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.

NAS proposes prudent US policy on 3-person IVF

3-parent babyA new report from the National Academies of Sciences, Engineering, and Medicine here in the US recommends a policy significantly more cautious on first read for 3-person IVF than that in place now in the UK.

My initial take on this new 164-page report is that it is very well thought out and the recommendations are both appropriate and prudent. I’m still reading the report so it is possible I may feel differently at the end or that others may point out concerns that I missed, but so far my impression is positive.

Keep in mind that 3-person IVF also goes by the monikers mitochondrial transfer, 3-parent IVF, and mitochondrial replacement therapy (MRT, the term used by the Academy).

Alta Charo Mito TransferNotably, the Academy committee explicitly stated that MRT is a form of human genetic modification and human germline therapy. Some proponents of MRT in the UK and even in the US had mysteriously claimed otherwise.

While the Academy committee wrote in the report that 3-person IVF is ethically permissible in theory, in practice the depth of their concern about the risks of this technology was reflected in the array of conditions that they detailed must be met first.

These conditions include, but are not limited to the following as taken in some cases verbatim from the report or the Academy press release:

  • initial MRT clinical investigations should be limited to women who are at risk of transmitting a severe mitochondrial genetic disease that could lead to a child’s early death or substantial impairment.
  • Only male embryos should be permitted to be used for pregnancy. This would avoid transmission of the donor mitochondria should something go wrong.
  • initial safety is established and risks to all parties directly involved in the proposed clinical investigations are minimized, although minimizing risk to future children should be of highest priority; (note, my interpretation of this is that more preclinical data are needed)
  • the likelihood of efficacy is established by preclinical research using in vitro modeling, animal testing, and testing on human embryos as necessary;
  • if the intended mother at risk of transmitting mitochondrial disease also desires to carry the pregnancy, it is determined by professional opinion that she is able to complete a pregnancy without significant risk of serious adverse consequences to her health or the health of the fetus;
  • clinical investigations are limited to investigators and centers with demonstrated expertise in and skill with the relevant techniques; and
  • FDA has reviewed the science surrounding matching the mitochondrial DNA subtype of the egg provider with that of the intended mother and if compelling, has considered such matching as a means of mitigating the possible risk of incompatibility that could arise from combining the egg provider’s mitochondrial DNA with the nuclear DNA of the intended mother.

I’m largely in agreement with the Academy on this policy recommendation. It seems wise and frankly more appropriately cautious based on the science than what is now allowed in the UK.

The policy recommendations were summed up by the committee Chair in this way:

“In examining the ethical, social, and policy issues associated with mitochondrial replacement techniques, we concluded that the most germane issues could be avoided if the use of these techniques were restricted by certain conditions, rather than prohibiting them altogether,” said Jeffrey Kahn, chair of the committee and the Robert Henry Levi and Ryda Hecht Levi Professor of Bioethics and Public Policy at The Johns Hopkins Berman Institute of Bioethics in Baltimore.  “Although MRT would not treat a person with a mitochondrial disease, its pursuit could satisfy prospective parents’ desire to bear genetically related offspring with a significantly reduced risk of passing on mitochondrial disease.  The limitations on MRT that we propose focus on protecting the health and well-being of children born as a result of the techniques.”

Now it will be interesting to see how and to what extent the FDA implements these policy recommendations as well as the response in the scientific community. I would predict that some proponents of MRT may view this policy as too restrictive. Perhaps some might interpret it as a green light, but so far in my reading I don’t see it that way.

The other fascinating thing is to compare this report on 3-person IVF to the organizer’s closing statement from the NAS Meeting on Human Gene Editing from December. Some of the issues raised in the new report on 3-person IVF seem to directly apply to human gene editing as well.

I appreciate that the committee was cognizant of larger issues such as transparency, access, and public discourse.

Here is the committee roster.

Jeffrey Kahn, Ph.D., M.P.H. (chair)

Robert Henry Levi and Ryda Hecht Levi Professor of Bioethics and Public Policy Berman Institute of Bioethics

Johns Hopkins University

Baltimore, M.D.

