The last few years in the stem cell and regenerative medicine arena have been complicated in many ways. Variables like surging stem cell clinics and COVID have made predictions harder. Even so, trying to make predictions is useful and it’s fun to go over past year’s predictions.
As I do each year, I’m now making predictions in this field for the new year of 2022. This year I’ve divided the predictions into categories.
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.
Unproven cell “therapy” clinic predictions
2. FDA goes big(ger) on clinics. The FDA takes some kind of unprecedented action against unproven stem cells 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!”
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.
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.
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.
Human embryos and embryo models
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?
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.
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.
I could add another prediction in this section that organoid research continues to be hot, but that has been true for years now and I don’t see it cooling off any time soon.
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.
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.
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.
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.
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.
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.
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.
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.