Stem cell predictions top 20 list for 2016

Stem Cell PredictionsWhat will the new year have in store for stem cells?

2016 promises to have many striking stem cell developments. Below are my top 20 stem cell predictions for what is to come this year in no particular order. Share your stem cell tea leaves in the comments please.

  1. Another stem cell biotech acquisition by pharma (recall Ocata (almost now finally sold) & CDI in 2015).
  2. Charging patients for clinical trial participation, particularly in Japan due to the new policy and here in the US related to predatory clinics, remains a hot topic
  3. Stem cell clinics and doping in sports flares up more
  4. Organoids continue to excite
  5. Bioheart and some other small stem cell companies struggle
  6. Stem cell stocks overall have a bad year
  7. Stem cell clinics ever more aggressively use celeb clients for PR and marketing Why? It is powerful, effective, and essentially free advertising
  8. More news on human-animal chimeras
  9. FDA continues its slow-go approach to action on stem cell clinics/unapproved stem cell products
  10. Pressure from industry and some academics on FDA to not regulate adipose products as drugs and/or to not enforce some other draft guidances including at the upcoming public hearing on the draft guidances
  11. FDA receives increasing public criticism for “slowness” on approving new stem cell therapies including from beyond the stem cell clinic industry
  12. One or more lawsuits against a stem cell clinic
  13. A new stem cell scandal pops up related to publication issues
  14. Some hiccups on mitochondrial transfer/3-person IVF in the UK or China
  15. The trend last year of increasingly blurred lines between legit research entities such as universities and dubious stem cell enterprises continues. This is worrisome.
  16. Stem cell-derived human germ cells stay in the headlines. This has exciting potential for providing new windows into human development and tackling infertility, but also raises thorny issues such as human genetic modification
  17. ViaCyte has some big news
  18. High-profile developments on veterinary use of stem cells
  19. Animal cloning, particularly in China, continues to proliferate
  20. More rumblings on possible human reproductive cloning attempts

Disclaimer: This post is not meant as financial advice. Consult an expert before making financial decisions.

Top 10 stem cell stories of the year 2015

liver organoidWhat were the top 10 stem cell stories of the year?

What were the biggest news and science headlines in the stem cell field in 2015, whether good or bad?

Below are mine. What are your top 10 stem cell stories of the past year? Let us know in the comments.

  • Organoids explode. Stem cell-based mini-organs (organoids) have exploded in terms of impact in the last couple years. 2015 had many examples of these super cool human organs in a dish made from stem cells. Organoid technology has huge potential for teaching us about human organ development and function as well as disease pathogenesis and treatment. I recommend this interview by Alexey with organoid pioneer Madeline Lancaster for more on this exciting trend.
  • Clinical trials pile up. More good news. The number of real, promising stem cell clinical trials continues to build. This is fantastic and shows the continuing maturation of the stem cell/regeneration medicine/cell therapy fields. Think about where things were 5-10 years ago with stem cell clinical trials and you see how much progress has been made.
  • Sale of CDI and Ocata to Japanese pharma. Japan continues to show leadership in commercializing stem cells and regenerative medicine, and this in part manifested in 2015 with the big-time purchases of two American stem cell biotechs by Japanese pharma companies. Does the nation of a biotech company matter in this day and age? Are pharma acquisitions of small stem cell biotech good news or bad or some combo of the two? We can expect more corporate acquisitions in the stem cell arena in the coming year. Stay tuned for my top predictions for the stem cell field in 2016.
  • Stopping of IPS cell clinical study in Japan with broader regulatory switch there to allogeneic IPS cell focus. The pioneering macular degeneration clinical study using IPS cells in Japan was stopped this year and now a second IPS cell study (Parkinson’s Disease) there has been delayed. Regulatory changes now mandate that IPS cell clinical work be done with allogeneic cells. This change still remains somewhat of a mystery given the big potential of IPS cells on the patient-specific front using an autologous approach. We’ll hear a lot more about this in 2016. Another regulatory change in Japan is the move to allow charging of patients for clinical trial participation. I’m not a fan of this change to put it mildly.
  • CRISPR. Of course CRISPR is not specific to stem cells, but it opens new doors to understanding stem cell biology and development, particularly in the human research sphere. The genetic research on human stem cells that has opened up because of CRISPR is very exciting.
  • Human genetic modification on the table. Related to the above, gene editing of human pluripotent stem and germ cells has big ethical and social implications. The potential future production of genetically modified human beings via primordial germ cell or pluripotent stem cell editing raises big, thorny questions. Whether we are talking about genetic modification of somatic cells for therapy or production of actual designer babies, this topic was a hot one in 2015. For more, check out my new book, GMO Sapiens.
  • Stem cell clinics continued to bloom. From Internet searches to patient contact, the data out there are consistent with a continued rapid growth of stem cell clinics pitching at best unapproved stem cell interventions to at worst stem cell snake oil.
  • FDA action absent still on predatory clinics. For two years the FDA has practically speaking done nothing to address the exploding problem of stem cell clinics (see above) selling unapproved stem cell “treatments” to tens of thousands of patients in the US. We cannot forget about stem cell tourism, but the stem cell clinic problem in the US is massive and growing, and only made worse by an FDA that appears to either have mixed feelings on it or simply be moving in slow motion. Something’s got to give on this and I thought that would happen in 2015. Maybe it will in 2016.
  • Celebrities as advertising for stem cell clinics. Also in the dubious sphere we saw in 2015 an acceleration of celebs being the top drivers of patient traffic to stem cell clinics. Gordie Howe made the biggest news, but then there was Bart Starr and many others. For every famous person who puts themselves at risk or who is put at risk by their families looking for hope from stem cells, how many ordinary people follow suit and how many people will lose precious savings and potentially be hurt?
  • CIRM 2.0 picks up pace. Ending with some good news, the new incarnation of The California Institute for Regenerative Medicine (CIRM) as CIRM 2.0 (a name I admittedly enjoy saying as I coined the term) continued to build momentum in 2015. CIRM 2.0 has a distinctly clinical focus. The agency is picking up the pace on issuing RFAs and new funding. 

