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.

How do you rate Trump on science so far?

How Scott Gottlieb may transform the FDA’s approach to stem cells

The Trump FDA commissioner nominee Dr. Scott Gottlieb Dr. Scott Gottliebcould dramatically alter how the agency regulates investigational stem cell therapies. How might such changes unfold? There are potential upsides and downsides to  the seismic shift that could be in the offing.

Gottlieb has written in the past about his perception of FDA over-regulation of stem cells such as in this piece in the WSJ. There he made a number of assertions that signal his view at least back then that when it comes to regulation of stem cells by the FDA, less is more.

More recently, a speech he gave at an ISSCR meeting in Berkeley last year is much more balanced in tone than the 2012 WSJ piece. This quote from the Berkeley talk, for example, is balanced and emphasizes standards:

“Expediting the development of these novel and transformative technologies like gene- and cell-based therapies doesn’t necessarily mean lowering the standard for approval, as I believe other countries have done. But it does mean having a framework that’s crafted to deal with the unique hypothetical risks that these products pose.”

Still that WSJ article is concerning.

For instance, he and his co-author Coleen Klasmeier, both former employees of the FDA, strongly criticized a federal court ruling in 2012 that the FDA could regulate laboratory-proliferated stem cells as a biological drug. Oddly, much of the basis for their criticism was far broader than the reality of the court ruling. They suggested, for instance, that the ruling opened a veritable Pandora’s box of FDA over-reach potential that extended to all autologous uses of stem cells (lab-grown or not) and beyond, but that was not the case as the ruling was focused on lab-grown stem cells. Even so they made largely unsupported generalizations such as the following:

“If the FDA’s victory is upheld on appeal, then conceivably nothing done as part of clinical practice is beyond the agency’s reach.”

In the intervening years since the 2012 court ruling and the WSJ article, we have seen that the sky hasn’t fallen from the FDA having obtained the defined authority to regulate lab-expanded stem cells as drugs. In fact, oddly enough if anything the more time that has passed, the less inclined FDA’s CBER biologics branch that oversees stem cells has been to take action on stem cell biologics. It’s hard to view the FDA issuing less than one warning letter per year to stem cell clinics even as there are upwards of 600 of these clinics as a form of overreach.

From a scientific and medical perspective, there are good reasons to regulate lab-grown stem cells as drugs. The cells are known to change their differentiation properties, accumulate mutations and epigenetic changes, and undergo other significant alterations in the dish in the lab. There is also, perhaps more directly related to the FDA’s mandate on biologics, substantial potential for contamination of cells in a lab during proliferation.

Since 2012, another big change has been the explosion of adipose stem cell clinics onto the scene, which use more than minimally manipulated liposuctioned material to make a biological drug product via enzymatic and other steps. However, the clinics argue their products aren’t drugs and they generally do not have any FDA approval to market stem cells. Nonetheless such offerings are being sold at hundreds of clinics around the country. How would a newly minted FDA commish Gottlieb view these clinics? Through the same lens as the autologous, homologous bone marrow-based approaches? What about the clinics using bone marrow and amniotic cells in non-homologous manners that make such products drugs? Continue reading

Poll: How will Trump’s FDA handle stem cells?

TGIF: Week’s Stem Cell Headlines, Papers, & Resources

TGIF science dart board

Modified Wikipedia image

What are the latest developments in stem cells and regenerative medicine in this first week of 2017 and right before the end of the past year? Here are some of the headlines and papers that have popped up that seem notable or surprising. Be sure to also check out our stem cell & regenerative medicine meeting hub for 2017.

Alexey’s review of Cell Therapy (including stem cells/regen med) in 2016. Must-read. This links to his part 2 and there is a link to part 1 there. This is on his Cell Trials blog.

A Rapid Pipeline to Model Rare Neurodevelopmental Disorders with Simultaneous CRISPR/Cas9 Gene Editing. Cool system described in Stem Cells article.

Scientists find link between maternal environment and neurodevelopment disorders. More on neurodevelopment. Environment and genetics do a tango in development.

NYSCF and PGP announce availability of unique new stem cell resource for scientific research. More stem cell resources the better.

What happens to rejected papers? Neuroskeptic takes a look. A little off topic, but very interesting.

Stem Cell Research: How Scientists Grew Stomach Tissue In A Lab. Does it get hungry?

How Trump Could Slow Medical Progress. Worries about Trump & biomedical sciences.

Will President Trump restrict embryonic stem cell research funding. More worries on Trump. 

Genetically engineered immune cells melt away deadly brain tumors (from CIRM blog) New approaches to brain tumors are urgently needed. Minor thing — not sure about the use of “melt away” as a metaphor here.