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How Do Pluripotent Cells Enable Drug Discovery?


for Members

How Do Pluripotent Cells Enable Drug Discovery?

Tuesday, March 23, 2010

The New York Academy of Sciences

Presented By


Recent major breakthroughs are propelling the field of stem cell research. Induced pluripotent stem cells (iPS cells) are being created to maintain all the potential of embryonic stem cells without using embryos, eliminating ethical concerns . The technical difficulties of creating stable cells with proper phenotypes to avoid the hazard of teratocarcinomas or other unwanted cell proliferation when cells are to be used therapeutically still remain, but research is progressing rapidly. The first embryonic stem cell trial is the US has recently been approved by the FDA for severe spinal cord injury. Beyond therapeutics, the promise of using differentiated human stem cells in drug discovery as disease relevant and toxicology models is maturing to mimic relevant human organ responses while reducing the need to use experimental animals.

Stem cells and cell lines derived from iPS cells of patients can accelerate the development of existing targets for different diseases and provide opportunity to explore innovative treatment opportunities in regenerative medicine. This symposium will review the current use and potential future of using pluripotent stem cells as enabling technology in drug discovery and therapeutic entity.


*Presentation times are subject to change.

1:00 – 1:15 PM

Katalin Kauser, MD, PhD, DSc , Boehringer Ingelheim Pharmaceuticals

1:15 – 2:00 PM

The Importance of Relevant Cells for Drug Discovery
John Hambor, PhD, Cell Therapy Group

2:00 – 2:45 PM

Factors Influencing Nuclear Reprogramming
Konrad Hochedlinger, PhD, Harvard Stem Cell Institute

2:45 – 3:15 PM

Coffee Break

3:15 – 4:00 PM

A Chemical Approach to Controlling Cell Fate
Sheng Ding, PhD, Scripps Research Institute

4:00 – 4:45 PM

Human Pluripotent Stem Cell-derived Cardiomyocytes for Drug Development
Timothy Kamp, MD, PhD, University of Wisconsin

4:45 – 5:00 PM

Closing Remarks
Huiping Jiang, PhD, Boehringer Ingelheim Pharmaceuticals



Sheng Ding, PhD

Scripps Research Institute

Dr. Sheng Ding is currently Associate Professor in the Department of Chemistry at The Scripps Research Institute in La Jolla, USA. He obtained his BS in chemistry with honors from Caltech in 1999, and a PhD in chemistry from Scripps in 2003. Dr. Ding has pioneered on developing and applying innovative chemical approaches to stem cell biology and regeneration, with a focus on discovering and characterizing novel small molecules that can control various cell fate/function, including stem cell maintenance, activation, differentiation and reprogramming in various developmental stages and tissues. Ding has published over 60 research articles, reviews and book chapters, and made several seminal contributions to the stem cell field. Ding is a cofounder of Fate Therapeutics, Stemgent, and Transfigure Medicine.

John Hambor, PhD

Cell Therapy Group

Dr. John Hambor is currently the Director of Stem Cell-based Drug Discovery with the Cell Therapy Group where he serves as a consultant for the regenerative medicine industry. Dr. Hambor was formerly the Chief Executive Officer of CellDesign, Inc., a global research and development company that specialized in the development of customizable stem cell tools, primary cells, and reagents for applications in drug discovery & research. Prior to founding CellDesign, Dr. Hambor was the CEO of Cognate BioServices, a contract manufacturer of cell-based products providing GMP-quality cells for clinical trials and pre-clinical studies. Previously, Dr. Hambor was an Associate Research Fellow at Pfizer. He spent his early years as a cellular and molecular biologist in the Inflammation and Immunology therapeutic areas. Dr. Hambor later joined the Genetic Technologies Department where he focused on applying stem cell technology in drug discovery for over 10 years, eventually coordinating global efforts in stem cell research as part of the Genetically Modified Models Center of Emphasis. Dr. Hambor also holds an adjunct faculty position at Connecticut College where he teaches Immunology. He is author of over 10 patents and 25 peer-reviewed scientific publications, and has been invited to lecture on his work at numerous international conferences. He is an active member of multiple scientific societies, serving as a member of the steering committee for the New York Academy of Sciences, organizing conference programs and chairing panel sessions. He is a scientific consultant for Expedition New England and a member of the Board of Directors for Vivo Biosciences. Dr. Hambor attended Miami University of Ohio where he graduated with BA and MS degrees in Microbiology. He received a PhD in Pathology from Case Western Reserve University and subsequently moved on to Yale where he did post-doctoral studies in Immunology.