Prospects for stem cell stocks in 2016

stem cell stocksMoney plays a major role in the arena of stem cells, cell therapy, and regenerative medicine. The financial side of this cutting-edge field ranges from grants to private investors to publicly traded stocks. One could argue that the flow of total investment going into this area has a direct and tangible impact on how rapidly and effectively the research moves forward.

In the specific area of publicly traded companies working in stem cells, cell therapy, and regenerative medicine, what’s the investment outlook for the coming year? How will stem cell stocks do?

I’m not an investment expert so don’t take this post as financial advice, but overall things look pretty mixed for 2016. On the one hand, there have never been more clinical trials and ones with real potential, which is a great positive hallmark. On the other hand, for many stem cell companies what seems to be “good news” does not always equate to consistent increases in the stock PPS or company valuation. It’s a super high-risk arena for investors.

Pharma purchases in 2015 of stem cell companies such as CDI and Ocata can also be viewed in polar opposite ways. This could signal more purchases/acquisitions of stem cell biotech’s coming in 2016 and that could both help get treatments to patients and benefit investors, but will the price be right and does it translate to a positive outcome for investors? Many investors are very unhappy about the price paid for Ocata. What do you think of that sale?

What are your favorite stem cell stocks as you look ahead to 2016 and why? Do you see acquisitions coming? What about IPOs? Could an exciting stem cell company such as ViaCyte do an IPO in the next 12-24 months?

TGIF: Stem Cell Stream of Consciousness for the Week

It seems like Fridays are always a good time for reflection.

It’s not like the scientist’s week ends on Friday sad to say, but still at least we can pause momentarily and reflect on the maelstrom that is our lives, while cracking open another diet Coke or pouring another cup of coffee.

This week was a great one for getting into some intense discussions with other scientists both in person and electronically.

CRISPR the talk of the biology town. Everyone is talking about CRISPR-Cas9. There are many subtopics. I’m just going to throw them on the table in a stream of consciousness here. Gee, CRISPR is not as specific as I thought. Uh oh, CRISPR is harder to get to work than I thought (as portrayed in early papers). CRISPR is fricking awesome. Oh, **** , people are going to CRISPR human embryos? The people who innovated CRISPR-Cas9 like Jennifer Doudna are amazing and deserve a Nobel Prize.

CIRM change leaves some feeling unsettled. There is starting to be a feeling of disconnect, especially amongst more basic stem cell researchers, with CIRM.

CIRM just isn’t–at least so far in the existence of CIRM 2.0–funding basic or early translational stem cell research much or at all any more. Maybe it will in the future as there have been hints about that, but support from CIRM in a major way for important, basic stem cell research is probably basically over. The wildly successful and positive CIRM Bridges Program is nearing an end. The CIRM T32 training program are winding down. Many of the early-mid-stage CIRM grants are ending.Lujan paper

There’s definitely a feeling in the California stem cell community of CIRM kind of having moved on from a whole class of stem cell researchers barring those fortunate few who still have grants. Maybe there can be a future reunion.

