Knoepfler Lab Stem Cell Blog | Building innovative …

In choices of fictional books to read I gravitate toward sci-fi and fantasy. For example,in late 2014 and through the first few months of this year I read all the Game of Thrones books.

The first three of those were the best and I thought definitely better than the TV show, although its pretty good too even if a bit violent for my taste.

My reading lately has been leaning more towardbooks about human transformation of various kinds including non-fictional works.

This reading has come to constitute, I realized, a somewhat electric mix with the common thread of science (and in particular genetics and biology) fantasy becoming reality. I recommend these as thought-provoking reads.

See any patterns there?

This reading is in part inspired by research I was doing for my own new book GMO Sapiens, hopefully coming out in December. By the way GMO Sapiens is a mashup (or portmanteau) of Homo sapiens and GMO.

It is focused on human genetic modification and the very real possibility of designer babies on the horizon.

In the coming weeks and months I will be posting excerpts from the book. Stay tuned.

Theres a growing stream of stem cell good news of late.

Stem cell biotech, TiGenix, reported encouraging Phase 3 allogeneicresultson stem cells. Abig milestone. I cant wait to see the actual data. Its product, Cx601, has so far beensafe and effective forperianal fistulas inCrohns disease. See more from Alexey on this.

CIRM has fundedthe largest public stem cell bank in the world. Want to make a withdrawal?

Asterias reported some great news. Its AST OPC-1 product showed some signs of encouraging efficacy in the first three patients treated. They are hoping to expand the trial.

In about two weeks on September 16th, the first winner of the new Ogawa-Yamanaka Stem Cell Prize will be announced at Gladstone Institutes. This should be a very exciting event honoring a top stem cell translational innovator in the area of cellular reprogramming, who will also received a cash prize of $150,000. According to the organizers, The Prize was established through a generous gift from Hiro Ogawa to honor the memory of Betty Ogawa, who passed away in May 2014. It continues the philanthropic legacy she shared with her husband of 46 years. The prize also recognizesShinya Yamanaka too of course.

Any guesses on who will win? Let us knowin the comments. Any kind of recognition of achievement and innovation in stem cells is wonderful.

On that note, Im also starting to think about candidates for my own stem cell prize, the annual Stem Cell Person of the Year Award.Its not to the same scale with a $2,000 prize, but I fund it out of my own pocket. The focus is different too. My award goes to the person who has had the single strongest, most innovative impact in the stem cell world for a given year and has taken risks either scientifically or in the area of advocacy. Im looking for outside-the-box thinkers and doers.

Past winners have included super patient advocateRoman Reed,scientist and politicianElena Cattaneo(who went on to win the ISSCR Public Service Award), and this past year, pioneering stem cell translational scientistMasayo Takahashi. Dr. Takahashi might be a good bet to win the Ogawa-Yamanaka Prize this year.

Who should be nominated this year for the 2015 Stem Cell Person of the Year Award?

Its not too soon to let me know your thoughts even though nominations dont officially open quite yet. Last year we had more than two-dozen amazing nominees. The process includes an Internet vote to choose finalists and then I pick the winner from those, sometimes consulting privately with global stem cell scholars.

For the past few years post-publication peer review (PPPR) has grown in influence and one particular website, PubPeer, has become the primary go-to place specifically for bluntPPPR. The kind that happens in journal clubs in labs across the world. Comments on PubPeer have led to numerous serious corrections and retractions of flawed articles. Im most familiar with its role in the STAP cell case.

Both the founders of and most of the commenters on PubPeer have remained anonymous.

That is until today.

The founders of PubPeer have publicly identified themselves in a blog post and formed a new non-profit organization, The PubPeer Foundation.

PubPeer founders include Brandon Stell, George Smith and Richard Smith. Also with the founders on the PubPeer Foundation Board of Directors will be Boris Barbour and Gabor Brasnjo.

Who are these guys?

RetractionWatch (big HT to them) has an interesting interview with PubPeer founder Stell. I highly recommend reading it. Jennifer Couzin-Frankel over at Science also has a nice piece on this development.

Stell is a neuroscientist and Co-Team Leader at the Brain Physiology Lab in Paris, the source of his picture above.

I was trying to learn more about the other two founders, George Smith and Richard Smith. From Couzin-Frankels piece it appears that the brothers want to remain relatively out of the limelight, and their very common names may very well aid them in that. Couzin-Frankel does write that Richard was a grad student who briefly worked in Stells lab and George is a web developer.

As to the Foundation, RetractionWatch has a helpful quote from Stell on looking ahead:

What role do you hope PubPeer plays moving forward? What plans do you have for the Foundation?

We hope that the PubPeer Foundation will provide us with more opportunities to develop the site in ways that will help grow the community of post-publication peer reviewers and further encourage quality science.As more of us scientists become accustomed to commenting on papers, and as that becomes more of a part of the overall scientific process, I think well be able to finally up-end the backwards reward structure that is currently in place in science. Hopefully we can get to a point where the data are much more important to a scientists career than the journal that published them.

To form the PubPeer Foundation, the leaders could not remain anonymous. It seems like a good thing that the founders of PubPeer have identified themselves. They deserve a lot of credit for having had a transformative impact on how science corrects itself. Theyve also faced tough situations such as being sued by Fazlul Sarkar, a case that is still working its way through the legal system. Sarkar wants to know the identity of some anonymous PubPeer commenters. I predict that the PubPeer Foundation will now receive substantial financial support, which in part can be used to get legal assistancefor possible future challenges.

PubPeer has grown quickly, now has a striking following with around 300,000 pageviews/month and contains 35,000 total comments.

