The Stem Cell Report with Martin Pera ISSCR

Biomedical researchers have long sought ways to repair spinal cord damage with the holy grail of the pursuit being the reconstitution of lost function. In the mid 1990’s with the successful culture of human embryonic stem cells, and about a decade later induced pluripotent stem cells (iPSCs), the field was energized with a potential new approach to replace the lost neurons and glia cells and restoring neural connections.  In the decades since that discovery some progress has been made, however many hurdles remain, including establishing a functional synaptic connection between the transplanted and host neurons which is crucial for motor function recovery. To boost therapeutic outcomes our guests tested an ex vivo gene therapy to promote synapse formation between the donor and host neurons by expressing the synthetic excitatory synapse organizer CPTX in hiPSCs-derived neural stem and progenitor cells. Tune in to learn what they discovered.

Host
Martin Pera, Editor-in-Chief, Stem Cell Reports and The Jackson Laboratory
@martinperaJAX
Guests
Hideyuki Okano, MD, PhD Keio University, Japan, Professor in the Department of Physiology and Chairman of the Graduate School of Medicine at Keio University. Professor Okano has spent decades studying neurogenesis and is currently leading a first-of-its-kind cell therapy for spinal cord injury. He has previously served as an Associate Editor for Stem Cell Reports and is a member of the Editorial Board. He is the current President of the Japanese Society of Regenerative Medicine and Vice President of the ISSCR. 
 
Yusuke Saijo, MD. Keio University, Japan, graduated from Kyorin University School of Medicine and following a two-year initial training period, he embarked on a clinical journey, working in the field of orthopedic surgery at Keio University, specializing in the spinal cord and spinal disorders. Dr. Yusuke currently works in the research laboratory led by Professors Okano and Masaya Nakamura, where his research focuses on ex vivo cell and gene therapy for spinal cord regeneration. 


Supporting Content
Human-induced pluripotent stem cell-derived neural stem/progenitor cell ex vivo gene therapy with synaptic organizer CPTX for spinal cord injury, Stem Cell Reports

About Stem Cell Reports
Stem Cell Reports is the open access, peer-reviewed  journal of the International Society for Stem Cell Research (ISSCR) for communicating basic discoveries in stem cell research, in addition to translational and clinical studies. Stem Cell Reports focuses on original research with conceptual or practical advances that are of broad interest to stem cell biologists and clinicians.
Twitter: @StemCellReports

About ISSCR
With nearly 5,000 members from 75+ countries, the International Society for Stem Cell Research (@ISSCR) is the preeminent global, cross-disciplinary, science-based organization dedicated to stem cell research and its translation to the clinic. The ISSCR mission is to promote excellence in stem cell science and applications to human health.

ISSCR Staff
Keith Alm, Chief Executive Officer
Yvonne Fisher, Managing Editor, Stem Cell Reports
Kym Kilbourne, Director of Media and Strategic Communications
Jack Mosher, Scien

Collectively, retinal degenerative disorders are a major cause of blindness worldwide. For example, one of the most common disorders is age related macular degeneration, which alone affects nearly 200 million globally. 
In humans, and other mammals, the loss of the retinal cells is an irreversible process. However, in some non-mammalian vertebrates like frogs and fish, retinal neurons can regenerate. This process is dependent upon Müller glia, which can re-enter the cell cycle and reprogram into neurogenic progenitors upon retinal injury or disease. Progress has been made in understanding the genetic program underlying these regenerative process, and proof-of-principle experiments in the adult mouse retina demonstrated that genetic programs in frog and fish can be coopted to induce neurogenesis in mammals. Our guests today have extended this research to genetically reprogram fetal or organoid-derived human Müller glia into retinal neurons. They will talk about this work, the background underlying it and its potential applications.

