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Sunday, August 28

5:30-6:30 Early Registration

Day One: Stem Cell Research and Its Application for Drug Discovery


Monday, August 29

7:30 Registration and Morning Coffee

Applications in Drug Development and Screening

8:15 Chair's Opening Remarks
Dr. Linda Griffith, Director, Biotechnology Process Engineering, Massachusetts Institute of Technology

Kick-Off Keynote Presentations 
(combined session with Tissue Models for Therapeutics)

8:30 Cells and Tissues in Context: Culture Models for the 21st Century!
Dr. Mina J. Bissell, Distinguished Scientist, Lawrence Berkeley National Laboratory 
The extracellular matrix is now widely recognized as an important source of signals for gene expression as well as cell division, survival, shape, and movement, all of which are altered during tumor progression. Construction of more realistic three-dimensional models of normal breast and breast cancer that mimic the normal and diseased conditions would allow gaining new insights into breast tumorigenesis and the findings continue to challenge convention. This presentation highlights the progress of three-dimensional breast cancer models and potential of utilizing this model system to study cancer pathogenesis and test anticancer drugs.

9:15 Chemical and Functional Genomic Approaches Toward Regenerative Medicine
Dr. Sheng Ding, Assistant Professor, Departments of Chemistry and Cell Biology, Genomics Institute of the Novartis Research Foundation, Scripps Research Institute 
Recent advances in stem cell biology may make possible new approaches for the treatment of a number of diseases. Such approaches could involve cell replacement therapy and/or drug treatment to stimulate the body's own regenerative capabilities, while they will require identification of renewable cell sources of engraftable functional cells, an improved ability to manipulate stem cell proliferation and differentiation, as well as a better understanding of the signaling pathways that control their fate. Cell-based phenotypic and pathway-specific screens of synthetic compounds have recently provided a number of small molecules that can be used to selectively control stem cell fate. Such molecules will likely provide new insights into stem cell biology, and may ultimately contribute to effective medicines for tissue repair and regeneration.

10:00 Coffee Break

10:10 Chair Remarks
Dr. Sheng Ding, Assistant Professor, Departments of Chemistry and Cell Biology, Genomics Institute of the Novartis Research Foundation,Scripps Research Institute

Featured Presentation 
10:15 Use of Zebrafish to Find Stem Cell Genes and Drugs 
Dr. Leonard I. Zon, Grousbeck Professor of Pediatrics, Howard Hughes Medical Institute Investigator, Children's Hospital, Boston 
Embryonic stem cells have the ability to make all tissues of the body. Blood cells have been derived from embryonic stem cells using a simple culture media in a semisolid media. Yet, when these cells are transplanted into lethal irradiated mice no reconstitution is evident. In an effort to find factors that increase stem cell numbers and confer a long-term potential to the hematopoietic stem cell, we have utilized the zebrafish system. Zebrafish are amenable to high-throughput screens for chemicals that regulate activity. Several chemical screens are underway to look for stem cell activators. These chemicals are then utilized on embryonic stem cells to evaluate whether there is a potential for long-term reconstitution. A comparative approach to stem cells should provide a better understanding of the pathways necessary for self-renewal and differentiation.

10:45 Pharmacological Potential of Embryonic Stem Cells
Dr. Shilpa Kadam, Research Investigator I, Epigenetics Group, Novartis Institutes for BioMedical Research
Stem cells are defined by the ability both to produce identical daughter cells (self-renewal), and to produce progeny with more restricted fates (commitment and differentiation). This property of stem cells underpins growth and diversification during development and sustains homeostasis and repair processes throughout adult life. An understanding of molecular mechanisms which govern stem cell fate is therefore of fundamental significance in cell and developmental biology and the capabilities arising from such knowledge have major biomedical applications. The ease with which these cells can be cultured under in vitro conditions serves as an important new tool for developing unique, in vitro model systems to test drugs and chemicals, and as a potential to predict toxicity in humans. The current focus of our investigation is to understand the mechanisms of self-renewal and differentiation of stem cells using both genomic and proteomic approaches, and to understand how to manipulate adult stem cells, which hold great promise in regenerative medicine.

11:15 Cloning and Stem Cells: Interrogating Development and Disease by Nuclear Transplantation
Dr. Kevin Eggan, Junior Fellow, The Harvard Society of Fellows, Department of Molecular and Cell Biology, Harvard University
Nuclear transplantation and embryonic stem cell technologies provide a novel route to investigate the mechanisms the underlie both embryonic development and disease progression. Nuclear transplantation using donor cell from individuals affected by genetic diseases would allow the production of embryonic stem cell lines that carry the compliment of genes which cause that disease. By differentiating these "disease specific" embryonic stem cell lines into the affected cells in vitro, the progression and pathogenesis of the disease might bee studied. In addition, studying the nuclear transplantation process itself will provide information into how cells transition from developmental state to another. 

