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Stem Cell Research Challenges Conference, November 7-9, 2005, La Jolla, CA

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    Stem Cell Research Challenges Conference, November 7-9, 2005, La Jolla, CA

    Agenda for Stem Cell Research Challenges

    - Radisson Hotel La Jolla - La Jolla, CA

    Pre-Conference Workshop
    Monday, November 7, 2005

    Characterization and Comparability of Stem Cell Populations or Lines
    7:30Registration and Coffee8:25Workshop Leader's Welcome and Opening Remarks
    Evan Snyder, M.D., Ph.D., Professor and Director, Stem Cells and Regeneration Program, The Burnham Institute
    8:30Measures of Stability for Cells Propagated in Culture
    Cells can undergo genetic and epigenetic changes in culture and need to be rigorously monitored for clinical therapy. The presentation discusses a proposed series of tests that are believed to be sufficient to evaluate overall stability of cells propagated in culture.
    Mahendra Rao, Ph.D., Investigator and Head, Stem Cell Biology Unit, National Institute on Aging, National Institutes of Health
    9:00Assessing the Genetic and Epigenetic Stability of Stem Cells
    The search for "stemness" genes has suggested that gene expression alone is insufficient to define plasticity & lineage-specification. It was hypothesized that stem cell behavior should also be characterized by epigenetic processes, specifically chromatin state, which in turn determines the degree of transcriptional activity. The dynamics of epigenetic chromatin modification was examined, first in a "pluripotent" human "embryonic" stem cell (hESC) and then in a "multipotent" human "somatic" stem cell (i.e., germ-layer-restricted).
    Evan Snyder, M.D., Ph.D., Professor and Director, Stem Cells and Regeneration Program, The Burnham Institute
    9:30Generating High Purity Cell Populations from Human ESC
    Human ESC (hESCs) demonstrate remarkable proliferative and developmental capacity. Clinical interest arises from their ability to provide an apparently unlimited cell supply for transplantation, and from the hope that they can be directed to desirable phenotypes in high purity. Lessons learned from the derivation of high purity, high yield oligodendrocytes and their progenitors from hESCs are presented. Experience indicates that a unique media formulation is required for each differentiation step.
    Jill Faulkner, Ph.D., Postdoctoral Fellow, Keirstead Lab, Reeve-Irvine Research Center
    10:00Networking Refreshment Break10:30Cell Line Safety and Quality Control
    Standardization of cell culture systems and development of characterization tools will be critical to the orderly progress toward hESC-derived products. The ideal stem cell expansion, manipulation and differentiation systems are completely defined and free of products of animal origin. hESCs need to be assessed for transfer of viruses, animal genetic material and potentially immunogenic carbohydrate epitopes. Standards for assessing the biological status of hESCs and their differentiated progeny need to be determined.
    Ian Lyons, Ph.D., Research Area Manager, Invitrogen
    11:00Cell Line Strategies and Applications for Research, Industrial, and Therapeutic Use
    The specifications and requirements of stem cell lines are different depending on the applications and the settings in which the cells are to be used. Strategies to accommodate and evaluate these criteria are discussed in the presentation. In addition, methodologies developed for research and industrial stem cell applications are highlighted.
    Peter Sartipy, Ph.D., Senior Scientist, Cellartis AB, Sweden
    11:30Panel Discussion with Workshop Speakers
    12:00Workshop Ends;
    Main Conference Registration Open

