The world of stem cell research and regenerative medicine is unpredictable but it’s fun to be a part of it and try to do predictions.
Each year in late December or early January I make prognostications for the coming year for stem cell research and regenerative medicine. I made 21 such predictions for the current year of 2021.
How’d I do?
Trying to be somewhat impartial, I give myself an A- overall so that’s solid.
Let’s go through the predictions and grades. I’ve included the predictions verbatim and my attempt now to grade them objectively.
For reference, you can see also how I fared in 2020.
Grades for my 2021 regenerative medicine predictions.
- More good news on gene-editing for Sickle Cell/Thalassemia front. Things continue to look encouraging with the ongoing clinical trial and there are other trials as well that bring more hope. Grade: A. It’s been an upbeat year for gene and cell therapy trials for sickle cell. The year didn’t get off to a great start with some Bluebird Bio trial participants reportedly having cancer (although it wasn’t clear if it was therapy-related), but they resumed their trial. With others like Vertex active in this space there is real hope for success in the coming years.
- CRISPR Therapeutics has a very good year. Related to the prediction above and additional work in the pipeline, this biotech has a positive 2021. Grade: C. It was a mixed year for the firm. While they got the okay to start a trial from Health Canada for VCTX210 developed with ViaCyte and biomedical science-wise things continued fine, the stock was bashed. No big good news for the year either.
- Stem cell & regenerative medicine biotech blues, maybe with a few exceptions. Unfortunately, overall, half or more of the top 10 or so biotech firms in the stem cell sector will have a net decrease in YTD share price at the end of 2021. I hope there are some notable exceptions to this of firms that do extremely well in the broader regenerative medicine space like CRISPR Therapeutics and other more focused cell medicine firms. Grade: A. While many regenerative medicine-related biotechs got a major COVID bounce at some point, in most cases that has faded, and only 4 out of 18 biotechs I check on have seen their shares go up in 2021. You can see 24 cell and gene therapy firms I follow in this area.
- Good news on the HSCT for MS and other autoimmune diseases front. While the prominent Burt-Northwestern HSCT program has remained shut down, other research on using hematopoietic stem cell transplantation for multiple sclerosis and other autoimmune diseases will continue looking very promising. Grade: A. Overall, it’s been an encouraging year on this front. While not yet approved in the U.S. by the FDA, this general approach in its many forms is gaining traction and can be given in Europe.
- The cell medicine for COVID-19 front doesn’t produce an FDA-approved treatment. It’s been an unpopular view, but I’ve never been very enthusiastic about the idea of stem cells for COVID-19. While the University of Miami 24-person trial enthusiastically reported some apparent effects a few days ago, there are quite a few caveats there so we need to see how a subsequent larger study goes by them as well as outcomes of dozens of other trials using cells for the novel coronavirus disease. I predict no fully FDA-approved cell therapy for COVID during 2021 and mostly discouraging results for the trials in this area overall. Grade: A. Other than a few small studies here and there, it doesn’t seem like a clearly encouraging avenue so far.
- But COVID cellular medicine continues as a hot area. Despite the first prediction above, we’ll continue to see new trials, probably new INDs, and a whole lot of exuberance about the idea of cellular medicines as a treatment for COVID-19, even from ISSCR. I hope I’m wrong with my skepticism on this. Grade: An “A”. This area is still generating a lot of hype. There’s more pressure here as pharma pills are showing efficacy against COVID, which is likely a farm simpler approach for most than almost any possible cell therapy.
- More CIRM clinical trials. CIRM funds at least 6 more new clinical trials in 2021. The new funding from Prop 14 California is a big boost, of course. CIRM also looks to be stepping up funding of more basic/translational research somewhat as well. Grade: A. By my count on the CIRM trial page there were 8 new trials listed as funded by CIRM in 2021 vs the total listed for 2020. Very impressive.
- An encouraging year for regenerative medicine related to vision loss. We see more concrete steps forward for stem cells and other cellular medicine approaches to vision loss including macular degeneration. Grade: B. It was a solid year but no major developments of the kind I expected.
- DIY human CRISPR will be back in the news. As the pandemic hopefully fades at least somewhat, we’ll see more attention given to CRISPR uses in people that defy norms. Hopefully not more CRISPR babies, but who knows. Grade A: Two teams in the U.S. filed for patents on heritable human CRISPR in 2021. I still get questions from people about CRISPR babies and rumors are circulating even these days after He Jiankui.
- State AG lawsuits vs. stem cell clinics expand. Good news as state-level efforts to reign in stem cell clinics continue and expand by at least 1 and probably 2 or more states taking concrete actions like lawsuits. Grade A: The Georgia AG joined the FTC to file suit against a stem cell clinic and in New York State the AG won a multi-million dollar settlement against a Manhattan stem cell clinic. I’m hearing rumblings of more possible AG activity.
