Last year around this time I took my annual plunge to make 20 predictions for the coming year for the stem cell and regenerative medicine field. I even made a crystal ball graphic to highlight the complexity of the stem cell ecosystem into which these predictions were embedded (see image) for 2019.
How’d I do?
Below I grade my 20 predictions for this year. You can in addition see my grading of my 2018 predictions here.
In the next week or so I’ll also do my 20 predictions for 2020. So stay tuned for that.
For 2019, I give myself a B+ on my predictions.
Here’s my final detailed report card for these 2019 predictions. Note the predictions are verbatim from when I made them and the grading is color-coded.
- Organoids. The upper size limit of organoids such as brain organoids will continue to grow as vascularization and other related work moves forward. This advance will enable more cool research, but keep raising tough ethical questions too related to this so-called human “mini brains”. The recent report of fetal human brain wave-like activity in organoids is relevant here. Final grade: Correct. There has been more reporting of “brain-like activity” in organoids. Are these neural activities just “noise” that is being over-interpreted or something meaningfully similar to what goes on in the human brain?
- Parkinson’s Disease. More concrete reasons for hope on clinical stem cell clinical for Parkinson’s Disease. The news of the 1st patient transplanted in Japan via an IPS cell-based product in 2018 is encouraging. Not a prediction exactly, but I’m hoping to see more IPS cell good news, possibly on PD, in the US too. Final grade: Correct. The clinical trials in Japan continue and work in the US is progressing including big advances by BlueRock Therapeutics (acquired by Bayer) and Aspen Biosciences getting more funding.
- Two biotechs on the move. Some major twist for the stem cell biotech ViaCyte including not just science-wise, but also or instead potentially talk of an IPO or an acquisition. Most likely this is good news. I hope! A second biotech, Athersys, has some much-needed good news. Further, unfortunately, on the whole the stem cell biotech arena doesn’t do great in 2019. Final grade: Incorrect. No real huge development for ViaCyte yet in 2019 and Athersys has been relatively quiet even as its stock continues a long slide.
- Trump-Pence. The Trump administration (including most likely VP Pence) at least talks about restrictions on embryonic stem cell research. I hope this doesn’t get ugly. Final grade: Fortunately, incorrect.
- CIRM. A formal announcement of either a 2020 state proposition for more funding for the California Stem Cell Agency, CIRM, or a major private philanthropy plan. Final grade: Correct. Notably, there’s even been some disagreement amongst different stakeholders about what form the new proposition should take.
- CRISPR babies A. Two opposing developments. Supposed claims of independent confirmation that the CRISPR baby guy He Jiankui did make mutations in human babies (e.g. an independent lab such as George Church’s analyzes the sequencing data), but probably also news that He didn’t actually make the mutations he said in the twins. Confusing, right? Unclear if anyone including the Chinese government will have access to independently (and ethically) sample the twins and parents for their own analysis of the data for real confirmation. Final grade: Incorrect. It’s odd that we’ve heard basically nothing one way or another.
- CRISPR babies B.The Chinese government will announce some kind of punitive action against He Jiankui. Could it end up being too harsh or just largely symbolic? Final grade: Correct. China announces prison terms for He and 2 others.
- The FTC will take some further action on one or more stem cell firms in the US. Final grade: Correct. Note that the FTC returned half a million USD from a clinic firm to customers in April.
- The FDA will issue at least 3 warning letters to stem cell businesses in 2019. It’ll do more on other stem cell fronts too I’m guessing. Final grade: Correct. See letters to Stemgenex, Liveyon, Stemell, and dozens of untitled letters went out too.
- State Medical Boards. One or more state medical boards will make news on action on stem cell clinics (I’m hoping including here in my state of California). The recent news of a California Medical Board task force makes this more likely. Final grade: Correct. I’m working on an update on this front for earl 2020, but one example of new medical board action is from Missouri.
- State Attorney Generals. At least one state Attorney General office will take legal action on stem cell clinics. It could even be two. There may also be local law enforcement action on stem cell clinics. Final grade: Correct. Note the lawsuit by the NY State AG.
- State law. At least one more state will have a bill emerge related to stem cell clinics, possibly Florida again. Final grade: Correct. Vermont is heading toward a new bill.
- Two big federal court cases. The FDA will win the initial rulings in its lawsuits against U.S. Stem Cell (USRM) and Cell Surgical Network/California Stem Cell Treatment Center. It’s possible that both the cases won’t be entirely resolved by year’s end though, pending appeals. Final grade: Mostly correct as FDA won against USRM, but case on Cell Surgical Network is still plodding along in court. In my opinion the US will most likely prevail here in California as well.
- U.S. Stem Cell metamorphosis. Despite the appeals in the suits mentioned above in prediction 13, USRM will undergo a dramatic change most likely involving either no longer being a U.S. publicly traded stock/FEC action, moving a sizable chunk of its focus overseas (maybe mostly in the Middle East), or shifting away from fat products. I’m not formally predicting this, but it’s also possible that in the next 12-24 months USRM may no longer exist. Final grade: Correct. No more SVF marketing or confirmed injections that I know of. There have been indications of maybe doing other stuff like bone marrow.