    Main Conference - Day One
    Monday, November 7, 2005

    1:30Chairperson's Opening Remarks
    Derek Van der Kooy, Ph.D., Professor of Medical Genetics and Microbiology, University of Toronto, Canada
    Point/Counterpoint: Can Plasticity Be Achieved in Adult Stem Cells?
    Important Issues Associated with a Commitment to Adult vs. ESC
    Keynote Presentation1:45Adult Stem Cell Plasticity and the Brain
    Adult rat and human bone marrow stromal cells (MSCs) have been differentiated into presumptive neurons in vitro. Consequent to transplantation to embryonic rat ventricles, the MSCs migrated throughout the brain, to both germinal and parenchymal areas, and expressed region-specific neuronal genes. To begin exploring the spectrum of potentially plastic cell types, amniocytes were examined, which have also been converted to neural cells.
    Ira B. Black; M.D., Founding Director, Stem Cell Institute of New Jersey; Professor and Chairman, Neuroscience and Cell Biology, Robert Wood Johnson Medical School
    2:30The Tissue Plasticity of Stem Cells
    Although the reports of others that adult brain stem cells can make blood cells in adult hosts or contribute widely to tissues in blastocyst chimera could not be replicated, two examples of stem cell tissue plasticity were seen. First, the primitive neural stem cells that arise clonally from single ES cells show some pluripotency in blastocyst chimeras. Second, adult pancreatic stem cells can produce neurons.
    Derek Van der Kooy, Ph.D., Professor of Medical Genetics and Microbiology, University of Toronto, Canada
    3:00From Blood to Brain: Can Circulating Blood Cells Help Repair the Brain?
    Neuronal regeneration in mice after stroke was studied by examining the entry of GFP positive bone marrow cells into the brain and their differentiation there. While most BM derived cells differentiate into microglia, neural cell types of donor origin were also observed. These findings suggest that BM derived cells may help patients with neurodegenerative diseases or with CNS injury.
    Eva Mezey, M.D., Ph.D., Senior Investigator, National Institutes of Health
    3:30Networking Refreshment Break4:15Commercial Challenges for Cell Therapy Manufacturing
    Cell therapy presents challenges to commercial development, including tissue acquisition, cell isolation, scale-up, regulatory compliance and distribution. Autologous and allogeneic products may require different manufacturing models. Bone marrow is a source for hematopoietic stem cells that produce blood lineages, and mesenchymal stem cells that produce musculoskeletal and connective tissue lineages. Specific signal transduction pathways appear to be involved in stem cell plasticity.
    Daniel R. Marshak, Ph.D., Vice President & Chief Technology Officer, Cambrex Corporation
    4:45Development of Pluripotent Stem Cell Therapies
    The tissue regenerative capacity of pluripotent stem cell cultures, such as the MAPC, hold much promise as cell therapeutics. This is due to the ability to expand cytogenetically and biologically stable stem cells to many clinical doses. Preclinical models using pluripotent stem cells in acute ischemic injury to the CNS and cardiovascular system show physiological improvement reflective of their tissue regenerative properties.
    Robert Deans, Ph.D., Vice President of Regenerative Medicine, Athersys, Inc.
    5:15Panel Discussion
    The Debate Over Adult Stem Cell Differentiation