Jeffrey R. Botkin, M.D., M.P.H.


Division of Medical Ethics and Humanities, and

Professor of Pediatrics

University of Utah School of Medicine

Salt Lake City, U.T.

David Chan, M.D., Ph.D.

Professor of Biology

California Institute of Technology

Pasadena, C.A.

R. Alta Charo, J.D.1

Warren P. Knowles Professor of Law and Bioethics

School of Law and Department of Medicine and Public Health

University of Wisconsin at Madison

Madison, W.I.

James F. Childress, Ph.D.1

University Professor and John Allen Hollingsworth Professor of Ethics

Department of Religious Studies

University of Virginia

Charlottesville, V.A.

Alan H. DeCherney, M.D.1

Associate Clinical Director and Branch Chief

Intramural Program in Reproductive Endocrinology and Gynecology

Eunice Kennedy Shriver National Institute of Child Health and Human Development

National Institutes of Health

Bethesda, M.D.

Marni Falk, M.D.

Assistant Professor of Pediatrics

Division of Human Genetics

Children’s Hospital of Pennsylvania

University of Pennsylvania Perelman School of Medicine

Philadelphia, P.A.


Jonathan Kimmelman, Ph.D.

Associate Professor in Biomedical Ethics

Biomedical Ethics Unit

McGill University

Montreal, Canada

Anna C. Mastroianni, J.D., M.P.H.


University of Washington School of Law

Seattle, W.A.

Vamsi K. Mootha, M.D.2


Howard Hughes Medical Institute, and

Professor of Systems Biology and Medicine

Harvard Medical School

Boston, M.A.

Laurie Strongin

Founder and Executive Director

Hope for Henry Foundation

Washington, D.C.

Keith Wailoo, Ph.D.1

Townsend Martin Professor of History and Public Affairs

Department of History

Woodrow Wilson School of Public and International Affairs

Princeton University

Princeton, N.J.



Anne B. Claiborne J.D., M.P.H.

Responsible Staff Officer

Rebecca English, M.P.H.

Program Officer

Morgan Stathem, M.S.

Associate Program Officer

Michael Berrios

Senior Program Assistant


1Member, National Academy of Medicine

2Member, National Academy of Sciences

Can StemCells Inc. Cheat Death?

stemcellsinc-logoCalifornia stem cell biotech, StemCells, Inc. ($STEM), has really struggled for years and its stock is now flatlining raising the question: can it cheat death?

Will 2016 be the year that STEM turns things around?

So far this year hasn’t started off that well. You can see the graph at right from today on the STEM stock price.

Ron Leuty over at San Francisco Business Times reported a couple of weeks ago on a juicy severance package for departing STEM CEO Marty McGlynn despite struggles at the company and layoffs.STEMcell stock

I sincerely hope that STEM can bounce back and that its clinical trial work including on spinal cord injury turns out to be successful, but the odds seem to grow longer financially looking to the future barring some big splash of good news soon.

What do you guys think of STEM’s future?

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.

Haruko Obokata (小保方 晴子) book is a #1 best seller

Obokata book AmazonHaruko Obokata wrote a book about the STAP cell scandal telling it from her perspective.

Fom all accounts I’ve seen, she has sought in her writings to shift the blame away from herself. She even claimed she was framed by other scientists. To say I’m skeptical would be an understatement.

Will this book have any impact? Many scientists thought it would make a splash and then fade away.

Not so, at least so far.

At present Obokata’s book is the #1 best-selling book on all of Amazon Japan, which is a huge deal. A hat tip to Dr. Robert Geller for the heads up on this.

Clearly in Japan a very large number of people are buying this book, which both raises the possibility that it could be influential on public opinion there and that she could profit handsomely from its sales. Her publisher apparently has given her a $50,000 USD equivalent advance, but if the book sells beyond expectations she could get even more.

Might it be translated into English?

Do people believe the claims in the book? The reviews on Amazon Japan are very mixed with many one-star reviews with harsh words and overall not a great rating, but there are five-star reviews too.