CDI Buyout Big Bucks. Fujifilm shelled out almost a 1/3 of a billion dollars for Jamie Thomson’s Cellular Dynamics International (CDI). I guess IPSC are really worth big bucks, although CDI isn’t so IPSC-specific anymore. Regenerative Medicine is now proving its worth big bucks. Billions and billions?

Cool New Wernig Lab paper in Nature, but what does it mean? I saw that new Wernig lab Nature paper with Garry Nolan’s uber-fancy FACS-mass spec data. Those SPADE analysis images that look like trees branches into rainbowy fractals sure are pretty. Is it like some kind of heatmap-esque, principle component, multidimensional analysis coolness? Yeah, I’m going to have to read this Wernig lab paper more carefully.

Over at the Stanford media the wonderful Krista Conger boiled the paper down to say that there is a defined “transition state” during reprogramming.

Was Fujifilm CDI acquisition a good move by the companies?

The big stem cell news already this week is the acquisition by Fujifilm of Cellular Dynamics International (CDI) for a whopping $307 million dollars or about $16.50 per CDI share. You can read the press release here.

It was less than two years ago that CDI did its IPO with an initial offering of about $46 million.Cellular Dynamics

Fujifilm reportedly has a multibillion-dollar war chest for buying into the life sciences sector and a growing interest in regenerative medicine. Note that there’s some fun, but perhaps over the top speculation going on in terms of whom they might snap up next.

CDI is an unusual company in the sense that it began about a decade ago with a very sexy idea of using human pluripotent stem cells to cure disease, but now as xconomy.com refers to it is focused more in the “unsexy” business of manufacturing cellular products from stem cells.Fujifilm Logo

Although the production side of regenerative medicine may not sound very innovative, it turns out that the reality is that making high-quality, functional, and pure differentiated cell products from pluripotent stem cells is no easy task. Throw in making such products from human induced pluripotent stem cells (IPSC) and it starts to become very exciting and important.

So was this a good move by Fujifilm and CDI?

Good news for the stem cell/regenerative medicine field overall?

My sense is an initial “yes” all around overall, but with some remaining questions.

If Fujifilm wants to grow a presence in IPSC and more generally the regen med arena, it now has made a big step forward. CDI has worked with CIRM, NIH, and a number of biotech companies. Again, the process of making the “real deal” pure, functional cellular products from IPSC is very important and valuable. The evolving CDI “superdonor” IPSC bank is very cool and could have huge value.

Perhaps something we could learn more about is how Fujifilm will make use of the unique IP of CDI specifically. Also how does the fact that CDI has to license IPSC technology via the Yamanaka Patents come into play? What about the overall price tag just over $300 million?

From the press release comes the Fujifilm perspective:

Commenting on the transaction, Shigetaka Komori, Chairman and CEO of Fujifilm, said, “We are delighted to be able to pursue the business from drug discovery to regenerative medicine with CDI, which develops and manufactures iPS cells. We have optimal scaffolding material, ‘recombinant peptides’, for cell generation and technologies useful for regenerative medicines such as material science and engineering. Our group company, Japan Tissue Engineering, markets regenerative medicine products in Japan. By welcoming CDI to the Fujifilm Group and by combining the technologies and knowhow of both companies, we will seek synergies and efficiencies to be more competitive in the field of drug discovery and regenerative medicine.”

From a CDI perspective, this seems like a major win for their team, who will benefit greatly financially from this deal. Although Fujifilm intends to maintain CDI as a Wisconsin-based subsidiary, as xconomy.com points out it’s not clear what this deal means for the employment of the 150+ CDI workers there longer term. “Synergies” and “efficiencies” after an acquisition can mean reductions in employees. It seems likely that Fujifilm will continue the momentum of CDI and help to give clinical meaning to the efforts of the CDI scientists:

Robert J. Palay, Chairman and CEO of CDI, added, “CDI has become a leader in the development and manufacture of fully functioning human cells in industrial quantities to precise specifications. CDI and Fujifilm share a common strategic vision for achieving leadership in the field of regenerative medicine. The combination of CDI’s technology with Fujifilm’s technologies, know-how, and resources brings us ever closer to realizing the promise of discovering better, safer medicines and developing new cell therapies based on iPSCs.”

For the field overall, this deal is good news. It would seem a clear, strong indicator of the great, concrete value of regenerative medicine and cellular therapies. Nearly a third of a billion dollars is nothing to sneeze at. Notably, this is not about clinical trials or that kind of specific pipeline, but more about concrete cellular products and technology. That’s an important distinction because in this sector oftentimes most of the value and growth emphasis is placed on rapid bedside potential.

Disclaimer: this post is not financial advice and the author has no financial interest in either company.