    6:00Networking Cocktail Reception; Exhibit and Poster Viewing

    Main Conference - Day Two
    Tuesday, November 8, 2005

    8:00Networking and Coffee 8:25Chairperson's Opening Remarks
    Ron McKay, Ph.D., Director, Stem Cell Unit, National Institute of Neurological Disorders and Stroke, National Institutes of Health
    Controlling Embryonic Stem Cell Differentiation
    What Methods Are Being Used to Control Differentiation?
    8:30Molecular Analysis of ES Cells and Differentiation
    The gene expression profiles and DNA methylation patterns of multiple lines of ES cells are being systematically characterized as they differentiate under controlled conditions. In addition, high-density SNP maps are being created for each line. The goal is to provide all of these data as a comprehensive database that will be offered by the Stem Cell Community, a privately funded website based at The Burnham Institute. Jeanne F. Loring, Ph.D., Director of Human Stem Cell Resource, Associate Professor, Program in Stem Cells and Regeneration, The Burnham Institute
    9:00Controlling Growth and Survival of CNS Stem Cells and Neurons
    In vitro differentiation methods have been developed that generate large numbers of functional dopaminergic neurons from human ES cells and other sources. These studies define a new link between dopamine function and neuron survival. These results suggest that the fundamental molecular biology of CNS stem cells and differentiated neurons may be manipulated to achieve regenerative responses to acute or chronic neuronal loss.
    Ron McKay, Ph.D., Director, Stem Cell Unit, National Institute of Neurological Disorders and Stroke, National Institutes of Health
    9:30Neural and Mesenchymal Derivatives from Human ES Cells
    This presentation includes data on the derivation of multiple neural derivatives from human ES cells and their application in pre-clinical models of neural disease. It will also present the derivation of multipotent mesenchymal precursors from human ES cells as a novel strategy to provide rapid access to a wide range of tissue types including cartilage, bone and skeletal muscle. Finally, efforts to translate these findings to autologous ES cell sources obtained via somatic cells nuclear transfer or parthenogenesis is discussed.
    Lorenz Studer, M.D., Professor, Laboratory of Stem Cell & Tumor Biology, Developmental Biology & Neurosurgery, Memorial Sloan Kettering Cancer Center
    10:00Networking Break, Exhibit and Poster Viewing10:30Chemical and Functional Genomic Approaches Toward Regenerative Medicine
    Cell-based phenotypic and pathway-specific screens of synthetic compounds and arrayed cDNA/siRNA libraries have recently provided a number of small molecules and genes that can be used to selectively control stem cell fate. Such studies will likely provide new insights into stem cell biology, and may ultimately contribute to the development of effective medicines for tissue repair and regeneration.
    Sheng Ding, Ph.D., Assistant Professor, Departments of Chemistry and Cell Biology, The Scripps Research Institute
    11:00In Vivo Differentiation of Stem Cells and Their Progeny
    The developmental signals that instruct ESC to choose a particular tissue pathway are still not understood. Left to their own devices in a culture dish, they will spontaneously differentiate into a hodgepodge of cell types. It would be ideal if progenitor-cells could simply be injected into the part of the body where regeneration is desired and let them take their cues from the surrounding environment.
    Robert Lanza, M.D., Vice President, Medical & Scientific Development, Advanced Cell Technology; Adjunct Professor, Institute of Regenerative Medicine, Wake Forest University School of Medicine
    11:30Technology Workshop
    For more information on holding a technology workshop at this event, contact Gary Hill at or call 508-614-1239
    12:00Networking Luncheon, Exhibit and Poster ViewingCase Studies and Lessons Learned in the Development of Stem Cell Therapeutics
    What Therapeutic Developments Can Be Expected from Our Investment in Time and Resources?

    1:25Chairperson's Opening Remarks
    Alberto Hayek, M.D., Professor of Pediatrics, University of California, San Diego; Director, Islet Research Laboratory, The Whittier Institute
    1:30An Update on Islet Cell Transplantation
    After the initial report from the Edmonton group five years ago, there has been a great deal of activity in the field of islet transplantation in the United States and Europe. The results, however, have not been as favorable as those from Edmonton. The presentation briefly reviews the current status of islet transplantation in the U.S. and discusses reasons for the different results obtained at other centers.
    Alberto Hayek, M.D., Professor of Pediatrics, University of California, San Diego; Director, Islet Research Laboratory, The Whittier Institute
    2:00Human ESC: Advancing Technology for Therapeutic Application
    Human embryonic stem cells (hESCs) can be propagated yet differentiate to all somatic cells. In vitro and in vivo studies have shown that differentiated cells resembling normal tissue counterparts can be produced from hESCs. Scaled production of hESCs has now been enabled for the manufacture of these therapeutic cell types. Reproducible and scaled methods for differentiation of the hESCs to the targeted cell type are now being optimized. These hESC-derived cells are being tested in preclinical safety and efficacy studies.
    Jane S. Lebkowski Ph.D., Senior Vice President, Regenerative Medicine, Geron Corporation
    2:30Stem Cells in Predictive Safety Models - A Focus on Cardiotoxicity
    Embryonic stem cell technology offers unprecedented opportunities for in vitro drug discovery and safety assessment of compounds. Cardiotoxicity is a major obstacle towards progression of multiple classes of compounds through development. Therefore, predictive in vitro models have the opportunity to reduce attrition and enable safer drugs. The presentation discusses opportunities, technology development and application of ES-derived cardiomyocytes in predictive cardiotoxicity assays.
    Gabriela Gebrin Cezar, DVM, Ph.D., Assistant Professor, Dept. of Animal Sciences, University of Wisconsin-Madison
    3:00Networking Break, Exhibit and Poster Viewing
    3:30So You Think You Have Found a Human "Stem Cell" . . . Now What?
    The pathway from discovery of a candidate human stem cell therapeutic is a long and arduous one. StemCells, Inc. discovered the human neural stem cell in 1999 and filed its first IND in 2004. Why did it take so long?
    Martin McGlynn, President, StemCells, Inc.
    4:00Brain Tumor Stem Cells
    It has been demonstrated that there are cells present in brain tumors that have many of the characteristics of neural stem cells. In some circumstances, these are brain tumor stem cells in that they can give rise to tumors in immunodeficient mice. Gene expression analysis indicates that pathways critical for the self-renewal of neural stem cells are also important for brain tumor cell proliferation. These pathways are being evaluated as novel targets for brain tumor therapy.
    Harley Kornblum, M.D., Ph.D., Director, Neural Stem Cell Research Center, University of California, Los Angeles School of Medicine
    4:30 The Future Course of Embryonic Stem Cell Research in the United States: Ethics, Regulatory and IP Influences on Scientific Progress
    Lawrence S.B. Goldstein, Ph.D., Professor of Cellular and Molecular Medicine, University of California, San Diego, School of Medicine
    5:00Close of Day Two