Konrad Hochedlinger, PhD

Harvard Stem Cell Institute

Konrad Hochedlinger is an Associate Professor at the Department of Stem Cell and Regenerative Medicine of Harvard University, a Principal Faculty at the Harvard Stem Cell Institute and an Investigator at the Massachusetts General Hospital Cancer Center and Center for Regenerative Medicine. He received his BSc in biology and his PhD in genetics from the University of Vienna. From 1998-1999, he worked with Erwin Wagner at the Research Institute of Molecular Pathology in Vienna and from 2000-2006 as a Visiting Graduate Student and then Postdoc with Rudolf Jaenisch at the Whitehead Institute/MIT. During his stay at the Whitehead Institute, he worked on nuclear transfer in mice to show that terminally differentiated lymphocytes and malignant melanoma cells remain amenable to reprogramming into a pluripotent state. In his own lab, Dr. Hochedlinger continued work on nuclear reprogramming by focusing on a novel method that had been previously developed by Dr. Yamanaka and involves introducing defined transcription factors into somatic cells, generating induced pluripoent stem (iPS) cells. Dr. Hochedlinger’s lab reproduced and improved this technology and contributed to an understanding of its mechanism. For example, his team showed that iPS cells can be generated without the use of integrating viruses, thus eliminating a major roadblock for their potential use in therapy. He is currently using the mouse and human system to further elucidate the mechanisms of in vitro reprogramming. Dr. Hochedlinger is a Kimmel and V Scholar and has been awarded the NIH Director’s Innovator Award in 2007, the International Society of Stem Cell Research Young Investigator Award and the Howard Hughes Medical Institute Early Career Award in 2009.

Timothy Kamp, MD, PhD

University of Wisconsin

Timothy Kamp is Professor of Medicine and Physiology at the University of Wisconsin – Madison. Dr. Kamp received his BS from the University of Notre Dame and attended medical school at the University of Chicago, receiving his doctorate in Pharmacological and Physiological Sciences and degree in medicine. Dr. Kamp completed his Internal Medicine residency training and fellowship in Cardiovascular Medicine at Johns Hopkins Hospital in Baltimore. He joined the faculty of the Division of Cardiovascular Medicine at the University of Wisconsin in 1996. Kamp also serves as director of the University of Wisconsin Stem Cell and Regenerative Medicine Center. Dr. Kamp’s research focuses on human pluripotent stem cells and their applications to cardiovascular research and cardioregenerative medicine. In collaboration with Dr. James Thomson, Kamp first demonstrated that the human embryonic stem cells can differentiate into the various types of functional cardiomyocytes found in the human heart including atrial, nodal and ventricular myocytes. Dr. Kamp’s current research is refining the conditions to differentiate human embryonic stem cells and induced pluripotent stem cells into defined populations of cardiac progenitor cells and cardiomyocytes for research and clinical applications. His laboratory is actively investigating human models of inherited cardiac diseases created using induced pluripotent stem cell technologies. Kamp and colleagues are engaged in preclinical studies in animal models of myocardial infarction evaluating various strategies employing stem cells for cardiac repair. Dr. Kamp is also co-founder of Cellular Dynamics International, a company focused on applying stem cell technologies to human health.


A Chemical Approach to Controlling Cell Fate

Sheng Ding, PhD, Scripps Research Institute

Recent advances in stem cell biology may make possible new approaches for the treatment of a number of diseases. A better understanding of molecular mechanisms that control stem cell fate as well as an improved ability to manipulate them are required. Toward these goals, we have developed and implemented high throughput cell-based phenotypic screens of arrayed chemical and gene libraries to identify and further characterize small molecules and genes that can control stem cell fate in various systems. This talk will provide latest examples of discovery efforts in my lab that have advanced our ability and understanding toward controlling stem cell fate, including self-renewal, survival, differentiation and reprogramming of pluripotent stem cells.

