UCSF Launches Institute for Stem Cell and Tissue Biology

By Jennifer O'Brien

Capitalizing on the strength of its research and clinical enterprise, UCSF is launching the Institute for Stem Cell and Tissue Biology, providing a framework for close collaboration among investigators from the University's Schools of Dentistry, Medicine, Nursing and Pharmacy. The Institute unites under one umbrella two major UCSF programs exploring the nature of stem cells and other early-stage cells - the UCSF Program in Developmental and Stem Cell Biology and the UCSF Program in Craniofacial and Mesenchymal Biology. UCSF Chancellor J. Michael Bishop, MD, says the Institute creates an integrated force that will enable UCSF scientists to maximize their ability to gain fundamental information about human development, with an eye toward illuminating and treating a broad range of diseases and disorders. UCSF investigators in some 60 laboratories are studying stem cells and related cells of humans, mice, zebra fish and worms, exploring their role in nearly every organ of the body, including the brain, heart, pancreas, liver, blood, bone marrow, skin, prostate, ovaries and testes. The scientists are studying the cells to identify the genetic missteps that cause diseases such as amyotrophic lateral sclerosis (ALS) and some cancers, and that account for some cases of birth defects and infertility. They also are conducting studies aimed at using the cells to replace or restore tissues damaged in conditions including diabetes, heart disease, Parkinson's disease, spinal cord injury, osteoporosis, arthritis and craniofacial defects. "Our goal is to continue and expand the basic research that ultimately could lead to therapeutic strategies," says Arnold Kriegstein, MD, PhD, who directs the Institute. "The Institute provides the framework that will allow us to drive the exploration toward investigations with our clinical-research colleagues at UCSF Medical Center, which has the medical and intellectual infrastructure to translate discoveries into therapeutic strategies." Kriegstein, a prominent neural stem cell researcher, was recruited from Columbia University in 2004 to lead UCSF's Program in Developmental and Stem Cell Biology. The Program includes the UCSF Human Embryonic Stem Cell Center, in which scientists have created numerous new lines of human embryonic stem cells for study and established a repository for human embryonic stem cells. The Institute is co-directed by Rik Derynck, PhD, who directs the Program in Craniofacial and Mesenchymal Biology. This program focuses on the development and biology of bone, cartilage, muscle, fat and connective tissue, and on mesenchymal stem cells, which, depending on their location in the body, give rise to these tissues. Researchers are exploring how abnormalities in mesenchymal stem cells give rise to the many craniofacial (cranium and face) and skeletal anomalies in humans. Scientists also are investigating the possibility of turning mesenchymal cells that have begun to specialize as fat cells into other tissues, such as bone and muscle. The goal of this research is to treat craniofacial defects, osteoporosis and osteoarthritis, as well as bone that is destroyed in some cancers. "The Institute draws together two programs that are asking interrelated questions," Derynck says. "The discoveries we've made in the mesenchymal biology program indicate that there is much to learn about the potential of cells that are no longer stem cells - such as early-stage fat cells - to treat disease." Key faculty recruits The establishment of the Institute reflects the growth and integration of UCSF's program during the last year. Recently, the University has recruited three premier young stem cell scientists to the faculty, including: • Robert Blelloch, MD, PhD, from the laboratory of Rudolph Jaenisch, at the Massachusetts Institute of Technology and Whitehead Institute for Biomedical Research • Holger Willenbring, MD, from the laboratory of Markus Grompe, at Oregon Health & Science University • Emmanuelle Passegué, PhD, from the laboratory of Irving L. Weissman, of Stanford University School of Medicine Blelloch will be an assistant professor in the Department of Urology; Willenbring will be an assistant professor in the Department of Surgery, and Passegué will be an assistant professor in the Department of Medicine. (See addendum for information on the new recruits' research.) The University also recently recruited Linda Giudice, MD, PhD, who will chair the UCSF Department of Obstetrics, Gynecology and Reproductive Sciences. Giudice, who chairs the U.S. Food and Drug Administration's Reproductive Health Drugs Advisory Committee and is a nationally renowned physician-scientist at Stanford University School of Medicine, currently chairs Stanford's Human Embryonic Stem Cell/Nuclear Transfer Committee in Stanford's Program in Regenerative Medicine. She joins UCSF in October. Stem cell training program On another front, the University has developed plans for a major stem cell training program designed for graduate students, postdoctoral fellows and physicians. On Friday (Sept. 9, 2005), the Independent Citizens Oversight Committee (ICOC), responsible for awarding funds for stem cell research under California's $3 billion bond initiative, announced that UCSF would receive full funding for this program, one of the largest training program proposals submitted to the California Institute for Regenerative Medicine. The stem cell training program will be led by Renee Reijo Pera, PhD, co-director of the UCSF Program in Developmental and Stem Cell Biology's Human Embryonic Stem Cell Center and associate professor in the Department of Obstetrics, Gynecology and Reproductive Sciences, and Kevin Shannon, MD, professor in the Department of Pediatrics, who studies genes that normally regulate the growth of immature blood-forming cells that are mutated in leukemias. The program will include courses in developmental and stem cell biology, embryology, human disease and transplantation, training in the rigorous ethics required to carry out the basic research and regenerative medicine, and a mentored research program under the guidance of leading basic science and/or clinical stem cell investigators. "This is an extremely exciting time in UCSF's history," says Keith Yamamoto, PhD, executive vice dean of UCSF School of Medicine. "UCSF's Gail Martin co-discovered embryonic stem cells in mice in the early 1980s, and coined the term 'embryonic stem cell.' More recently, UCSF was one of two U.S. universities that launched the human embryonic stem cell field. Today, the University continues to advance the field. "The new UCSF Institute reflects the breadth of the University's research program in the stem cell field, and the collaborative spirit for which UCSF is known. This umbrella program will be a powerful integrating force that will speed important discoveries." UCSF's School of Dentistry, School of Nursing and School of Pharmacy each rank first nationally in funding from the National Institutes of Health (NIH). UCSF School of Medicine ranks third nationally in NIH funding. For further information about UCSF or its stem cell research programs, see www.ucsf.edu. ### Additional information on UCSF's new stem cell scientists: • Robert Blelloch, MD, PhD, one of the country's experts in somatic cell nuclear transfer, or therapeutic cloning, is using the technique to study the genetics and epigenetics of disease and development. • Holger Willenbring, MD, is exploring the way transplanted bone marrow cells fuse with liver cells, with the goal of using this technique to regenerate diseased livers and eventually other tissues. • Emmanuelle Passegué, PhD, is studying blood-forming stem cells, which could play a key role in treating blood cancers and restoring tissues in failing organs.