Host
Martin Pera, Editor-in-Chief, Stem Cell Reports and The Jackson Laboratory
@martinperaJAX
Guests
Thomas Reh is a Professor of Biological Structure, and a member of Institute for Stem Cell and Regenerative Medicine at the University of Washington. His research is focused on understanding retinal disease in disorders such as glaucoma and macular degeneration, and on developing treatments for vision loss. The Reh laboratory studies regeneration in animal models, including invertebrates like frog and fish, to understand the key genes involved in endogenous regeneration.  The ultimate goal of this research is to stimulate regeneration in the mammalian retina, and ultimately to restore sight in patients. 
Juliette Wohlschlegel is a postdoctoral scholar in the Reh laboratory. Dr. Wohlschlegel received her undergraduate degree in neuroscience from the Sorbonne Université, and her PhD from Institut de la Vision in France, where she studied inherited retinal diseases. 
Supporting Content
ASCL1 induces neurogenesis in human Müller glia, Stem Cell Reports

About Stem Cell Reports
Stem Cell Reports is the open access, peer-reviewed  journal of the International Society for Stem Cell Research (ISSCR) for communicating basic discoveries in stem cell research, in addition to translational and clinical studies. Stem Cell Reports focuses on original research with conceptual or practical advances that are of broad interest to stem cell biologists and clinicians.
Twitter: @StemCellReports

About ISSCR
With nearly 5,000 members from 75+ countries, the International Society for Stem Cell Research (@ISSCR) is the preeminent global, cross-disciplinary, science-based organization dedicated to stem cell research and its translation to the clinic. The ISSCR mission is to promote excellence in stem cell science and applications to human health.

ISSCR Staff
Keith Alm, Chief Executive Officer
Yvonne Fisher, Managing Editor, Stem Cell Reports
Kym Kilbourne, Director of Media and Strategic Communications
Jack Mosher, Scientific Advisor

Voice Work
Ben Snitkoff

Since the 1930’s research using human fetal tissue has been used in numerous scientific and medical advances that have saved millions of lives, including the development of vaccines and treatments for diseases. Despite its substantial contribution to medicine and science, significant public debate and misinformation persists surrounding the ethical use of human fetal tissue in biomedical research. 

The ISSCR, led by its Public Policy Committee, have been tireless champions and advocates for sound science policy across the globe. This includes advocacy for fetal tissue research and working to inform policymakers and the public on the vast medical applications and advances that have, and will, come from the use of HFT in biomedical research. Towards that end, the ISSCR and the Lawrence Goldstein Policy Fellows have authored a recent paper in Stem Cell Reports entitled, Human Fetal Tissue is Critical for Biomedical Research.

Host
Martin Pera, Editor-in-Chief, Stem Cell Reports and The Jackson Laboratory
@martinperaJAX

Guests
Lawrence (Larry) Goldstein, PhD, is a Distinguished Professor Emeritus, Department of Cellular and Molecular Medicine and Director Emeritus of Stem Cell Program at the University of California, San Diego. He is the namesake for the ISSCR’s Lawrence Goldstein Science Policy Fellowship which is training the next generation of scientists to impact public policy. 

Tyler Lamb, JD, is the ISSCR’s Director of Policy and leads the Society’s global policy efforts.  
Tamra Lysaght, PhD, University of Sydney, Australia, is an Associate Professor in Health Ethics.


Justin Brumbaugh, PhD, University of Colorado Boulder, USA, is an Assistant Professor in Molecular Cellular & Developmental Biology.

Supporting Materials
Drs. Brumbaugh, Lysaght, and Goldstein, along with Brian Aguado, are authors of the recently published paper, Human Fetal Tissue is Critical for Biomedical Research. 

About Stem Cell Reports
Stem Cell Reports is the Open Access journal of the International Society for Stem Cell Research (ISSCR) for communicating basic discoveries in stem cell research, in addition to translational and clinical studies. Stem Cell Reports focuses on original research with conceptual or practical advances that are of broad interest to stem cell biologists and clinicians.
Twitter: @StemCellReports

About ISSCR
With more than 4,800 members from 75+ countries, the International Society for Stem Cell Research (@ISSCR) is the preeminent global, cross-disciplinary, science-based organization dedicated to stem cell research and its translation to the clinic. The ISSCR mission is to promote excellence in stem cell science and applications to human health.

