Highlights of ISSCR2017 – A Biomedical Engineer’s Perspective

By Agnes SoosAgnes Soos

Amid the hustle and bustle of downtown Boston, nearly four thousand researchers and exhibitors gathered for the 15th Annual ISSCR Meeting. With presentations from over thirty plenary lectures, and dozens of others featured in concurrent sessions and the daily Innovation Showcases, there definitely was no shortage of exciting research to discover and discuss. There was a record number of exhibitors this year with over 150 companies set up in Exhibit Hall. Added to this, the always-busy Meet-Up Hubs, the job boards peppered with posted offerings, and the popular Meet-the-Experts luncheons and Junior Investigator Career Panel (both with full wait-lists) offered an array of networking opportunities that definitely did not disappoint!ISSCR2017-book&badge,closeup

Though the program broadly covered stem cells in development, disease, and their applications to human health, iPSC-related work featured prominently throughout the Meeting. Central to this focus on iPSC research – and quite popular among the attendees – was the plenary talk by Shinya Yamanaka. His lecture discussed the two major medical applications of iPSCs: cell therapy and disease model/drug development. Under the cell therapy topic, he relayed the high costs associated with autologous treatments and outlined the research that has gone into identifying HLA homozygous “super” donors to overcome the prohibitive nature of allogeneic therapies. Though a first transplant was successfully performed in March of this year, this work still faces some challenges. Namely, facility-dependent handling practices appear to impact the occurrence of mutations within cancer-driving genes of the iPSC clones, necessitating further study and perhaps tighter control of how these cultures are handled. On the topic of disease modeling and drug development, Yamanaka outlined early successes in using iPSCs to gain a better understanding of Fibrodysplasia Ossificans Progressiva and the effective use of patient-derived cultures to conduct in vitro drug screening.

Organoids and organogenesis was a hot topic at this year’s Meeting with dedicated plenary and concurrent sessions offering an array of excellent talks. Juergen Knoblich’s plenary lecture presented the advantages offered by 3D organoid cultures – namely, their ability to recapitulate features of brain development and disease which cannot be easily studied using animal model systems. For example, cerebral organoids have been developed using patient-specific iPSCs to model microcephaly. Knoblich also discussed some of the advantages of introducing bioengineered matrices to manipulate organoids, demonstrating improved organoid generation with specific scaffold materials. Although organoid ‘mini-brains’ to study neural development and disease were the topic of several talks, other areas of organoid research included intestinal epithelium (‘mini guts’), pancreas, kidney, liver, and lung.ISSCR2017-Overlooking (part of) Exhibit Hall

Other highlights included talks from the recipients of the McEwen Award for Innovation, Elaine Fuchs, and the Dr. Susan Lim Award for Outstanding Young Investigator, Jayaraj Rajagopal. Fuchs expertly summarized four decades of research on skin stem cells in a mere 20 minutes, covering the current understanding of stem cell activity and function in normal tissue, with aging, and in cancer, using the skin as a model system, with focus on cells within the hair follicle niche. Rajagopal lectured on the topic of airway epithelium and the interactions of three constituent cell types – a stem cell, progenitor cell, and post-mitotic differentiated cell. He presented the value of this model in studying regeneration and outlined the complexity of cellular interactions in this relatively simple cellular ensemble. Both of these talks were presented as part of the plenary session on Tissue Regeneration and Homeostasis.

Unsurprisingly, lectures in all sessions presented some very exciting science. Topics covered in the other plenary talks included: Chromatin and RNA Biology in Stem Cells, Stem Cells and Cancer, Stem Cells and Stress, Senescence, and Aging, and the Frontiers of Cell Therapy.

In addition to the above, perhaps the most memorable talk of the Meeting was the address by Sanford Greenberg on his quest to end blindness. Greenberg discussed his own experiences, relaying the struggles he faced when he become blind at 19 years of age. He outlined the goals of the Greenberg Prize, a $3 million award (in gold), which has the intent of rallying the international research community to focus efforts towards ending blindness by year 2020.

Developing effective treatments – or perhaps even cures – through clinical applications of some of the research presented at the Meeting does appear promising. This was exemplified by the work presented in the final plenary session by Masayo Takahashi on the successful use of iPSC for retinal cell therapy. However, although the results of this work are cause for excitement, Takahashi emphasized the importance of being cautious in our optimism. Though there may be progress, she is often faced with the challenge of being unable to accommodate all patients seeking treatment. Thus, she emphasized, although we must promote our successes, we should avoid placing too much emphasis on champion cases and avoid hype, so that together we may see healthy progress within the stem cell field.

New Yamanaka interview gives key insights into future of IPS cells

Shinya yamanaka

Wikipedia photo

Where is the field of IPS cells going and how will this impact the overall field of stem cell-based regenerative medicine?

