Final Agenda
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Recommended Pre-Conference Short Course *
(SC4) Identification, Characterization and Targeting of Cancer Stem Cells
*Separate Registration Required.
The new generation of stem cell research offers viable insights and resources of replacement cells to treat and reverse diseases, leading to regenerative medicine and ultimately personalized therapies. The primary objectives of Cambridge Healthtech Institute’s Stem Cells are the basics of regenerative medicine, including stem cell sources (embryonic, adult, cord blood or iPS) and technologies to harness their potential, pathways to deliver the new therapies, and translation of basic stem cell research into clinical applications.
8:55 Chairperson’s Remarks
Larry A. Couture, Ph.D., Senior Vice President, Center for Applied Technology Development, Beckman Research Institute of City of Hope
9:00 Comparison of Human Pluripotent Stem Cells
Kiichiro Tomoda, Ph.D., Research Scientist, Gladstone Institute of Cardiovascular Disease, The Gladstone Institute, University of California, San Francisco
Human Pluripotent stem cells (human embryonic stem cells (hESCs) and human induced pluripotent stem cells (hiPSCs) which can differentiate into cell types of the three germ layers in vitro and in vivo, are attractive sources for human cell replacement therapies; however, we still do not fully understand many properties of hiPSCs. To gain insight into hiPSCs, we have been comparing several hiPSCs with hESCs lines in terms of gene expression profile, proliferation and differentiation potentials. Our latest results from the comparison will be discussed in the meeting.
9:30 Challenges for Efficient Production of Human iPS cellsBonnie Barrilleaux, Ph.D., Postdoctoral Fellow, CIRM Stem Cell Training Program, Department of Cell Biology and Human Anatomy, School of Medicine, University of California, DavisViral gene delivery is currently the most reliable option for delivering reprogramming genes to produce human induced pluripotent stem cells (hiPSCs). Using amphotropic virus, we have generated and characterized three hiPSC lines. However, commonly used pluripotency-inducing genes (including c-Myc, Oct4, SOX2, and KLF4) all have known links to cancer, necessitating higher biosafety level handling requirements when producing pantropic lentivirus encoding these genes. To address these biosafety issues, we describe the use of ecotropic lentivirus for overexpression of oncogenes in human cells, as well as polymer complexation to enhance transduction while avoiding aerosol-forming centrifugation of viral particles.
10:00 The Road to Translation of iPS Cells
Marie Csete, M.D., Ph.D., Executive Vice President, Research & Development, Organovo, Inc.
The lecture will review promising (closest to clinic) potential applications for iPS cells, with emphasis on the bottlenecks. The lecture will review the importance of iPS cells in drug discovery, with emphasis on the need for three-dimensional in vitro tissue culture models.
10:30 Refreshment Break in the Exhibit Hall
11:30 Regulatory and Manufacturing Considerations for the Development of Autologous Induced Pluripotent Stem Cell Therapies - Click here for Podcast
Larry A. Couture, Ph.D., Senior Vice President, Center for Applied Technology Development, Beckman Research Institute of City of Hope
Perhaps the most important potential application for induced pluripotent stem cells (iPSC) is their use as autologous cell therapeutics. While human embryonic stem cells (hESC) and iPSC are treated by the FDA according to established regulations for cell therapeutics, the application of these regulations in establishing safety and manufacturing reproducibility of an autologous cell therapy will be a significant regulatory and manufacturing challenge. We will consider the nature of these challenges and discuss possible approaches to overcoming them.
12:00 pm Panel Discussion with Morning Speakers
12:30 Luncheon Presentations or Lunch on Your Own
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1:45 Ice Cream Refreshment Break in the Exhibit Hall
2:15 Chairperson’s Remarks
Dan S. Kaufman, M.D., Ph.D., Associate Professor, Medicine, Division of Hematology, Oncology, and Transplantation; Associate Director, Stem Cell Institute; University of Minnesota
2:20 Mesoderm Commitment and Blood Formation from Human Pluripotent Stem Cells
Gay M. Crooks, Professor, Pathology & Laboratory Medicine, University of California, Los Angeles
The ability to generate hematopoietic cells from human pluripotent stem cells (hPSC) has generated the possibility of providing an unlimited supply of patient-matched hematopoietic stem cells (HSC) in the future. However, it is apparent that CD34+ cells produced from hPSC harbor intrinsic, functional differences from the “adult” CD34+ cells used in clinical transplantation. By identifying the earliest stage of mesoderm commitment from hPSC, we can understand and manipulate the process by which blood formation from hPSC is regulated.
2:50 Therapeutic Human Pluripotent Stem Cell-Derived Cells to Treat Cancer and HIV/AIDS
Dan S. Kaufman, M.D., Ph.D., Associate Professor, Medicine, Division of Hematology, Oncology, and Transplantation; Associate Director, Stem Cell Institute; University of Minnesota
Our recent studies have demonstrated the ability to use hESC and iPSC-derived natural killer (NK) cells to kill diverse human cancer cells using an in vivo xenograft model. Additionally, we demonstrate these hESC and iPSC-derived NK cells can preferentially kill HIV-infected cells. We now aim to advance this work to use human pluripotent stem cells as a resource for new clinical immunotherapeutic applications against a variety of lethal diseases. Separate studies by our group have also derived vascular cells and osteogenic cells from hESCs and iPSCs suitable for regenerative medicine therapies.
3:20 Novel microRNAs in hESC and Pluripotent Cells
Ronald P. Hart, Ph.D., Cell Biology & Neuroscience, Rutgers Stem Cell Research Center, Rutgers University
Using deep sequencing of small RNAs, we identified novel candidate microRNA genes in human embryonic stem cells and early neuronal precursor cells. Since a large number of differentiation mechanisms require microRNAs, we propose that these novel microRNAs, in addition to previously described pluripotency-specific microRNAs, serve to establish phenotypic stability during commitment to new cell types.
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3:50 Refreshment Break & Poster Awards in the Exhibit Hall
4:30 Generation of Functional Dopaminergic Neurons from Human iPSCs under Xeno-Free Defined Conditions for Treating Parkinson’s Disease
Xianmin Zeng, Ph.D., Associate Professor and Director, CIRM Shared Research Laboratory for Stem Cells & Aging, Buck Institute for Age Research
We have shown that functional dopaminergic neurons can be generated from multiple human iPSC lines derived from different somatic cells. Neural and dopaminergic differentiation measured by various molecular and cellular methods in iPSCs is efficient and similar to in hESCs using a scalable defined cultured system readily transferable to a GMP facility. Importantly, iPSC-derived dopaminergic neurons could improve symptoms of PD in a pre-clinical rodent model, and be genetically modified efficiently.
5:00 Reprogramming Tumor-Specific T Cells Using iPS Technology for the Treatment of Patients with Cancer
Nicholas P. Restifo, M.D., Principal Investigator, National Cancer Institute
Anti-tumor T cells can induce long-lasting disappearance of metastatic cancer, but terminal differentiation, exhaustion and senescence of these T cells can hamper their effectiveness. Our laboratory and clinical teams at the NIH Clinical Center seek academic and industrial partners to rapidly translate iPS technology into new clinical cancer immunotherapy trials.
5:30 Panel Discussion with Afternoon Speakers
6:00 Close of Day
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