ISSCR Staff
Keith Alm, Chief Executive Officer
Yvonne Fisher, Managing Editor, Stem Cell Reports
Kym Kilbourne, Director of Media and Strategic Communications
Jack Mosher, Scientific Advisor

Voice Work
Ben Snitkoff

This November marks the 25th anniversary of the successful isolation and culture of human embryonic stem cells by Jamie Thompson.  This breakthrough was a turning point in biomedical research. This discovery provided scientists with a limitless source of human cells to understand human biology and model disease. The discovery also provided a novel pathway to develop tissues and cells that could potentially be used to provide curative diseases like diabetes and Parkinson’s, among others. To date, numerous companies are advancing human embryonic stem cell-based therapies in over 50 approved and regulated clinical trials.  
However, in parallel to the legitimate, approved clinical trials, the great promise of this field has in a sense fostered a plague of rapid and continuous growth in the marketing of unregulated and unproven treatments, via direct-to-consumer clinics across the globe. These predatory clinics offer so-called stem cell therapies to treat myriad conditions with approaches not supported by rigorous scientific evidence. During the pandemic these treatments expanded to include COVID-19, including the use of what’s being advertised as exosome therapy.
 
Guest
The guest on today’s podcast is an expert on the stem cell marketplace of unproven treatments.  Leigh Turner is the Executive Director of the University of California Irvine Bioethics Program, and a Professor in the Program in Public Health’s Department of Health, Society, and Behavior. He is also a member of UCI’s Stem Cell Research Center.  His current research addresses ethical, legal, and social issues related to stem cells and regenerative medicine products. Dr. Turner served on the task force that developed the ISSCR Guidelines for Stem Cell Research and Clinical Translation and is also a member of the ISSCR’s Ethics Committee. Dr. Turner and colleagues authored the recent paper in Stem Cell Reports entitled, Businesses Marketing Purported Stem Cell Treatments and Exosome Therapies for COVID-19: An Analysis of Direct-to-Consumer Online Advertising Claims.  

Host
Martin Pera, Editor-in-Chief, Stem Cell Reports and The Jackson Laboratory
@martinperaJAX

About Stem Cell Reports
Stem Cell Reports is the Open Access journal of the International Society for Stem Cell Research (ISSCR) for communicating basic discoveries in stem cell research, in addition to translational and clinical studies. Stem Cell Reports focuses on original research with conceptual or practical advances that are of broad interest to stem cell biologists and clinicians.
Twitter: @StemCellReports

About ISSCR
With more than 4,800 members from 75+ countries, the International Society for Stem Cell Research (@ISSCR) is the preeminent global, cross-disciplinary, science-based organization dedicated to stem cell research and its translation to the clinic. The ISSCR mission is to promote excellence in stem cell science and applications to human health.

ISSCR Staff
Keith Alm, Chief Executive Officer
Yvonne Fisher, Managing Editor, Stem Cell Reports
Kym Kilbourne, Director of Media and Strategic Communications
Jack Mosher, Scientific Advisor

Voice Work
Ben Snitkoff

Human stem cell technology has led to remarkable insights into human biology in health and disease. However, for the results and outputs from this research to be accurate, meaningful, and durable, it is important that the field have agreed upon standards that ensure reproducibility and reliability of the data. The International Society for Stem Cell Research (ISSCR) has developed a set of recommendations, including reporting criteria, for scientists in basic research laboratories. These criteria are designed to be technically and financially feasible and, when implemented, to enhance the reproducibility and rigor of stem cell research.  
The guests on this episode  recently served as the co-chairs of the ISSCR Standards Task Force that developed the “Standards for Human Stem Cell Use in Research" and will discuss the need for the standards, the challenges they faced, and how they will help advance the field.  The initiative was made possible through contributions from the Burroughs Wellcome Fund, Doris Duke Foundation, and the Simons Foundation Autism Research Initiative.


Guests
Tenneille Ludwig is a Senior Scientist and Director of the WiCell Stem Cell Bank in Madison Wisconsin. As a postdoctoral fellow, Tenneille developed the TeSR media, the first defined, feeder-independent culture media for human embryonic stem cells. She holds leadership roles in numerous international efforts to improve the standards, characterization and banking of pluripotent stem cells including serving on the International Stem Cell Initiative and International Stem Cell Banking Initiative. Currently, Tenneille serves on the ISSCR’s Manufacturing, Clinical Translation, and Industry Committee and co-chaired the ISSCR’s Standards Task Force that developed the new document, “Standards for Human Stem Cell Use in Research.”  


