Stem cell good news: CIRM funds new cutting edge studies

CIRM announced recently the funding of a number of exciting new studies.

ViaCyte received additional funding to support its development of its hESC-based pancreatic progenitor cell product PEC-Direct clinic trials. This work is very promising. CIRM also funded additional diabetes-related research by Humacyte on engineering blood vessels for use in dialysis, which is very creative.

VC-01 post-implant final

VC-01 post-implant

I was happy to see that my colleague here at UC Davis School of Medicine, Professor David Segal, received funding for a cutting edge grant on using gene editing for Angolan Syndrome. The grant is entitled, “MSC delivery of an artificial transcription factor to the brain as a treatment for Angelman Syndrome”.

This funding along with a new grant for Jeanne Loring on stem cells for Parkinson’s came via CIRM’s new basic biology type of funding mechanism. A number of other cool projects got funded via this RFA including one by David Schaeffer at Berkeley on stem cell-produced oligodendrocyte precursor cells to treat neurological injury.

This all is very good news for the stem cell field.

Stem cell checkup: how are my 2016 predictions doing half way?

Stem Cell PredictionsEach December I make stem cell predictions for the coming year and I did that for 2016 where I made 20 predictions. At around mid year I do a checkup on how my predictions are doing halfway and that is the purpose of this post.

Below are my predictions that I made in 2015 for stem cells in 2016 and my general sense in green of where they stand. Overall, I’m doing reasonably well, but I kind of wish I wasn’t because so many of these are not positive developments. However, in general I remain very optimistic for the field and expect major positive advances in coming years on a number of fronts using both adult and pluripotent stem cells.

The predictions and status so far.

  1. Another stem cell biotech acquisition by pharma (recall Ocata (now finally sold to Astellas) & CDI in 2015). Checkup: Not yet.
  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. Checkup: Correct.
  3. Stem cell clinics and doping in sports flares up more. Checkup: Clinics yes, doping not yet.
  4. Organoids continue to excite. Checkup: Correct. What a great technology.
  5. Bioheart and some other small stem cell companies struggle. Checkup: Correct so far. The PPSs of small stem cell biotechs have generally not been pushed up this year by investors, but rather the reverse. Note that Bioheart is now called US Stem Cell, Inc. We can all hope that there is a turnaround for small stem cell biotechs in the market in the 2nd half of the year.
  6. Stem cell stocks overall have a bad year. Checkup: Correct so far also sadly. Note, by way of disclosure I do not currently have any direct stem cell stock investments.
  7. Stem cell clinics ever more aggressively use celeb clients for PR and marketing Why? It is powerful, effective, and essentially free advertising. Checkup: Correct.
  8. More news on human-animal chimeras. Checkup: Correct. Another hot topic.
  9. FDA continues its slow-go approach to action on stem cell clinics/unapproved stem cell products. Checkup: Correct.
  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. Checkup: Correct. REGROW and other efforts have been unprecedented. Note that the FDA public meeting will now be held in September rather than in April.
  11. FDA receives increasing public criticism for “slowness” on approving new stem cell therapies including from beyond the stem cell clinic industry. Checkup: Correct in a big way. 
  12. One or more lawsuits against a stem cell clinic. Checkup: Correct and several more seem to be brewing. Note that it appears that the part of the suit involving US Stem Cells, Inc. has been settled, while a separate part of the case against other defendants continues.
  13. A new stem cell scandal pops up related to publication issues. Checkup: Correct. You just have to go visit Retraction Watch (e.g. the Spain mess) or PubPeer, and then also see the continuing Macchiarini saga.
  14. Some hiccups on mitochondrial transfer/3-person IVF in the UK or China. Checkup: Correct. Diseased mitochondrial carry-over and mito-nuclear cross-talk issues have popped up and deserve serious attention.
  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. Checkup: Correct.
  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. Checkup: Correct.
  17. ViaCyte has some big newsCheckup: Not yet. What a great company.
  18. High-profile developments on veterinary use of stem cells. Checkup: Correct. For instance see this piece in Scientific American. Cool stuff!
  19. Animal cloning, particularly in China, continues to proliferate. Checkup: Correct.
  20. More rumblings on possible human reproductive cloning attempts. Checkup: Not much concretely yet. See this piece on cloning focusing on 20th Anniversary of Dolly.

Stay tuned as near the end of 2016 I will do a final assessment of how I did on my stem cell predictions and then make stem cell predictions for 2017. What are your stem cell predictions?

ISSCR Releases Flood of Stem Cell Policy Docs

A committee of the International Society for Stem Cell Research (ISSCR) did one heck of a document dump yesterday on stem cell policy, releasing a whole bunch of policy recommendations on stem cells and more.

The torrent from ISSCR included a 37-page policy statement itself as well as several papers in top journals including the Lancet, Science, and Nature.

This output was the product of the members of a special  ISSCR Task Force, whose members I have listed at the bottom of this post. Who are the members? These are knowledgable, extremely bright people who care deeply about the issues.

ISSCR Policy Guidelines 2016

The stem cell policy positions of ISSCR and those in the associated publications were wide-ranging, touching upon everything from avoiding stem cell research hype to policies on human embryos to CRISPR of human embryos to three-person IVF/mitochondrial transfer, to clinical trials generally to patient-funded trials and more.

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ViaCyte on the Rise: First Diabetes Trial Data & Acquires BetaLogics IP

Clinical research on Type I Diabetes is one of the most exciting and promising areas of stem cells and regenerative medicine for human disease.

Two of the coolest companies out there in this arena have been ViaCyte and BetaLogics (owned by J&J). For more on ViaCyte see my interview with President and CEO Paul Laikind from 2015.