I recently chatted with Sean Morrison, current President of ISSCR, on his goals for the Society, where the stem cell field stands today, top challenges, and the future.

What are your goals for your tenure as President of ISSCR?

SM:ISSCR is the international voice for research in the scientific community. Theres been less effort though amongst policy makers and the general public. I want to expand the reach beyond just the scientists. ISSCR will be building its capacity to participate in stem cell policy issues worldwide and also its capacity to communicate with the public.

Whats the plan to make this happen?

SM: A range of things. Beefing up ISSCR communications programs. Blogging, twitter and other efforts.

What are the main challenges & opportunities that ISSCR faces now?

SM:ISSCR has had steady and remarkable growth. It has grown and its scope has grown too. Managing the growth is a challenge. Prioritizing future opportunities. How can we have the most impact for the membership? How can we grow that impact? The annual meeting continues to grow. International ISSCR symposia have grown. Publishing activities have expanded including withCell Stem Cell and nowStem Cell Reports. We continue to expand those kinds of activities. There is a set of opportunities related to going beyond the science. Our mission is to improve human health through stem cells. We cant do that solely by meetings and publishing. Those are critical core activities, but theres more that we can do such as in industry. Reaching out to those stakeholders. Expanding the activities of the Global Advisory Council (philanthropists, Susan Lim, and Deepak), stem cell policy, and communication.

How can ISSCR bring in more industry people?

SM: I do think more involvement of industry is important. How best can we do that? I attended the Industry Committee meeting at ISSCR meeting in Stockholm, where we had that conversation. I really do hope that we can increase the attractiveness and value provided by the annual meeting to people in industry. We want people talking about exciting unpublished data. One difficulty comes up though in this regard: who from industry is willing to talk about unpublished data? Some people have shown up in the past and are not willing to talk about data. The program committee needs to address that.

Whats your view on the evolution of the IPSC subfield?

SM: Im excited about stem cells generally. We need all kinds of stem cell research to move forward. Historically weve not been very good at predicting which cell type will work. Im very excited about somatic stem cell research and pluripotent stem cells, both embryonic and IPSC. Look at the things going on at Shinyas institute. Theyve been bold at diverse ways that they can have impact. With each year that goes by, the more plausible scenarios arise for possible therapies using pluripotent stem cells.

The pioneering IPSC trial in Japan was put on hold. How serious is that?

SM: If youve been culturing cells, some of the time theres going to be mutations. The fact that they found the mutations says that the process that they have in place worked. Its not uncommon in clinical trials for things to go on hold. Its not unique to stem cell trials or IPSCs. I have every expectation that theyll get that back on track.

What are you most excited about with your own research?

SM: Were doing a lot of work to characterize the HSC niche. Weve now identified the cells that are sources of the key factors for stem cell maintenance. In many ways the hematopoietic system is a paradigm. This will allow us to understand at a single cell level how the niche works and look for novel growth factors. Each time we identify one of those it has the potential to provide new tools.

What do you see as the most important and exciting stem cell development or trend of 2015 so far?

SM: Were at an inflection point in terms of stem cell therapies moving into high-quality clinical trials. In 2008 when we were fighting the public policy battles about where the line should be drawn on ESC research. Also there were people making claims about difficult problems. I was skeptical at the time. I felt that a lot of the problems could be too biologically complicated for cell therapies to work. Yet the science has surged forward much more quickly. Theres really exciting preclinical data and actual clinical trials that are about to start or have started. Spinal cord. Macular degeneration. And others. Some people have spent 10-20 years trying to understand the biology. Parkinsons with Lorenz Studer. Cell therapy for heart disease with Chuck Murry. Its important for the general public to understand the timeframe and that some will fail. In the past some were just squirting cells into tissuessort of like buying a lottery ticket. People now understand the biology better. Now we have a rational chance of success.

The debate that is shaping up for CIRM is also very important now. Stem cell research is already delivering and theres opportunity looking ahead. More funding for CIRM makes sense and it would be a major setback to not do that, especially with declining federal funding. One thing that will distinguish the winning states from the losing states is who has the vision to keep the biomedical research enterprise going at the state level. The conversation changed with CIRMs birth. It became, How do we keep up with California?

The spread of stem cell clinics selling non-FDA approved offerings in the US has accelerated. What should ISSCR and individual stem cell scientists be doing to address this growing problem?

SM: Theres a lot that youve done and we appreciate that. Theres also a lot that ISSCR has done. We have our A CloserLook website. ISSCR has spoken out more on this topic than any other topic relevant to the general public. Although stem cell research has enormous promiseand this is the most exciting time that weve ever hadunfortunately most people in the general public dont understand how long it takes to go from the idea or proof of principle in a mouse to do it in humans. Sadly there are fraudulent people out there that are preying on patients. These are at best unproven and in many cases not even plausible therapies.

Where do you see the stem cell field in 5-10 years?

SM:We should be excited, but Im always wary of these kinds of predictions. If we look over the last 10 years theres been a lot of twists in the plot. Theres been both good news and bad news. Some things have surged forward more quickly and some things that we are most excited about now werent on our radar screens then. We dont really know where were going to be. But Im very optimistic. The thing that we have to remember that we always forget, even when we identify an idea that works, it takes a lot of years to get that to a patient. Look how long it took for bone marrow transplant to develop. We now talk about bone marrow transplant as an example but it took 14 years. We should bear in mind that even if some of the things now in clinical trials are correct, it could take years to develop them in a safe and efficient way.

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Knoepfler Lab Stem Cell Blog | Building innovative ...