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  Newsletter Issue 4, 9 September 2015

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STEM CELL SOCIETY SYMPOSIUM 2015

"Opportunities & Challenges in Stem Cell Based Medicine"

17 -18 November 2015

Matrix Building
30 Biopolis Street, Singapore 138671

 

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Dear COLLEAGUES,

 

In this issue of the Newsletter we also would like to present one of our keynote speakers, John RASKO from the Centenary Institute, University of Sydney.

John is an Australian pioneer in the application of adult stem cells and genetic therapy and his keynote lecture titled "The Cell and Gene Therapy Soup: Disentangling Hope, Realism and Quakery" will certainly discuss important issues with regards to cell & gene therapy. Don't miss what a leading expert in the field has to share with us during the symposium.

The second featured speaker for today is Ernest ARENAS from the Karolinska Institute who will talk about the importance of Developmental Biology in generating differentiated cell types from stem cells using Parkinson's disease and dopaminergic neurons as an example.

 

Future deadlines to note:

1) Poster Abstract submission: 9 October 2015

2) Online registration closes: 30 October 2015

 

A tentative programme can be accessed HERE.

To learn more about the symposium, follow this LINK.

To register, click HERE.

In case you have any queries, contact us HERE.

The Organizing Committee "Stem Cell Society Singapore Symposium 2015"

 

 

 

 

 

 

 

 

 

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Featured Speakers  

John RASKO - KEYNOTE SPEAKER

Centenary Institute, University of Sydney, Australia

 
Abstract  
The Cell and Gene Therapy Soup: Disentangling Hope, Realism and Quakery

 

It is an exciting time for genetic and cellular therapies. Since 1989 over 1500 Phase I/II studies of direct in vivo and cell-mediated gene therapy in diverse diseases have been completed. Substantial evidence of improved clinical outcomes has been shown in haemophilia B, immune deficiencies, haemoglobinopathies, immunotherapies and blindness. In the field of cellular therapeutics, applications have expanded beyond the foundation in autologous and allogeneic hemopoietic cell transplantation to mesenchymal and other adult cell therapy trials. With over a decade of experience in AAV-mediated gene therapy for haemophilia with biopharma, our recent clinical focus has been on beta thalassemia major. Transplantation of patients with thalassaemia using autologous CD34+ cells transduced with a replication-defective, self-inactivating LentiGlobin BB305

Indeed, if pluripotent cells can be differentiated ex vivo to recreate and repair mature human tissues and organs then regenerative medicine will become a reality. Our international collaboration in dissecting molecular mechanisms of reprogramming has uncovered a new category of “F-class” cells. However embryonic stem cells have been mired in controversy and clinical development has been forestalled. Medical and, in particular, stem cell tourism has become a billion dollar industry with increasing examples of false claims. The administration of unregulated, undefined, untested or unsafe ‘stem’ cells place the field at a challenging crossroad.

 

Biography Lab webpage PubMed

 

John Rasko is an Australian pioneer in the application of adult stem cells and genetic therapy. He directs the Department of Cell and Molecular Therapies at Royal Prince Alfred Hospital and heads the Gene and Stem Cell Therapy Program at the Centenary Institute, University of Sydney. John Rasko is a clinical hematologist, pathologist and scientist with a productive track record in gene and stem cell therapy, experimental haematology and molecular biology. In over 150 publications he has contributed to the understanding of stem cells and haemopoiesis, gene transfer technologies, oncogenesis, human aminoacidurias and non-coding RNAs. He serves on Hospital, state and national bodies including Chair of GTTAC, Office of the Gene Technology Regulator – responsible for regulating all genetically-modified organisms in Australia - and Chair of the Advisory Committee on Biologicals, Therapeutic Goods Administration.

Contributions to scientific organisations include co-founding (2000) and past-President (2003-5) of the Australasian Gene Therapy Society; Vice President, International Society for Cellular Therapy (2008-12) and founder (2009) ISCT-Australia; Scientific Advisory Committees and Board member for philanthropic foundations; and several Ethics Committees. He is a founding Fellow of the Australian Academy of Health and Medical Sciences. He is the recipient of national (RCPA, RACP, ASBMB) and international awards in recognition of his commitment to excellence in medical research, including appointment as an Officer of the Order of Australia.

Interview Read full interview as PDF here  

Could you elaborate on main regulatory issues concerning clinical cell and gene therapy in Australia?

I should firstly stress that I'm not a spokesperson for any Australian regulatory agency. My role over many years is to serve as Chair for two separate Advisory Committees, the Gene Technology Technical Advisory Committee (Office of the Gene Technology Regulator) and the Advisory Committee on Biologicals (Therapeutics Goods Adminsitration). We do have a tremendously sophisticated regulatory environment for the development of clinical cell and gene therapeutics in Australia. In the year 2000, the Gene Technology Act was promulgated, ....cont'd.

How does the regulatory environment in Australia impact on the progress of stem cell therapies?

