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Main Conference

WEDNESDAY, FEBRUARY 25

7:30 am Registration Open

8:45 Plenary Keynote Introduction 
Kathryn Lowell, Deputy Secretary, Life Sciences, California Business Transportation & Housing Agency

8:55       Plenary Keynote
Therapy Development in a Networked World
Jay M. Tenenbaum, Ph.D., Chairman and Chief Scientist, CollabRx, Inc.
A new paradigm for translational research will be described that combines the integrative and collaborative power of the Internet with personalized molecular analysis to slash the time and cost of therapy development. A key element is the creation of Health Commons, an open web-based ecosystem of researchers, clinicians, patients, pharma/biotechs, and service/technology providers that can be rapidly mobilized to develop targeted therapies for disease subclasses. This ecosystem will stimulate the same radical increase in efficiency for therapy development that ecommerce brought to business in the 1990s, ushering in a new age of collaborative, personalized medicine where every patient can afford custom therapies and discovery is driven by collectively interpreting the outcomes across all patients.

9:40 Grand Opening Refreshment Break in the Exhibit Hall

THE STEM CELL NICHE

11:00 Chairperson’s Remarks
Mark E. Levenstein, Ph.D., Senior Scientist, CDI Biosciences

11:10 Deconstructing the Hematopoietic Stem Cell Niche: Revealing the Therapeutic Potential 
Gregor B. Adams, Ph.D., Assistant Professor, Cell and Neurobiology, Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research, Keck School of Medicine, University of Southern California
The success of hematopoietic stem cell based therapies relies on the ability of the stem cells to both engraft and self renew sufficiently in the bone marrow microenvironment. Understanding and manipulating the microenvironmental niche and the intracellular signals that allow for expansion of stem cells may have therapeutic potential. We have identified key components of the hematopoietic stem cell niche and further demonstrated that these can be targeted to enhance therapies aimed at the stem cells. These results suggest that the niche may be a pharmacologic target for altering stem cell function in settings of regenerative medicine.

11:40 The Cancer Stem Cell-Vascular Niche Complex in Brain Tumor Formation
Victor Tse, Ph.D., Associate Professor, Neurosurgery, Stanford University School of Medicine 
The transition of senescence cancer stem cells to a proliferative cell mass is the most intriguing event in tumorogenesis. The formation of the neoangio-architecture is thought to play a pivotal role in this epoch. Pro-angiogenic progenitors migrate to and coalesce in forming the vascular niche to support the malignant potential of these cancer stem cells. The result is the formation of the cancer stem cells vascular niche complexes.

12:10 pm  Primary Cilia in the Regulation of Neural Progenitors and Cancer
Young-Goo Han, Ph.D., Postdoctoral Fellow, Neurological Surgery and Institute for Regeneration Medicine, University of California San Francisco
Signaling pathways from the stem cell niche control stem cell behavior and deregulation of these pathways can lead to tumor formation. Neural stem cells, like many other cell types, have a primary cilium, a hair-like appendage extending from the surface of a cell. This organelle plays a critical role in sonic hedgehog (SHH) signal reception and processing. Defective cilia are associated with diverse human disorders including mental retardation and ataxia. We found that primary cilia are essential for the expansion of embryonic neural precursors and their transition into postnatal neural progenitors. We also found that primary cilia are required for the growth of a certain brain tumor, but for another the presence of primary cilia inhibits tumor growth.

1:10 Walk & Talk Luncheon in the Exhibit Hall

CONTROLLING AND UNDERSTANDING DIFFERENTIATION

2:15 Chairperson’s Remarks
Patricia Olson, Ph.D., Director, Scientific Activities, California Institute of Regenerative Medicine

2:20 Molecular Engineering of the Stem Cell Microenvironment
David Schaffer, Ph.D., Professor, Chemical Engineering, Bioengineering, Neuroscience, University of California Berkeley
The successful integration of stem cells into therapies will hinge upon three critical steps: their expansion without differentiation, differentiation into a specific cell type or collection of cell types, and functional integration into existing tissue. Precisely controlling each of these steps will be essential to maximize their therapeutic efficacy, as well as minimize potential side effects that can occur when the cells numbers and types are not properly controlled. We combine experimental and computational approaches to understand basic mechanisms by which microenvironmental signals regulate of stem cell fate choice, including neural stem and human embryonic stem cells. Furthermore, we have applied this basic information towards the engineering of synthetic biomaterials based "artificial niches" for the controlled expansion and differentiation of stem cells, which offer significant advantages for safety and scalability.

