Top 20 2017 stem cell predictions: score card at 1/2-way point

In December of each year I make a list of stem cell predictions for the coming year, and I made 20 such predictions for 2017 so I’m wondering how I’m doing so far on these now that it’s June. Below is my work-in-progress scorecard for these so far.

  1. Positive news from Asterias on trial for stem cell-based therapy for spinal cord injury. Status: Correct, trial looking encouraging.
  2. Upbeat news from ViaCyte on stem cell-based therapy trial for diabetes. Status: Correct, raising $10M and things looking promising.
  3. More positive news from the old Ocata now under Astellas umbrella on trial use of stem cell-derived RPE for Macular Degeneration. Status: Not Yet.
  4. Good news on the adult stem cell front on trials for one or more major diseases. At least one and probably more positive developments here. Status: Correct, lots of good news. The potential for stem cell generated blood is just one example.
  5. Fake news hits stem cell arena. Stem cell clinics use fake news. For instance, this might be a media mouthpiece for one or more stem cell clinics actively using fake news-like approaches to promote them. Status: Correct, unfortunately.
  6. More clarity on clinics: data. More academic publications on the practices and outcomes of stem cell clinics are published, bringing greater clarity to what is going on with actual data. Status: Not Yet.
  7. More lawsuits against stem cell clinics. There has been a lot of buzz on this behind the scenes already and cases popping up in 2016. This is going to grow in 2017. Status: Not Yet.
  8. Concrete clinic harms. We learn more about additional examples of patient who feel they’ve been harmed by American stem cell clinics including in particular alleged clinic-caused blindness. Status: Not Yet.
  9. Some other federal agency besides the FDA makes news on stem cells. This may not be until 2018, but we’ll see. Status: Not Yet.
  10. At least one FDA guidance is finalized. The FDA finalizes at least one of its four recent stem cell-related guidances, but probably not all four. Status: Not Yet.
  11. More than one warning letter. The FDA issues more than one warning letter to stem cell clinics in this year. Will it still be a drop in the bucket or some kind of decisive action? The FDA may have more difficulty taking action within the Trump context and much will depend on who is the new Commissioner.  Status: None Yet.
  12. Japan IPSC trial starts. Great news as at least one IPSC trial begins in Japan. Maybe two. Status: Correct.
  13. Cures yields regen med IND. The FDA takes at least one accelerated stem cell-related IND action traceable to the Cures Act related to a promising new stem cell/regenerative medicine therapy. Hopefully no direct to consumer businesses try to tap in. Status: Looking correct based on many RMATs granted.
  14. Athersys, Cytori, and Mesoblast have some ups & downs amongst them. Status: Correct.
  15. Prop 71 2.0. CIRM and/or Prop 71 supporters start more openly talking about a new round of CIRM funding. This may include mention of Trump as problematic for the stem cell field and the continuing need for California to take the lead. . Status: Correct. Will they pull the trigger though this year?
  16. Trump somewhat, but not entirely limits ES cell funding. The Trump administration probably does not outright ban federal funding of embryonic stem cell research, but there may be some effort to limit it in some way such as not supporting generation of new lines perhaps à la Bush.  Status: Not yet.
  17. Fetal tissue research restriction effort. The Trump administration and/or the GOP attempt to restrict human fetal tissue research.  Status: Not yet.
  18. CRISPR of human embryos is blocked or limited in some way in the U.S. (e.g. FDA is not permitted to review applications related to this area as was the case with the rider on spending bill for 2016).  Status: Not yet.
  19. Trump creates something like Bush’s President’s Council on Bioethics. It’s packed with conservatives including someone tied to the Witherspoon Institute. Deja vu all over again.  Status: Not yet.
  20. Florida acts on clinics. The state of Florida takes some action on stem cell clinics, which are out of control there. Things are a mess clinic wise here in California too, but I’m not so sure the state will do anything helpful to deal with it.  Status: Not yet.

Overall, I’m doing OK so far, but much is up in the air for the final 6 months of the year.

Sunday brunch buffet of stem cell good news, fun links, & cool papers

Enjoy! A brunch for the brain.

News and links

CBER Director Focuses on Flexibility to Advance Regenerative Medicines

Lab-Grown Blood Stem Cells Produced at Last

Transplanted stem cells become eggs in sterile mice

hair stem cells

Liao, et al Figure 2D

Maryland fund awards $8.5 million for stem cell research

Positively good news from Asterias for CIRM-funded stem cell clinical trial for spinal cord injury

Sergio Canavero: Will His Head Transplants Roll?