VC-01 post-implant final

VC-01 post-implant

Today brings great news for ViaCyte on two fronts. Incidentally, this gives me a correct prediction on my Top 20 stem cell predictions for 2016.

First, it has acquired the assets and IP of BetaLogics, bringing together two of the best biotechs to use stem cells to fight Diabetes. This is a very exciting move and will strengthen ViaCyte. The terms of the deal were not released. This is also good news for Diabetes patients as this seems likely to give ViaCyte a big boost. ViaCyte’s press release quoted Laikind:

“For more than a decade BetaLogics and ViaCyte have been independently working toward a stem cell‐derived therapy for diabetes. By combining the intellectual property and other assets of BetaLogics with ViaCyte, we will further strengthen our advanced program focused on

insulin‐dependent diabetes and solidify our leadership in the field,” said Paul Laikind, PhD, President and CEO of ViaCyte. “We look forward to delivering effective new treatments for this difficult disease.”

Second, ViaCyte has had some notable success in producing the first data in its Phase 1/Phase 2 clinical trial called STEP ONE (Safety, Tolerability, and Efficacy of VC‐01 Combination Product in Type One Diabetes). From the company on this development:

  • “The data show that pancreatic progenitor cells (PEC-01) within VC-01 can engraft, vascularize, and differentiate into pancreatic beta islet cells (insulin-producing cells) 12 weeks after implantation.
  • This is an important milestone in the development of a “functional cure” for type 1 diabetes.  And an important milestone for the industry — the clinical trial is supported by JDRF and CIRM (California Institute for Regenerative Medicine)
  • The goal is for the PEC-01 cells in VC-01 to mature and secrete insulin and other regulatory factors in response to blood glucose levels.  “

Together these events solidify ViaCyte’s leadership in the area of stem cell-based treatments for Diabetes.

First impressions of the US stem cell environment from an Aussie

Heather_MainBy Heather Main

I moved to the San Diego from Australia in August 2015, and Paul asked me if I could write something on my first impressions of doing science in the US, as opposed to other countries I have worked/studied in (Australia, Scotland, Sweden and Singapore).

If you look at the land size of Australia and consider that we have only 7% of the US population you will understand directly that funding and collaborative opportunities are not as easy to find. There’s probably little collaborative advantage in the US compared to Europe, which has over double the number of people as the US and only 3% larger land mass. In defense of Australia we punch above our weight, with the first stem cell company (Stem Cell Sciences) and currently the world’s largest (Mesoblast) originating there…even Viacyte has Australian origins.

It seems like it is hard not to be political here in the US….I guess I have arrived at a special time, but after working in Sweden for 5 years and living through gun control regulation following the 1996 Port Arthur massacre in Australia, I don’t understand the opposition to publicly funded healthcare, how a regarded education is ‘purchased’ and the 17th century resistance to gun control regulation. …I guess you need to be born into it. I think the political minorities have almost as much backing in Australia but are not as extreme and have less volume, so the international effects are minimised.

Relevant to stem cell research, the Colorado Springs Planned Parenthood shooting in November 2015, made me wonder how extreme the fight to protect the rights of a fertilised egg still is. My only explanation for these people is a 16th century belief in a ‘homunculus’ (fully formed miniature human present in every sperm)…Someone please get them a microscope.


Above. The : A 16th century theory for human development. Obviously not real though but an image that seems to exist in the mind of the anti-choice movement

All of this seems contradictory to a country with arguably the most advanced thinking and technologies but I was pleased to see the numbers don’t support it with;

– 2008 Bush allocation of $88 million in NIH funds to human embryonic stem cell research (compared to $381 million to adult stem cell research)

– 2014 Obama allocation of $166 million, while non-embryonic only slightly increased ($443 million), with a large allocation to iPSC projects.

This even indicates the recognised importance of pluripotent technologies over adult somatic technologies.

I wonder though if this balance is also recognised in the public and adult somatic cell spaces. In an effort to sell an alternative to hESC, the potential of adult somatic stem cells was drastically inflated. Recently I have attended a number of adult somatic stem cell talks who describe the expression of Oct4/nanog etc. in their population of choice, even call them pluripotent….though I am yet to see the teratoma/PluriTest, or clinical trial for functional long term grafting behaviour. Literally hundreds of clinical trials on MSCs have been done over the last 15 years so I guess if the data were there, we would have seen it.

MSC clinical trials

Number of registered clinical trials of mesenchymal stem cells-based therapy on

My fears were allayed when reading Don Reed’s book, in which he interviews fantastic adult stem cell researcher and patient advocate, Dr Jan Nolta. When asked if injection of mesenchymal stem cells was a possible cure for Huntington’s Disease, she answered straight out “No,…..But it buys us time”. Herself and Dr Vickie Wheelock agreed that hESC or hiPSC would be needed for the actual cure. It’s refreshing to see this honesty, a recognition that we need to work together, promote both sides, but realise the timelines of expected contribution from each. Saying it is not a cure does not mean it is not important.

So far I have enjoyed a range of meetings, including both Stem Cells on the Mesa and the World Stem Cell Summit on US soil and look forward to ISSCR in June. Unfortunately from Australia it is wildly expensive and time-consuming to travel to all of these meetings….you can fly LAX to Sydney in 15 hours as opposed to the 12 hours it took my husband to get from San Diego to Florida….

Australia US map

Good or bad I think the US is a place where anyone with a decent backing can make changes with great relevance on the International scale. If you can legalise hESC research under a Republican government, you can do anything!