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NIH Holds First Symposium on Human Embryonic Stem Cells

Scientists at the first NIH research symposium on recent progress and future promise of human embryonic stem cells (hESC) proved that, as in many things, timing and location are everything. Understanding the molecular and cellular processes that regulate normal human embryonic cell differentiation into multiple types of cells poses many challenges. Other challenges are embedded in understanding the unique capacity of these fledgling cells to be both pluripotent and indefinitely self-renewing.

On June 12, an overflow crowd in Masur Auditorium witnessed, in person and by webcast, the intriguing possibilities of emerging knowledge about hESCs and opportunities to interweave and to share breaking information. Principal investigators representing laboratories both within NIH and from across the country raised key questions about molecular signaling that leads to the differentiation of hESCs into specific cell types.

Dr. James F. Battey, Jr., director of the National Institute on Deafness and Other Communication Disorders and chairman of the NIH stem cell task force, opened the meeting with the hope and plan that this should be the first of many such symposia. The task force is a team of leading scientists who meet with and seek advice from scientific leaders in order to advance the field of stem cell research.

NINDS' Dr. Ron McKay

In introducing the first plenary panel devoted to discussion of the characterization of undifferentiated stem cells, NIEHS's Michael McClure harkened back to the Yankees' accidental pundit Yogi Berra's famous observation — "If you don't know where you're going, you'll be lost when you get there." Leading off the session, Ron McKay, a principal investigator in the NINDS intramural program, cautioned that in looking at therapeutic possibilities of hESCs in the future, it is imperative that the hESC "makes exactly the right kind of neuron." Daniella Gerhard from NCI described her investigation of the transcriptome of a number of currently available cell lines. Her presentation underscored the importance of shared resources. Mahendra Rao from NIA is also developing infrastructure resources for human embryonic stem cell research, and focused on understanding of developmental mechanisms that are likely to lead to novel approaches in preventing and treating neurological disorders of aging. Using a prototype for the targeted acquisition of desired neuronal phenotypes, Steven Goldman, an extramural investigator at Weill Medical College of Cornell University, described the induction and isolation of spinal motor neurons from hESC.

NIDCD director Dr. James Battey Jr. (foreground) listens to presentation as part of Masur Auditorium audience.

John Thomas from NHLBI introduced the second panel exploring topics related to differentiation of hematopoietic stem cells. George Daley from the Whitehead Institute described characterization of signaling pathways that trigger self-renewal. His team is using methods of viral gene transduction to accomplish this work. Dan Kaufman, from the University of Minnesota, focused his discussion on the role of stromal cell co-culture to promote differentiation into hematopoietic cells. Timothy Kamp, from the University of Wisconsin Medical School, described the ability of hESCs to provide a renewable source of distinct types of human cardiac myocytes. This ability may have application for pharmacological testing and possible therapeutic applications. Esmail D. Zanjani from Sierra Biomedical Research Corp., reported on his work using fetal sheep. His team's work shows that cells derived from hESCs can engraft and undergo multilineage differentiation. Gordon Zeller from the Mt. Sinai School of Medicine focused on ways to promote differentiation into one of the three developmental lineages: ectoderm, mesoderm and endoderm.

Participating in a panel discussion are (from l) Dr. John McDonald III, Washington University, St. Louis; Dr. Su-Chun Zhang, University of Wisconsin-Madison; and Dr. James Huettner, also of Washington University, St. Louis.

Thaddeus Golos, University of Wisconsin, presented his findings on hESC differentiation into placental trophoblast cells and on placental morphogenesis. James Huettner from Washington University in St. Louis emphasized in his discussion that "the development of successful therapies for neurodegenerative disorders involving hESC cells will require the functional evaluation of electrical signaling capability." Su-Chun Zhang, University of Wisconsin Medical School, described the neural specification of hESC, noting the importance of understanding neural induction and patterning in humans; this can lead to an optimized procedure for generating enriched or purified neuroepithelia and specialized neural cells. John McDonald III's group, also at Washington University in St. Louis, is investigating ultrastructural features of hESC that can lead to novel strategies of CNS repair in both mouse models and, eventually, in humans.

At the conclusion of the plenary session, Dr. Elias Zerhouni, NIH director, noted, "There is no better way to assess the state of the science than to have the best scientists who are really doing it assembled in one place together." Observing that the acceleration of research on hESCs must be built upon a foundation of facts, Zerhouni added, "I have a personal commitment and NIH is fully committed [to move] as fast as we can and provide the resources to do so...I want the science to drive the agenda."

Dr. Elias Zerhouni
A noontime poster session of work from six laboratories was followed by workshops on material transfer agreements and intellectual property issues of stem cell research; hESC research training; federal guidelines on stem cell research; NIH implementation; and culture techniques for hESCs.

The symposium was developed by a planning committee that included representatives from 9 institutes and the Office of the Director, NIH, including NIDCD's Battey, chair; L. Tony Beck, NCRR; Jill Carrington, NIA; Della Hann, OD; Eleni Kousvelari, NIDCR; Michael E. McClure, NIEHS; Belinda Seto, OD; Beth-Anne Sieber, NIMH; Richard J. Tasca, NICHD; John W. Thomas, NHLBI; Baldwin Wong, NIDCD; and Marion Zatz, NIGMS.

For more information about hESC research visit To view the webcast visit (check under VideoCasting).

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