Stem Cells: Shaping the Future of Regenerative Medicine

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Wednesday, February 3

7:00 AM Registration and Morning Coffee

8:10 When Drug Research is Personal

John F. Crowley, Founder, Novazyme Pharmaceuticals, Inc.

Mr. Crowley’s emotion-packed presentation will focus on his personal struggle to find a cure for Pompe disease, a rare and fatal illness that is caused by a defective or missing enzyme. Pompe disease affects fewer than 10,000 people world-wide, including Mr. Crowley’s two small children. Mr. Crowley, a Harvard educated businessman, created and built a pharmaceutical company devoted expressly to finding a cure for the disease. He will detail his journey through the labyrinth of scientific and business fronts, which lead up to a first-round clinical trial.

8:55 Technology, Aging, and the Brain

Gary W. Small, M.D., Parlow-Solomon Professor on Aging, Professor of Psychiatry & Biobehavioral Sciences, Director, UCLA Center on Aging, Director, Memory & Aging Research Center, Director, Geriatric Psychiatry Division, Semel Institute for Neuroscience & Human Behavior, David Geffen School of Medicine at UCLA

New neuroimaging and other technologies are teaching us about how the brain ages and what we can do about it. Although memory declines as we age, medical and nonpharmacological strategies may protect brain health and improve memory performance. At the same time, innovation in digital technology is not only changing the way we live and communicate, it appears to be altering how our brains function. As a consequence of this high-tech stimulation, we are witnessing the beginning of a new form of the generation gap – a brain gap dividing younger digital natives, immersed in the technology early in life, from older digital immigrants, who adapt to the new technology more reluctantly. This lecture will describe this current pivotal point in brain evolution and how we can harness the new technology and lifestyle choices to improve memory and brain function so we can live better and longer.

9:40 Grand Opening Refreshment Break in the Exhibit Hall

ADVANCING REGENERATIVE MEDICINE

Addressing the numerous topics central to the theme of regenerative medicine, this meeting assembles prominent researchers, clinicians, businessmen, and regulators who not only are at the cutting edge of their respective fields, but also represent wide areas of expertise. The cross-fertilization of the information presented in the area of stem cell research engineers brainstorming and provides a forum for discussion to enable the rapidly expanding therapeutic potential of regenerative medicine.

11:00 Chairperson’s Remarks

Dawn Driscoll, MBA, Ph.D., Principal, DCi Biotech

11:10 Keynote Presentation

Stem Cells: Moving from Discovery Towards the Clinic

Alan Trounson, Ph.D., President, California Institute of Regenerative Medicine

11:55 Featured Presentation

Diabetes Under Control

Andrew Rakeman, Ph.D., Scientific Program Manager, Regeneration, Juvenile Diabetes Research Foundation

Diabetes treatments necessitate the replacement or regeneration of pancreatic beta cells to improve glucose control and avoid serious side effects. Replacement of beta cells has attracted considerable attention with the use of cadaveric islets, pig islets and a variety of adult stem cells. It appears that the best source to date is cells obtained from human embryonic stem cells and there is hope that iPS cells may one day also be an appropriate source.
More recently, interest has focused on regenerating the pancreas as a result of access to human progenitors as well as a better understanding of cell proliferation and neogenesis will accelerate the development of beta cell regenerative medicines.

12:40 PM Luncheon Presentation Sponsored by

Personalized Medicine: The Missing Pieces
Jim Karkanias, Senior Director of Applied Research & Technology, Microsoft Health Solutions Group
Discoveries that will make personalized medicine a reality depend on leveraging the “open universe” of life sciences data. To assist investigators with ad hoc questions, hypothesis generation, and validation, investigators must be able to describe and verify systems about the life science universe. This session will introduce a strongly typed repository of linked data that makes it possible to conceive and deliver game-changing therapies.
Objectives of this session are to learn:

1. Benefits of the free and secure flow of information between consumers, physicians and scientists

2. How to connect silos throughout the ecosystem and design processes to match true outcomes

3. How one information superhighway can revolutionize personalized medicine

1:45 Dessert in the Exhibit Hall

CONSIDERATIONS FOR ADVANCING
REGENERATIVE MEDICINE INTO THE CLINIC

2:15 Chairperson’s Remarks

Dawn Driscoll, MBA, Ph.D., Principal, DCi Biotech

2:20 hESC-Derived Oligodendrocyte Progenitor Cells-GRNOPC1 for Acute Spinal Cord Injury

Edward Wirth III, M.D., Ph.D., Medical Director, Geron Corporation

2:50 From Tissue Engineering to Regenerative Medicine: An Evolution in Understanding

Damien Bates, M.D., Ph.D., Chief Medical Officer, Organogenesis, Inc.

3:20 MSC-Derived SB623 Cells for Stable Stroke

Casey Case, Ph.D., Vice President of Research, SanBio, Inc.

SB623 cells are derived from bone marrow stromal cells (MSCs). They have shown great potential in models of CNS regeneration. The cells are used allogeneically and implanted directly at the site of injury. Our first clinical application will be in stable ischemic stroke patients. Models of efficacy and safety will be discussed as will issues pertaining to manufacturing and clinical plans.

3:50 RNAi tools for Stem Cell Research
Katie Jansen Spayd, Ph.D., Research Scientist, Thermo Fisher Scientific
RNA interference (RNAi) has become a powerful tool in functional genomics. Recent advances in the design of novel siRNA’s and of vector-based synthetic RNAi such as shRNAmir constructs expressed from viral vectors have overcome many of the past limitations of siRNA. In this presentation we will describe how a combination of different strategies now ensures high potency and specificity of chemically synthesized siRNA’s and review available options for RNAi based methodologies for use in difficult to transfect stem cells or to afford long term stable knockdown of the desired target in these cell types. Whilst most RNAi technology has focused on the use of endogenous short synthetic RNA, RNAi can also be exogenous and these are known as microRNA’s (miRNAs). miRNA’s operate as key regulators of gene expression by repressing mRNA translation or targeting transcripts for degradation. Recent reports on the role of miRNAs in various stem cell cellular responses such as differentiation, proliferation and apoptosis suggest novel opportunities to employ synthetic, stabilized or viral-based miRNA inhibitors and/or mimics as molecular tools in a variety of in vitro or in vivo stem cell applications. These applications include miRNA expression profiling for prognostic or diagnostic indicators of normal or disease states or screening together with synthetic miRNA inhibitors and mimics to elucidate key regulators of specific biological pathways. Therefore, in this talk we’ll review several independent examples of siRNA, shRNA and miRNA-based inhibitor and mimic screens to highlight the utility of these tools in basic stem cell biology and drug discovery platforms.