Nobel Laureate Shinya Yamanaka, the discoverer of IPS cells, gave a really interesting recent interview to Nikkei that provides some fascinating insights into the future of this exciting technology that is now more than a decade old.

For simplicity I have indicated top highlights from the Yamanaka interview below as bullet points.

  • More IPS cell trials are on track to start as soon as 2018 in Japan.
  • Yamanaka said that trials for Parkinson’s, Spinal Cord Injury, and Heart Disease are amongst the planned IPS cell trials in Japan.
  • There are also plans for clinical studies on cancer and kidney disease, perhaps further down the road such as 2019-2020?
  • Immune rejection and cancer risks must still be evaluated, he said.
  • There are likely to be important differences in the new studies versus use in the eye.
  • CiRA has started working with Takara on QC of IPS cells and products.
  • Their main focus for all these trials still seems on allogeneic use from IPS cell banks.

It will be interesting to see how trials in Japan develop versus those in other countries such as here in the US where I know of planned autologous IPS cell clinic efforts.

10 provocative stem cell questions: what are your answers?

heart stem cellsThere’s always debate about stem cells and regenerative medicine, right? There are very strong opinions in our field. Here is a list of 10 current, provocative questions. I have my own ideas about the answers to these. Weigh in with comments with your answers.

I’m going to do a follow up post with some discussion of the questions. I may do another follow up with the top 10 policy questions in our arena.

Here’s today’s list in no particular order. Continue reading

Top 10 reasons for optimism on Stem Cell Awareness Day 2016

Happy Stem Cell Day!

It is a tumultuous time for the stem cell and regenerative medicine fields, but despite this there are concrete reasons for optimism on this Stem Cell Awareness Day. I’ve listed my top 10 below. What else gives you a sense of optimism? You can also check out CIRM’s nifty stem cell awareness day page too, from which I borrowed the below image.stem-cell-awareness-day-2016

  1. More trials = road to progress. There are more real, robust clinical trials than ever and they are progressing past the early phases in some cases. The trials are piling up and while not all will succeed, some will. Keep an eye open for the for-profit, non-FDA-approved ones and steer people away from those.
  2. We are seeing a flow of clinical trial data too pointing to encouraging outcomes, but also to challenges to overcome (witness the preclinical study on IPSC for heart attacks that found efficacy but also arrhythmias). With that kind of awareness such hurdles can be overcome in many cases as the work progresses.
  3. The FDA held public meetings on stem cells. We can grouch about certain things about these meetings and we can ding the FDA for various issues, but it never before has engaged with the community like this on stem cells so it’s a good thing.
  4. Stem cells firing on all cylinders: adult and pluripotent. Adult stem cell trials are building, but so now are pluripotent ones. The best way to help the most people in the long run is with all the tools (types of cells) we can utilize. The notion of “adult versus embryonic”, for instance, as some sort of cosmic battle seems out of date. We need both and also IPSC as well as other types as yet to come.
  5. The stem cell clinic problem out in the open. Never before has there been this much information and awareness out there on the problem of stem cell clinics taking advantage of vulnerable people. For instance, see my recent article with Leigh Turner and the one from John Rasko’s group. I believe awareness will translate into action for the positive.
  6. Putting the fun back in funding? NIH funding trends are looking at least slightly better overall which will help with stem cell research. CIRM is continuing its life extension and will fund many more projects in years to come. Other states are funding stem cell research too. It’s still a bad time for funding but the trend lines are at least moving the right way.
  7. Much more educational outreach on stem cells. When I started blogging about stem cells in early 2010 it was very quiet out there on the Internet in terms of those of us trying to educate a wider community in a positive manner. That’s really changed now with quite a few blogs that at least touch on stem cells and a number entirely dedicated to stem cells and regenerative medicine. This is a positive change and it means the public has more resources than ever to learn about stem cells.
  8. IPSC clinically-relevant work is looking up. It was a decade ago that IPSC cells were “born” and there were great expectations. Now 10 years later there are tangible signs that these cells will have lasting, huge impact including both from disease modeling and more recently via potential future clinical use.
  9. Stem cells meet CRISPR and…boom! Okay so everyone is nuts about CRISPR no matter what kind of cells they study including me, but CRISPR-Cas9 gene editing combined with stem cells in particular equals great potential both for new insights such as into human development and also potentially clinically through designer stem cells.
  10. Stem cell biotechs & stocks hanging in there. It has always been tough going for stem cell biotechs and that is likely to continue quite a while longer, but many are hanging in there and could surprise you down the road. Others have been acquired by pharma companies or inked collaborative deals in the last year or so. In the long run some of these companies are going to change medicine.