- Cell Surgical Network 2019 predictions. This clinic chain will exhibit shrinkage of its affiliates or other related negative news. They could also move a chunk of their work overseas. Final grade: Probably correct. Obtaining concrete new numbers can be a challenge. However, evidence of possible shrinkage (network clinics stopping stem cell offerings or websites becoming inactive) was apparent in the data in my recent Regenerative Medicine paper tracking the 2015-2016 stem cell clinic cohort over 3 years up until early 2019.
- Stemgenex. The proposed class action suit against Stemgenex either gets its class status or continues to heat up. Final grade: Correct. Class status was granted and Stemgenex is in bankruptcy.
- Bad actor chiropractors. At least one and probably more chiropractors find themselves on the hot seat because of their stem cell business, potentially facing criminal action or temporary loss of their licenses. Final grade: Correct. The Ohio Chiropractic Board has reportedly started cracking down on chiropractors doing stem cell injections and I’ve heard similar actions are pending in other states.
- Birth-related “stem cell” products & clinics take a hit. The unproven birth-related (amniotic, placental, umbilical, Wharton’s jelly) unproven clinic and supplier arena, intimately tied to chiropractor firms, finds itself in more hot water. I don’t think it’s a good year for supplier Liveyon. Final grade: Correct. Look at all the recent Liveyon news for instance on a warning letter and it has suspended sale of its product according to the WaPo. Other firms have been subject to action as well. I believe even more action is coming too.
- Exosome hype. More clinics sell sketchy unproven, non-FDA approved exosome therapies. Final grade: Correct. There also have been some undefined harms from exosomes in Nebraska. I’m hoping to have more details on this soon.
- Multiple sclerosis (MS). Ending on a more positive note, more encouraging news on the legit front of stem cells for multiple sclerosis (MS) most likely via HSCT combined with some kind of immunoablation. Many of us are eagerly awaiting a new paper on this front in 2019. Final grade: Correct. A number of papers reported encouraging results on HSCT for MS and other autoimmune conditions.
Paul, any news of Fujifilm Cellular Dynamics’ planned Parkinson’s trial?
Good report card. For PD, there is also the Studer work at Sloan-Kettering, he transplants restrictively to young patients, they just built a large facility, not sure if theirs is approved yet.
Martin, Studer is with Bluerock Therapeutics. AFAIK, his team is transplanting people with between 8 and 10 years of PD.
Many thanks, Paul. As usual you help keep us awake to new ideas and their progress with your finger on many pulses.
From the New Idea angle, might I make a plea for “Synthetic Stem Cells” ? These are in the form of biomimetic nanoparticles using FDA-approved gel to deliver LIF. A major value is that they are chemically and physically defined, whilst biodegrading to carbon dioxide and water, proving safe and potent for myelin repair in gold standard models. Interestingly, LIF formulated as LIFNano™ is a thousand times more potent than soluble LIF.
As a cell-free biomimetic stem cell, LIFNano™ escapes the pinch points of cell-dependent therapies – meaning it is also “green” in the context of climate change, with universal applicability at low cost. Topical as you look into your 2020 crystal ball.
I don’t know if Gene therapies fall under the purview of this blog. However, if they do please go ahead and blog about voyagers AADC gene therapy and axovant’s AXO LENTI PD. The former restores AADC expression in the nigro striatal Pathway through the overexpression of AADC in the striatum, that way hoping to increase Levodopa response to the miracle it was in early stage disease.
Axovant are going one step further and packaging all three Genes for endogenous dopamine synthesis in the striatum. This derived from an earlier construct called prosavin that had limited success but lot of promise. Hopefully this will allow for a much more manageable OFF state and an excellent ON state through oral medication. Santa however tells me that the strategy is fraught with risks since it leaves and unregulated amount of dopamine in the striatum, needing to possible dyskinesias. The future of stem cell therapy and gene therapy looks bright.
I’m pretty sure no one cares if they have the disease as long as the symptoms don’t show up.
Thanks for sharing your views on these 2 gene therapy efforts.
You forgot to mention Bluerock Therapeutics ahead of Aspen neuroscience. They’re literally one step away from their cell transplant trial. Just awaiting final FDA go ahead before they begin dosing their cohort of 10 patients
Excellent point. I rushed on that one and goofed. I have added BlueRock in there first as an udpate.
Hi Paul,
Don’t be so hard on yourself for Athersys.
Athersys did release positive results from a phase 1/2 trial for ARDS and also received Fast Track designation from the FDA.
http://www.athersys.com/investors/press-releases/press-release-details/2019/Athersys-Presents-Data-From-Its-Acute-Respiratory-Distress-Syndrome-Clinical-Trial-at-American-Thoracic-Society-International-Conference/default.aspx
You just missed 365 day data from the ARDS trial which should be released in the next month or so. Other catalysts include Japanese partner Healios with their Treasure stroke trial completion mid year and ARDS trial estimated completion by year end.
Stay tuned, 2020 could be an interesting year to follow Athersys.