Use of Induced Pluripotent Stem Cells in Drug Discovery: Challenges and Opportunities

The Scientist brings together a panel of experts who parse the hope and hype in an effort to educate the audience about the successes and caveats of using iPSCs.

By | September 27, 2013

The Scientist brings together a panel of experts who parse the hope and hype in an effort to educate the audience about the successes and caveats of using iPSCs.

A lot of optimism and promise surrounds the use of induced pluripotent stem cells (iPSCs) for a number of drug discovery and development applications. Human-derived iPSCs are thought to be more physiologically relevant and better suited for modeling disease pathophysiology and for understanding a drug’s mechanism of action. Hence, cell-based in vitro screening using iPSCs is gaining recognition as a tool for disease modeling, predicting drug efficacy, and toxicology testing. However, technical challenges exist in culturing, differentiating, and characterizing these cells, and skeptics remain unconvinced about the validity of the results obtained.

The Scientist brings together a panel of experts who will parse the hope and hype in an effort to educate the audience about the successes and caveats of using iPSCs. Attendees can interact with the experts during the live webinar by asking questions and sharing their experiences using stem cells.

Meet the Speakers:

DR. KENNETH S. ZARET is the Joseph Leidy Professor in the Department of Cell and Developmental Biology at the Perelman School of Medicine, University of Pennsylvania. He is also the associate director of UPenn's Institute for Regenerative Medicine and the codirector of UPenn's Program in Epigenetics. His laboratory discovered the mechanism by which gene regulatory factors, termed “pioneer factors,” endow the competence of embryos to produce different types of cells. Zaret's laboratory also identified a dynamic signaling network that coordinately induces liver or pancreas cell fates in the embryo. The information is being used by many laboratories to direct the programming of liver and pancreatic stem cells for biomedical and therapeutic applications. Recently, his group used stem-cell technology to reprogram human pancreatic cancer cells and found that the reprogrammed cells could progress through the early stages of pancreatic cancer. The cells are being used as a means to discover marker proteins that are released from live human pancreatic cancer cells at early stages of the disease.

DR. LIOR GEPSTEIN obtained both his MD and PhD from the Technion - Israel Institute of Technology, Haifa, Israel. During his studies, he was involved in the development of a three-dimensional electroanatomical mapping technique, which became the state-of-the-art method for treatment of complex cardiac arrhythmias. Currently, he is a professor of physiology and medicine (cardiology) at the Technion’s Faculty of Medicine and heads the Sohnis Family Research Laboratory for Cardiac Electrophysiology and Regenerative Medicine. Gepstein's research focuses on basic and clinical cardiac electrophysiology, stem cell biology, early cardiac tissue development, and the establishment of novel gene and cell-based strategies for the treatment of different cardiac disorders. His group was among the pioneers in developing unique strategies to differentiate cardiomyocytes from human embryonic stem cells and human induced pluripotent stem cells. More recent studies from his group demonstrated the important implications of these technologies for several basic and translational cardiovascular research fields such as developmental biology and physiology, drug development, disease modeling, personalized medicine, and cell therapy and tissue engineering. /p>

DR. STEVEN A. GOLDMAN is the URMC Distinguished Professor of Neuroscience and Neurology at the University of Rochester Medical Center, and codirector with Maiken Nedergaard of its Center for Translational Neuromedicine, in which he also holds the Dean Zutes Chair in Biology of the Aging Brain. Goldman moved to Rochester in 2003 from the Weill Medical College of Cornell University, where he was the Nathan Cummings Professor of Neurology. A summa cum laude graduate of the University of Pennsylvania, he did his PhD studies in the lab of Fernando Nottebohm at the Rockefeller University in 1983, and obtained his MD from Cornell in 1984. Goldman interned in medicine and did a residency in neurology at New York Hospital-Cornell and the Memorial Sloan-Kettering Cancer Center, before joining the Cornell faculty. He is interested in cell genesis and neural regeneration in the adult brain, with a focus on the use of neural stem and progenitor cells in treating demyelinating and neurodegenerative diseases, such as Huntington’s Disease, as well as for the treatment of glial diseases such as the pediatric leukodystrophies and multiple sclerosis. Goldman also has a strong interest in the conversion of resident stem and progenitor cells into brain cancers. He has published more than 200 papers in his field, over 100 as first or senior author.



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