed. I can’t find a glimmer of good news for them in 2022. Their Medical Director, Joanne Kurtzberg, even said in a recent interview that private, for-profit cord banking “isn’t necessary.” That was a stunner.  You can see that the company’s stock dropped more than 60% YTD by mid-December above.

Human embryos and embryo models in regenerative medicine

6. Human embryo models generate more buzz. Models of human embryos, sometimes called blastoids, are going to get a lot of attention in 2022. You could see the buzz building in 2020-2021.

As I’ve asked before, at some point will these embryo models become too close to the real thing? How will we know?

Grade: A. These models are still a hot topic. I wrote in August about the Jacob Hanna startup Renewal Bio that apparently intends to grow up human fetus-like models and harvest their organs for clinical use. What could go wrong there?

7. Someone crosses the 14-day rule on human embryos. Now that ISSCR has backed away from a firm 14-day rule (cutoff point) on growing human embryos in the lab, someone is likely to grow human embryos beyond 14 days and probably too far in 2022. By too far, I mean they generate problems or bad press. I can guess who it might be but we’ll see. Long-term growth of mouse embryos in the lab in 2021 paves the way for attempts with human embryos too this year.

One challenge with this prediction is that what happens in 2022 might not come to light until 2023.

Grade: A-. As I suggested in the prediction, my best sense is that this did happen in 2022 but it hasn’t been publicly disclosed yet. Stay tuned. It may be Jacob Hanna. 

8. Human embryo editing. There is more talk of the idea of reproductive CRISPR in humans even as there are more reports on in vitro studies that suggest concerning risks like chromosomal damage. It’s hard to say if we’ll learn any more about the three children reportedly generated from the He Jiankui misguided gene-editing of human embryos.

US fears CRISPR baby Whoopsie.

Verve discloses FDA put 'hold' on US trial of its base editor for ⏬ cholesterol pending additional preclinical data.

Regulator asked about risk of "germline" editing and wants more 'contraceptive measures' in trial
https://t.co/57Akko3eay https://t.co/U4E3KSl3m1 pic.twitter.com/c4Nfiuyi7X

— Antonio Regalado (@antonioregalado) December 12, 2022

Grade: A. He Jiankui is back in the research lab, according to his blog. There’s also the possibility that some biotech effort will accidentally lead to a CRISPR baby being born when targeting a specific organ for CRISPR, like the liver, and the delivery mechanism gets it into gametes. Thankfully the FDA seems to be cautious about this possibility. See the Tweet above from Antonio Regalado. 

Biotech, Cell and Gene Therapies

9. Combo cell and gene therapy efforts continue to give real hope and some concrete results. When the power of cell therapies and genetic technologies are combined, some of the resulting possible treatments are very impressive such as certain CAR-T therapies. 2022 will bring more good news in this space.

Grade: A. This is one of the hottest areas in the regenerative medicine space. I’m excited about its potential. 

10. But related to the above at least one safety concern makes fairly big news. Safety is an important issue in this arena. I’m hoping we don’t have any complications on the safety front this year, but I think most likely there will be at least one such story that arises.

Grade: A. Unfortunately, this was an accurate prediction on several fronts. Here are two of the reports:

• 2 deaths after Novartis’ Zolgensma put gene therapy’s liver safety in the spotlight once again, Fierce Pharma.
• Death in CRISPR gene therapy study sparks search for answers, AP. It’s still unknown if this death had anything to do with the gene-editing drug.

11. Cells for diabetes continues to generate headlines. Vertex got front-page NY Times coverage for a short-term cell infusion result with one diabetes patient with the word “cure” present in the headline as a question. Talk about jumping the gun. Still it was a bit of encouraging early news and I expect this firm, ViaCyte, or others in this space to make some more news, hopefully good news. One challenge for ViaCyte is that based on their recent published data their capsule device has a heterogenous mix of pancreatic cells and not just beta cells, but that could be a plus too if it functions overall in a more natural way or lasts longer. Vertex needs to get some progress on its own capsule approach as I don’t see the infusion route as viable in the long run.

Grade: A. It was another big news year here. Vertex acquired ViaCyte. For years I predicted that ViaCyte would either be acquired or do an IPO, probably the former. Of course, it finally happened in the year that I didn’t specifically predict it.

12. Another rough year for regenerative medicine biotech overall. Of the publicly-traded cell therapy biotech firms I’ve been following, over half end 2022 down in terms of stock prices. I hope I’m wrong on this.

Grade: A. Scanning through the panel of two dozen stem cell stocks that I follow, almost every one had a bad year. It’s so discouraging. Regeneron and Capricor are exceptions as their PPS are up.

13. RMATs. We see at least 10 more of these still relatively new FDA designations being granted. RMAT stands for regenerative medicine advanced therapy designation. Take a look at my current RMAT list.

Grade: A. There were 54 RMATs in my late 2021 list. Now in 2022 there are 70 in the public domain, an increase of 16 over last year. I’ll write later how certain sectors have gotten many RMATs.

See my updated RMAT list.

Tech advances including RNA drugs

14. RNA drugs are a hot topic. What with the major success of RNA vaccines for COVID, we’ll see more attention given to the potential of RNA as a basis for medicines. This focus will include more RNA vaccines beyond those for COVID and other applications of RNA as a type of medicine.

Grade: A. RNA as a drug are still hot in 2022. The notion has been around for a long time, but as I said in my prediction, the COVID pandemic has brought more attention to potential RNA therapies.

15. Lab-grown meat. Meat-like products grown in the lab overall continue as a hot area. Such fake or “clean” meat as they are sometimes called can be grown from different kinds of cells, including sometimes animal stem cells.

Grade: A. It was another big year for clean meat research and news. I’m hoping there won’t be some kind of major contamination issue or some other major bad news to derail the momentum. Can you imagine if in 10-20 years a large segment of the population is eating mostly “meat” grown in a lab?

16. Stem cell-derived sperm and eggs. There continues to be more research and discussion about making reproductive cells in the lab from stem cells for potential use in human reproduction. It’s a super risky area.

Grade: A. This area continues to push forward and make headlines. We saw rat sperm made from stem cells and many other developments. There is enthusiasm for trying some of this eventually in people. This all resonates with the stem cell-derived embryo model work too.