The Importance of Relevant Cells for Drug Discovery

John Hambor, PhD, Cell Therapy Group

The cell is the most important tool in pre-clinical drug discovery and development. The safety and efficacy of all drugs is directly related to the extent to which their pharmacological action alters human cellular physiology. The high level of failure of late stage drug candidates in the clinic has revealed a general loss of translation in pharmacology using traditional cell lines for pre-clinical studies. These increased clinical trial attrition rates indicate that the successful development of safe and efficacious new drugs will require better pre-clinical understanding of the mechanisms by which pharmacological agents influence cellular physiology. Since many biochemical mechanisms are unique to individual cell types, the phenotypic cellular response to compounds that modulate selective targets should be assessed using physiologically and clinically relevant cell types. Although primary cells ideally serve this purpose, their availability, variability and expandability is severely limited. Accessibility to a variety of human cell types is now possible by the directed differentiation of renewable pluripotent human stem cells into desired target cell types, providing a stable supply of cells for a range of applications in drug discovery and toxicity testing. The utilization of human pluripotent stem cells in drug discovery now spans from early target identification and validation studies, via the use of functional human cells in screening and pharmacokinetic studies, to the use of various stem cells technologies in toxicological testing. Recently, pluripotent cells have been employed as screening tools for the development of new regenerative drugs that can activate and mobilize endogenous stem cell populations residing in various tissues throughout the human body. This talk will focus on emerging platforms using pluripotent cells and their application in state-of-the-art functional cellular assays and automated high throughput screening systems. Recent progress towards the successful identification, validation and development of drug candidates will be highlighted.

Factors Influencing Nuclear Reprogramming

Konrad Hochedlinger, PhD, Harvard Stem Cell Institute

My lab is studying the mechanisms of cellular reprogramming using transcription factor-mediated conversion of somatic cells into induced pluripotent stem (iPS) cells. For example, we have identified biomarkers to track and prospectively isolate intermediate cell populations during the reprogramming process and are currently using these to understand the transcriptional, epigenetic and proteomic changes in cells undergoing reprogramming. In addition, we have shown that terminally differentiated beta cells and lymphocytes can be reprogrammed into iPS cells, thus demonstrating that induced pluripotency is not limited to rare adult stem cells as has been suggested. Interestingly, however, we discovered that immature hematopoietic cells give rise to iPS cells more efficiently than any tested mature cell types, suggesting that the differentiation stage of the starting cell can influence the efficiency of reprogramming. We have further identified the p53 and p16/p19 tumor suppressor pathways as roadblocks during the reprogramming process, pointing out similarities between pluripotent cells and cancer cells. One major roadblock for the therapeutic use of iPS cells is the fact that integrating viruses are used to deliver the reprogramming genes to cells, resulting in genetically altered iPS cells. By using adenoviruses expressing the reprogramming factors transiently in cells, we were able to produce iPS cells devoid of any viral elements and thus any genetic manipulation. More recently, we have developed a “reprogrammable mouse” carrying a single doxycycline-inducible cassette with the four reprogramming genes in all tissues. We are employing this system to perform genetic and chemical screens to identify molecules important during the reprogramming process as well as for comparative studies between iPS cells and embryonic stem cells.

Human Pluripotent Stem Cell-derived Cardiomyocytes for Drug Development

Timothy Kamp, MD, PhD, University of Wisconsin

Significant differences in the expressed proteins in heart cells exist across animal species including man. Therefore, access to human cardiomyocytes (CMs) that can be readily studied in vitro may provide more predictive cell-based systems for cardiac drug discovery and safety testing than available with animal models. Human embryonic stem cells (ESCs) and human induce pluripotent stem cells (iPSCs) can provide theoretically unlimited supplies of human cardiac myocytes. Furthermore, generation of iPSCs from genetically diverse samples will enable creation of libraries of iPSCs that will better reflect the genetic diversity of the population as well as generate disease-specific models. The differentiation of stem cells to CMs has been highly inefficient, but recent advances in directed differentiation techniques have allowed production of large quantities of cardiomyocytes. In addition, genetic selection strategies taking advantage of the cardiac-specific troponin-T promoter have allowed isolation of cell populations that are >90% CMs in spontaneously contracting cardiospheres or plated as monlayers of CMs. The CMs exhibit human cardiac action potentials and respond to β-adrenergic stimulation with increases in spontaneous rate and shorter action potential duration. Comparison of hESC-derived CMs and iPSC-derived CMs action potentials have revealed similar properties including CMs derived from vector and transgene free iPS cells. Characterization of the monolayer cultured CMs on microelectrode arrays have shown robust extracellular field potentials, and exposure to known QT prolonging agents increases the measured field potential durations as an indicator of toxicity. Overall, human pluripotent stem cells provide unprecedented access to large quantities of genetically defined human CMs for research, drug development and toxicology applications.

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