    Main Conference - Day Three
    Wednesday, November 9, 2005

    7:30Networking and Coffee
    7:55Chairperson's Opening Remarks
    James Kovach, M.D., J.D., President and Chief Operating Officer, Buck Institute for Age Research
    The Impact of Regulatory and IP Issues on the Development of Your Stem Cell Projects
    How Outside Forces Can Affect Even the Most Sound Science
    8:00Moving Stem Cell Research to Clinical Trials: Coping with Regulatory Challenges and the FDA
    Ongoing research efforts to elucidate complexities of stem cell biology are vital to producing novel safe and effective cellular therapies. Reviewing investigational stem cell-based therapies is the responsibility of FDA's Office of Cellular, Tissue, and Gene Therapies, Center for Biologics. This presentation introduces the listener to FDA regulatory expectations for developing nascent stem cell-based therapies, including those derived from human ESC.
    Donald Fink, Jr., Biologist, Office of Cellular, Tissue and Gene Therapies, U.S. Food and Drug Administration
    8:30Stem Cell Intellectual Property: The WARF Patents and Recent Decisions
    The presentation discusses the impact of the WARF (Wisconsin Alumni Research Foundation) patents on the commercialization of stem cell-based products, and provides current information on recent Supreme Court decisions affecting product development in this field.
    George Xixis, JD, Partner, Nutter, McClennan and Fish LLP
    9:00Intellectual Property Implications of State-Funded Stem Cell Research
    Patents are critical to the commercialization of discoveries in the pharmaceutical industry. Because many inventions relating to stem cell research will be developed without any federal funding, they are not subject to the Bayh-Dole Act, under which the grantee institutions control patents and benefit from resulting royalties. The presentation explores the complex ramifications of how state-funded stem cell inventions are best protected and commercialized to promote the public interest.
    Cathryn Campbell, JD, Ph.D., Partner, McDermott, Will & Emery LLP
    9:30Networking Refreshments, Last Chance for Exhibit and Poster Viewing
    10:00Panel Discussion
    What Needs to Happen to Enable the Advances in Academic Research to be Applied in the Clinic?

    The substantial body of academic research now being conducted on stem cells must transition from the laboratory through a series of commercialization steps if these breakthrough developments are to be applied routinely in clinical practice. This will require the adoption of a more product-focused mindset on the part of academic researchers - and issues of funding, development infrastructure and suboptimal industry commitments to incubation-stage research must all be overcome. The panel brings together academic, clinical, industry, funding and legal perspectives to discuss and debate the steps needed to transition stem cell research to commercial applications, with the goal of helping delegates foster successful collaborations in this field.
    James Kovach, M.D., J.D., President and Chief Operating Officer, Buck Institute for Age Research
    Peter G. Carroll, Esq., Partner, Medlen & Carroll, LLC Donald Fink, Jr., Biologist, Office of Cellular, Tissue and Gene Therapies, U.S. Food and Drug Administration
    Standish Fleming, Managing Member, Forward Ventures
    Martin McGlynn, President, StemCells, Inc.
    Evan Snyder, M.D., Ph.D., Professor and Director, Stem Cells and Regeneration Program, Burnham Institute
    11:30End of Conference
    Last edited by Faye; 22 Sep 2005, 4:32 PM.