Peter Andrews is Professor Emeritus at the University of Sheffield in the UK. A founder of the field of human pluripotency, Peter has had a distinguished career studying embryonal carcinoma cells and human embryonic stem cells and was the first to discover that human pluripotent stem cells could develop genetic abnormalities during long term culture. He chaired the International Stem Cell Initiative, the first international effort to standardize methods for the characterization of human embryonic stem cells. Most recently, he co-chaired the ISSCR’s Standards Task Force that developed the new document, “Standards for Human Stem Cell Use in Research.”

Host
Martin Pera, Editor-in-Chief, Stem Cell Reports and The Jackson Laboratory
@martinperaJAX

Supporting Content
https://www.isscr.org/standards
https://www.isscr.org/standards-document

About ISSCR
With more than 4,600 members from 75+ countries, the International Society for Stem Cell Research (@ISSCR) is the preeminent global, cross-disciplinary, science-based organization dedicated to stem cell research and its translation to the clinic. The ISSCR mission is to promote excellence in stem cell science and applications to human health.

ISSCR Staff
Keith Alm, Chief Executive Officer
Yvonne Fisher, Managing Editor, Stem Cell Reports
Kym Kilbourne, Director of Media and Strategic Communications
Jack Mosher, Scientific Advisor

Voice Work
Ben Snitkoff

The use of organoids as an experimental system is rapidly advancing in pace and complexity. Derived from pluripotent or tissue stem cells, organoids are three-dimensional, in vitro, structures intended to model functional or developmental aspects of in vivo organs. They are also being used to model complex physiological systems in organ-on-chip devices and in assembloids, the combination of organoids from different tissues. Organoids are especially useful to model and understand aspects of human biology and pathology. This approach has led to much hope, and proof-of-principle findings, that these structures can serve as human avatars to advance the discovery of personalized therapies and serve as better models for drug discovery.  
However, the rapidity of advances and the influx of diverse scientific backgrounds also presents challenges in an immature field, notable among them the adoption of standards.  Our guests today will talk about some of those challenges, the areas when standards are critically important, and how they will help advance the field.   

Guests
Professor Melissa Little is the CEO and Executive Director of the Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Chief Scientist at the Murdoch Children’s Research Institute, and leader of the Kidney Regeneration Laboratory in Melbourne, Australia. Melissa is a former President of the ISSCR and holds an honorary position as Professor in the Department of Pediatrics, University of Melbourne. 


Kim Jensen is Professor at the University of Copenhagen in Denmark and Deputy Director at the Novo Nordisk Foundation Center for Stem Cell Medicine. He is also a Scientific Advisory Board Member of the Lund Stem Cell Center and participated in the development of the recently released ISSCR standards document. 

Host
Martin Pera, Editor-in-Chief, Stem Cell Reports and The Jackson Laboratory
@martinperaJAX

Supporting Content
Organoids are not organs: Sources of variation and misinformation in organoid biology
About Stem Cell Reports
Stem Cell Reports is the Open Access journal of the International Society for Stem Cell Research (ISSCR) for communicating basic discoveries in stem cell research, in addition to translational and clinical studies. Stem Cell Reports focuses on original research with conceptual or practical advances that are of broad interest to stem cell biologists and clinicians.
@StemCellReports

About ISSCR
With more than 4,600 members from 75+ countries, the International Society for Stem Cell Research (@ISSCR) is the preeminent global, cross-disciplinary, science-based organization dedicated to stem cell research and its translation to the clinic. The ISSCR mission is to promote excellence in stem cell science and applications to human health.

ISSCR Staff
Keith Alm, Chief Executive Officer
Yvonne Fisher, Managing Editor, Stem Cell Reports
Kym Kilbourne, Director of Media and Strategic Communications
Jack Mosher, Scientific Advisor

Voice Work
Ben Snitkoff