There are a number of significant challenges facing the regulatory environment in Australia. They include how we deal with new gene modification technologies that in some cases may leave no evidence that they ever occurred in the first place. I'm thinking in particular of technologies that involve the CRISPR/Cas9 system and other recombinant technologies. There is a reignited debate occurring within academia and in some public forums ...cont'd

What do you think about adult and pluripotent stem cells as a discovery and research tool for the future?

There is a lot of the excitement in Japan at their new regulatory environment. Certainly, Japan sees itself as a true centre for developing induced pluripotent stem cell-based technologies owing to the fact that Takahashi and Yamanaka have sustained their extraordinary output over the last decade. I think despite the excitement, we have to take a realistic look at what’s available here and now. And that really emphasises the importance of somatic cell therapies as opposed to embryonic or iPS cell technologies. The simple fact of the matter is that....cont'd

What projects are you working on currently?

In our Department of Cell & Molecular Therapies we are currently active in the BlueBird Bio sponsored multinational study in β-Thalassemia Major, which is testing transplantation of autologous hematopoietic stem cells transduced ex-vivo with a lentiviral vector.

Over the last decade we have also maintained a consistent involvement with the clinical development of AAV-mediated gene therapy for haemophilia, now sponsored by Spark Therapeutics. Outside of the cell manufacturing obligations at the Royal Prince Alfred Hospital, a vibrant Gene and Stem Cell Therapy research programme at the Centenary Institute, University of Sydney, is focused on stem cell biology,....cont'd

Which scientist/clinician has made the biggest impact in your field and why?

My PhD supervisor Professor Donald Metcalf AC lived an incredibly productive life in medical science until his recent passing in late 2014. His studies on the colony stimulating factors directly led to the use of granulocyte-colony stimulating factor for clinical use in 1991. To date over 20 million people have been treated with this white cell growth factor to boost the immune system in people treated for cancer or with immune deficiencies, and to facilitate blood stem cell transplantation. Don truly inspired my own research career - sometimes by a grudging encouragement that was very hard to earn. But more importantly he conveyed his passion for research by daily example and ritual after fifty years of consistent hard work that didn’t stop until his last few days. He remained a dedicated bench scientist throughout and yet still found time to mentor some of the world’s most successful haematology researchers. That legacy of dedication and mentoring has never left me.

What would you tell a student asking for advice whether to pick up a career in the stem cell/gene therapy field?

Do so! In my opinion the stem cell/gene therapy field is a true growth area in biomedical research that will be sustained for many years to come. Especially now since some mighty biopharmaceutical forces have taken interest in the last few years. Whether you approach it from a basic/translational research angle or pursue a more commercially-oriented manufacturing career, my advice is to always keep an eye to the horizon for the most promising opportunities. Always seek to work with the best people you can find!.

 

 

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Ernest ARENAS

Karolinska Institute, Sweden

 

 
Abstract  
Improving Stem Cell-based Therapies for Parkinson’s Disease: Learning from Development  

Our work focuses on understanding midbrain dopaminergic (mDA) neuron development and improving regenerative medicine for Parkinson’s disease (PD). We previously found that different members of the Wnt family of morphogens regulate mDA neuron development in a cooperative manner and that endogenous brain oxysterols activate Liver X receptors to control mDA neurogenesis and survival.

Recent transcriptomic analysis has allowed us to identify transcription factors and extracellular matrix components enriched in the developing midbrain, which contribute to mDA neuron development. We think that these factors can lead to improvements in current mDA differentiation protocols and in stem cell-based therapies for PD.

Biography Lab webpage PubMed  

Ernest Arenas is an MD, PhD from Barcelona, who is currently Full Professor in Stem Cell Neurobiology at the Karolinska Institutet in Stockholm (Sweden). He is chairman of the Department of Medical Biochemistry and Biophysics and member of the Center for Developmental Biology and Regenerative Medicine. Professor Arenas has received awards from the Swedish Royal Academy of Sciences (Researcher position) and from the Swedish Foundation for Strategic Research (INGVAR award). He is an elected Member of the Nobel Assembly in Medicine or Physiology, since 2010. Professor Arenas’ research focuses on understanding the development of midbrain dopaminergic neurons and on improving regenerative medicine strategies for the treatment of Parkinson’s disease. His research has contributed to understand the mechanism of action of neurotrophic factors, Wnt signaling and nuclear receptors, such as liver X receptors, as well as their function on midbrain dopaminergic neurons, leading to subsequent improvements in stem cell differentiation protocols. 

His current main focus of research is the regulation midbrain dopaminergic neurogenesis and the specification of substantia nigra neurons. Professor Arenas has more than 125 international original articles in peer-reviewed journals, several in prestigious journals (Nature and Cell press), has supervised 15 PhD students and 31 postdoctorals, of which 13 are currently in his lab and 18 are independent group leaders. His research has been supported by international funding organizations such as network projects from the European Union since 1996. Professor Arenas has served as consultant for the pharmaceutical industry in the area of stem cells and neurological disease and is currently serving in several external scientific advisory boards for different Stem Cell Centers.

© 2015 Stem Cell Society Singapore