11:45 Panel Discussion with Morning Speakers or Technology Spotlights

12:15 Lunch on your own
(Technology Workshops Available)

Stem Cell Models for Screening Assays 
(combined session with Tissue Models for Therapeutics)

2:00 Chair's Remarks
Dr. Jonathan A. Garlick, Professor and Director of Cancer Biology and Tissue Engineering, Tufts University School of Dental Medicine

2:05 Stem Cell Models for Target Validation, Drug Screening and Safety Assessment
Dr. John Hambor, Associate Research Fellow, Experimental Medicinal Chemistry, Pfizer Global Research and Development
Cell models play a significant role in all phases of drug discovery and development from target validation studies, to high-throughput screening of chemical libraries, and finally to testing drug metabolism, transport and toxicity, and they provide critical information allowing selection of safe, efficacious compounds with the best chance of success in the clinic. Recent advances in stem cell technology have led to the development of renewable cultured cell systems that more closely match real human target tissues allowing for more predictive assessment of compound safety and efficacy. Moreover, the advent of simple, quick, robust strategies that reliably lead to differentiation into the desired tissue has permitted the utilization of stem cell-derived cell types in screening assays, as well as provided unique opportunities to explore their developmental pathways for the discovery of regenerative small molecules. Progress in applying stem cell models as research tools for target validation, drug screening and safety assessment will be discussed.

2:35 Using Motor Neurons Derived from Embryonic Stem Cells for Drug Discovery
Dr. Amy Sinor, Researcher, Assay Development, Curis
We are able to generate cultures derived from embryonic stem (ES) cells that are highly enriched for motor neurons. This ability provides us a unique opportunity to perform high-throughput screens in motor neurons. Since these cultures represent the actual cell type that is affected during the progression of many motor neuron diseases, such as Spinal Muscular Atrophy and Amyotrophic Lateral Sclerosis, this provides a strong rationale to use motor neuron cultures to screen for potential drugs. Our pioneer work endorses the great potential of using stem cells for drug discovery. Since ES cells have the ability to differentiate into many different neuronal cell types in cultures, we expect this model system to be valuable for screening drugs for many other neurodegenerative diseases.

3:05 A Novel Functional Assay of Cell Binding by a Medical Device/Implant
Dr. Mary Zacour, Principal Scientist, in Vitro Assay Development, Metabolism Resource, MDS Pharma Services
Implanted medical devices or scaffolds that are designed to harness a patient's endogenous stem or precursor cells have enormous potential as a therapeutic approach for a variety of pathologies, since they can target tissue regeneration and/or repair responses to relevant sites in vivo. In order to develop such devices effectively, there is a need for functional in vitro assays, both for assessing the effects of developmental modifications and for eventual quality control of the final product. Many assays of cell binding to scaffolds are largely qualitative, labor-intensive, and very low throughput. We have recently developed a 96-well plate format, quantitative cell-based assay for direct functional assay of cell-binding of a medical device. The assay is adaptable to other devices and cell phenotype specificities, and as such may provide a valuable research tool to developers of such devices.

3:35 Refreshment Break, Poster and Exhibit Viewing

Sponsored by:

 

Engineering Higher Throughput Screening Environments 

4:15 Using Automated Microcarrier-Based Cell Culture to Improve Human Cell Phenotype
Dr. Robin Felder, Professor of Pathology and Medical Automation, University of Virginia
3-D cell culturing produces cells that more closely resemble in vivo phenotypes, which will increase their value in screening, research, and ultimately as a cell source for regenerative medicine. Novel microcarriers, bioreactors, and automation systems are being used (and integrated) to allow hands-free growth and maintenance of cells for just-in-time delivery of contamination-free product directly into high throughput screening systems. This talk will focus on and review the technologies available to both the bench scientist and those interested in production-scale cell production.

4:45 Panel of Experts: Engineering a Functional High-Throughput Tissue Screening System
High-throughput assay systems (HTS) are needed to most fully take advantage of 3D tissues as a target system for therapeutics. This panel will focus on the issues and challenges facing tissue biologists, pharmacologists and engineers that need to be addressed to accelerate development of these assays. Discussion will include such issues as:
1- Can tissues be scaled to an HTS format? 
2- What are the plasticware and media requirements? 
3- How can production and screening of 3D tissues be robotized?
Panel members will include experts in each of these areas of HTS application and development. By engineering 3D tissues suitable for HTS, novel screening and therapeutic applications for tissues will be feasible. 
Panel Moderator: Dr. Jonathan A. Garlick, Professor and Director of Cancer Biology and Tissue Engineering, Tufts University School of Medicine 
Panel Participants
Dr. Angela Cacace, Senior Research Investigator II, Lead Discovery, Bristol Myers Squibb Co.
Dr. Linda Griffith, Director, Biotechnology Process Engineering, Massachusetts Institute of Technology
Dr. Robin Felder, Professor of Pathology and Medical Automation, University of Virginia
Dr. Debra Hoover, Applications Manager, Life Science, CorningDr. 
John D. McNeish, Senior Director, Genetic Technology, Pfizer Global Research and Development
Dr. Paul Price, Resident Fellow, Cell Culture Res, GIBCO Invitrogen

5:30 Networking Reception in Exhibit Hall

6:30 Close of Day One


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