Century-old tumours offer rare cancer clues

Papers

Identification of hair shaft progenitors that create a niche for hair pigmentation (see Figure 2D above)

Tip110 Deletion Impaired Embryonic and Stem Cell Development Involving Downregulation of Stem Cell Factors Nanog, Oct4, and Sox2

Modeling Psychomotor Retardation using ipscs from MCT8-Deficient Patients Indicates a Prominent Role for the Blood-Brain Barrier

Elevated FOXG1 and SOX2 in glioblastoma enforces neural stem cell identity through transcriptional control of cell cycle and epigenetic regulators

Prc2 facilitates the regulatory topology required for poised enhancer function during pluripotent stem cell differentiation

Upbeat prospects for some California clinical trial efforts from CIRM

Over at the California Stem Cell Report, David Jensen is reporting on some good news from CIRM for California on the stem cell clinical trial front.

Stem cell biotechs Asterias and Capricor have stem cell trials supported by 20+ million in CIRM funding each and have been hitting milestones. These trials are progressing and so far have good safety profiles. Asterias and CIRM have mentioned some possibly encouraging early hints at efficacy as well in its trial, and apparently there are hopeful hints from the Capricor trial too.

See the posts from CIRM here (a weekly summary kind of post that begins discussing Asterias) and here. For background, also see past posts I’ve done on both companies here and here in the archives, and see especially my interview with Asterias leadership from a few months back.

It’s early days for these trials and at these phases they are not really designed to look for efficacy so a conservative approach to discussing such trials is in order given the stage, but at this phase of the game for early clinical trials the news has been all one could hope for so far in both cases.

asterias-cells

Asterias cells

The Asterias and Capricor trials are for spinal cord injury and Duchenne muscular dystrophy, respectively. The latter trial utilizes the Capricor CAP-1002 product, which is a cool allogeneic cardiosphere technology made from donor human heart tissue. A beating cardiosphere from a different source (IPSCs) can be seen in the video above. Asterias’ trial employs their OPC product made from hESCs, which is also inherently allogeneic. The idea of potentially repairing the injured spine via stem cells is intriguing.

I’m hoping in the next month or so to do a broader update on the stem cell and regenerative medicine biotech arena. By way of disclosure, I do not have any financial stake in either company discussed here.

Good stem cell news on trials, FDA, cool new papers & more

pericyte-paper

Guimarães-Camboa, et al, Cell Stem Cell figure

In the whirlwind that is the stem cell and regenerative medicine world, there are many concerning things that need attention, but also good stuff happens too and this post focuses on the positive.

The Asterias spinal cord injury clinical trial, a phase 1/2a trial called SCiStar, continues to make encouraging news with a clean safety profile and additional hints at possible positive indicators of efficacy. With the usual, important caveats such as that this is early and it is not an RCT, the SCiStar momentum is positive. I’m excited to see what the future holds for this one including from an RCT. You can read my interview from last month with Asterias leadership here.

I remain very enthusiastic about ViaCyte’s trial as well using a stem cell capsule product for treatment of Diabetes. Their joining forces with BetaLogics a year ago just made their position even stronger.

I’m going to do a post soon on an analysis on the total number of stem cell and regenerative medicine trials compared to historical data I collected. Stay tuned on that. I’m guessing it’ll be good news.

Recently, we also saw evidence of fast action from FDA in response to the 21st Century Cures Act in terms of providing a clear document on Regenerative Advanced Therapy designations and applications. It’s still unclear how the Cures stem cell provisions will play out, but I consider quick, clear action from the FDA to be a positive. I wish they were this fast on other stuff like dealing with stem cell clinics marketing unapproved drug products.

There have been a number of cool papers recently that I recommend reading:

Top 20 Stem Cell Predictions for 2017

stem cell crystal ball

Stem cell crystal ball

Each year I make a list of predictions for the stem cell and regenerative medicine field for the coming new year. Later in this post I list my top 20 stem cell predictions for 2017. In looking at my past predictions I realized this will now be my 7th year doing stem cell/regenerative medicine yearly predictions.

You can see below links to these predictions for past years, which sometimes seems rather far removed from today and in other cases strike me as strangely apropos of our times.

What will 2017 bring? Below are my top 20 predictions in no particular order except starting with a few hopeful visions for the coming year.

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