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.

CRISPR Update: Patents, Embryos, & IPOs, oh my

It’s been a busy few weeks for the CRISPR arena so I’ve made a CRISPR Update. I’ve listed below links to some commentaries and key developments.Joanne Manaster

 

Fun Video interview on Read Science! with Joanne Manaster on my new book on CRISPR in humans, GMO Sapiens.

CRISPR: Pursuit of profit poisons collaborationNature piece by Jacob Sherkow

HIV Fights Off CRISPR Gene-Editing Attack. HIV adapts when CRISPR attacks.

2nd group CRISPR’s human embryos and things don’t go well. More Indels than precise gene edits, mosaicism and more.

CRISPR biotech Intellia strikes licensing deal with Regeneron, readies IPO. It’s interesting that there are these CRISPR IPO’s when the CRISPR patent situation remains entirely up in the air.

George Church versus Marcy Darnovksy on human modification in the WSJ

CRISPR/Cas9 Used to Create Knockout Chickens. Bock bock adoodle moo

CRISPR embryo OK signals ongoing liberal UK trend on human modification

Kathy Niakan

Dr. Kathy Niakan, Francis Crick Institute Photo

With the approval today of the use of CRISPR in human embryos, the UK continues its recent trend toward a more permissive regulatory policy on human genetic modification. There are both risks and scientific benefits that come along with this trend.

Last year the UK approved an experimental technology with the goal of preventing the transmission of mitochondrial disease. The approach would try to prevent this terrible genetic disease but in the process would also create genetically modified babies. I lobbied the UK Parliament to not approve this technology (variously called 3-person IVF, mitochondrial transfer, and more controversially “3-parent babies”) at this time.

My view was that there wasn’t even close to enough data to support its safety and efficacy at present, but in the future we might know more that tells us it is wise to proceed. I still feel that way today and I’ve heard rumors that the UK scientists even now with the 3-person IVF approval are taking a relatively slow-go approach on its implementation. If correct, I think that’s wise of them. The 3-person IVF technology could do more harm than good. We just don’t know.

With a green light now for CRISPR’ing human embryos at the request of Kathy Niakan, a biologist at Francis Crick Institute, the UK continues its broader trend toward being more open to genetic modification on the human front. The stated goal of the UK CRISPR research on human embryos is not to make designer babies, but rather to advance our understanding of human development, which remains poorly understood:

“I promise you she has no intention of the embryos ever being put back into a woman for development,” Robin Lovell-Badge, group leader at the Crick Institute, told TIME. “That wouldn’t be the point. The point is to understand things about basic human biology.”

Dr. Lovell-Badge is right on this research potentially teaching us very important things about human biology and I can’t imagine that Dr. Niakan has any interest in designer baby research. I’m not worried about that and I’m excited about the research potential here.

It’s frustrating for us biologists that we still know more about the development of other animals (e.g. mice or fruit flies) than that of our own species. CRISPR could change that and I believe it could do it in a big way. So with the appropriate oversight, bioethics training, and transparency, I could support this CRISPR work in the UK. I need time to read up on what exactly they have planned. Also, see my ABCD plan on human germline modification.

However, at the same time we have to be clear that thing aren’t so simple in terms of keeping control of technologies and there are big risks here at a more global level. Collectively we are walking a fine line in this area. All it would take is someone going rogue by taking the same CRISPR’d embryos and implanting them in a surrogate for the field to find itself in a very dangerous situation.

The UK is heading in a more permissive direction on human modification. That may be appropriate to some extent and worth some risk, but how far should it go? What about the US, China, and other countries? We need more talk (meaning dialogue, debate, etc.) to go along with the increasing action (research) in this area.