- FDA still strongly engaged on unproven clinics. More good news as the FDA will start ramping up more actual warning (not just untitled) letters to unproven stem cell and regenerative clinics, including at least 3 new warning letters and probably more. There will be more untitled letters too. Grade: B. The FDA sent a warning letter to Surgenex in November 2021 and Utah Cord Bank in June so that makes 2 and I was close on this one. There have been several untitled letters too in 2021. I expected more. It seems the agency is waiting on a verdict from Judge Bernal here in California in the big stem cell clinic injunction suit before doing much more related to adipose cell clinics at least. Why not take more action in 2021 on the ubiquitous perinatal-related stem cell clinics and suppliers? I don’t know.
- Hahn is out and the new FDA Commissioner will not be Robert Califf, Scott Gottlieb, or David Kessler. Ned Sharpless and current CBER Director, Peter Marks, would be excellent. They would be somewhat unexpected picks because they are not politician-type people. Peggy Hamburg, past Commissioner under Obama-Biden, could be selected again. Another name being floated is current Principal Deputy Commissioner of Food and Drugs, Amy P. Abernethy, MD, PhD, who could be selected. As of January 12, Biden still does not have a pick announced as of the writing of this post, which could signal an unconventional pick or just that they are busy with other stuff. Grade B-: Hahn is out but I am probably wrong on part 2 as it looks like Califf probably will become the new Commissioner for a second time in his career.
- Post-pandemic stem cell clinic marketing. Some unproven suppliers and operations selling stem cell and regenerative offerings try to make up for lost profits by being even more egregious in their marketing and hype. It is also possible on the more positive side that some clinics will not recover as businesses to be active post-pandemic. Grade C: Ha, post-pandemic? What was I thinking? We aren’t post-pandemic yet so that was off base by me. Clinics remain very active though and seemingly more active than earlier in the pandemic.
- Production of gametes from stem cells in the news. More developments on making human eggs/sperm from stem cells. While this gives hope for possible future fertility treatments, it also raises some thorny ethical/societal issues. Grade A: It’s still a hot area. For example, Megan Molteni at STAT had this: Researchers create mouse eggs from stem cells in a test tube and Antonio Regalado over at Tech Review had this: How Silicon Valley hatched a plan to turn blood into human eggs. How could you ever know this was safe for reproduction before trying it with huge risk?
- Exosome troubles. More iffy exosome situations in 2021. This problem is not going away. Grade A: Patients have been increasingly asking me about exosomes from clinics. IV or intranasal use of exosomes could be very risky. There are no solid safety data.
- No luck for stem cell clinic US Stem Cell in its appeal. This clinic firm will be unsuccessful in its court appeal of the permanent injunction against it or the appeal will still be pending due to the pandemic before years end, leaving the injunction in place. Grade A: US Stem Cell lost its federal appeals case. What’s more, the appeals court seemed to be critical of the judge here in California in terms of his ruling denying summary judgment request by the FDA.
- As to the other federal fat stem cell case, I predict by year’s end it’ll be FDA 1, Cell Surgical Network 0. I expect that this stem cell clinic chain will lose in federal district court and then they will appeal the ruling. The appeal could take a long time. If I’m wrong about this prediction and CSN prevails, then the area of FDA regulation of cellular medicine and in particular of fat stem cells could get shaken up. It’s also possible the case could remain unresolved due to the pandemic. Grade: B. Judge Bernal is taking an extremely long time to deliberate before making his ruling in this case after a slow trial. I don’t know if that bodes any particular way for the FDA or the defendants. I may have been overly optimistic on this case.
- Japan has stem cell ups and downs. Stem cell clinical trials continue to build in Japan which is great, but the country also faces challenges on the regulatory front including from for-profit clinics. I also expect we’ll see more such up and down developments from Australia and Italy too. Grade A: Unfortunately, the number of stem cell clinics seems to be growing in Japan. The country’s more open stem cell oversight framework may be giving these firms the space they need to exploit patients.
- Young blood. This idea of using young people’s blood to help stem aging in older folks just won’t go away in 2021 and/or the FDA/FTC will have to take some major additional action in this area. Grade A: The hype over young blood infusions continues. Now we might have another offshoot of this: “Athletes blood”.
- PRP stays hot. Stem cell clinics and universities/large medical centers continue to market platelet rich plasma or PRP for a variety of conditions. Does the evidence back it up? We’ll see if there’s more data in 2021, but so far I’d say the evidence for most applications is not clear-cut. Some stem cell clinics will switch to mainly PRP as the regulatory window to use fat stem cells or SVF closes per the FDA enforcement period. Grade A: While there isn’t much more evidence as to PRP efficacy, it is being sold more than ever. It’s not really a drug product so I’m not sure about a role for the FDA but what the FTC? State medical boards?
- Perinatal products. Unfortunately, selling and using iffy birth-related “stem cell” products will continue to be hot (including via chiropractors) and spark more FDA action. Note that these products often are just useless, dead cellular debris. Grade A: This area is truly a hot mess. The FDA issued more letters to firms in this space, but should have done more. The number of locations selling perinatal products is like sand at the beach.
Stay tuned for my 2022 predictions.