2:50 Identification of Human Embryonic Stem Cell-Derived Blastocyst- and Epiblast-Stage Progenitors
Micha Drukker, Ph.D., Postdoctoral Scholar, Stanford Institute for Stem Cell Biology 
The potential of human embryonic stem cells (hESCs) to differentiate into any type of adult tissue makes these cells a unique model for studying early human development and at the same time, a source of cells for regenerative medicine. To address the challenge of isolating lineage committed progenitors that are specified during the earliest stages of differentiation, we used flow cytometry in conjunction with libraries of commercial and novel monoclonal antibodies that we prepared against surface markers of hESCs. Of over 30 different subsets defined by specific cell surface markers, we discovered only four distinct precursor profiles, two of which likely correspond to early visceral endoderm cells and later-stage mesododerm progenitors. Purification of these precursor types may improve the derivation of desired lineages and facilitate study of early differentiation programs with unprecedented resolution.

3:20 Macro- and Micro-Scale Control of Stem Cell Aggregate Differentiation in Suspension Culture
Todd McDevitt, Ph.D., Assistant Professor, Biomedical Engineering, Georgia Institute of Technology and Emory University 
Differentiation of ESCs in vitro is commonly induced via 3D cell aggregates in suspension, referred to as “embryoid bodies” (EBs), which typically yield a heterogeneous population of ectoderm, endoderm and mesoderm cells. Currently, most strategies to direct the differentiation of ESCs consist solely of applying soluble factors exogenously to stem cell cultures, however, improved methods to spatiotemporally control physico-chemical cues influencing cell morphogenesis may result in increased efficiency and homogeneity of stem cell differentiation. We have developed and characterized the effects of novel methods to control the differentiation of EBs via 1) hydrodynamic forces imposed during suspension culture and 2) microparticle-mediated delivery of morphogenic factors directly within stem cell aggregates. These novel enabling technologies to enhance the directed differentiation of ESCs represent scalable approaches capable of being directly integrated into bioprocessing methods for the engineering of stem cell therapies and diagnostics.

3:50 Module Map of Stem Cell Genes Guides Creation of Epithelial Cancer Stem Cells
David Wong, M.D., Ph.D., Instructor, Dermatology, Stanford University
Cancer cells and normal stem cells share the unique property of self-renewal. A gene module map of stem cells and differentiated cells revealed two distinct transcriptional signatures that distinguish embryonic stem cells (ESC) and adult tissue-specific stem cells. The ESC-like transcriptional program is activated in human epithelial cancers and strongly predicts metastasis and death. MYC is sufficient to activate the ESC-like program in normal and cancer cells and increase the fraction of tumor-initiating cells by 150-fold, enabling tumor formation and serial propagation with as few as 500 cells. These results suggest that activation of an ESC-like transcriptional program in differentiated adult cells may induce pathologic self-renewal characteristic of cancer stem cells.

4:20 Reception in the Exhibit Hall

5:00 Breakout Discussions in the Exhibit Hall
Menstrual Stem Cells – A Novel Source of Cells
Moderator: Julie Allickson, Ph.D., Vice President Laboratory Operations, R&D, Cryo-Cell International, Inc.

• Introduction on the menstrual stem cell 

• Cell procurement methods and cell processing

• Cell characteristics including cell phenotype (adult and embryonic cell surface markers)

• Cell Culture proliferative capacity including doubling time

• Cell longevity demonstrated by cell passages in culture and telomerase activity

• Safety Studies

• Cell capability for differentiation

• Pre-clinical studies

Cell Therapies: Preparing the Case for Reimbursability 
Moderator: Lee Buckler, B.E.d, LLB, Principal, Cell Therapy Group 

• How do insurers view cell therapies now? 

• What must cell therapy companies do now to position their therapies for reimbursement when they hit the market?

The Epigenetic Landscape of Human Embryonic Stem Cells
Moderator: Mark Levenstein, Ph.D., CDI Bioscience
The unique status of hESC, their ability to self-renew indefinitely and to develop into any cell type in the body, drives their promotion as a therapeutic Rosetta Stone. This informal discussion will go beyond the morphological and genetic characteristics of pluripotent cells and focus on their epigenetic state. Discussion topics include:

• Chromatin modifications

• DNA methylation

• small RNAs

• imprinting 

• How epigenetic analyses can aid in the characterization of reprogrammed (iPS) cells

6:00 Close of Day One

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