The gift will create a center that will allow basic scientists and clinicians to use the tools of biotechnology -- genetics, genomics and proteomics -- to create ways to diagnose childhood diseases earlier, predict which children will respond to treatment and determine which children will have serious side effects from therapies.
''We know that all of the issues that face children are a result of their genetics and their environment,'' said Harvey Cohen, MD, PhD, chair of pediatrics and chief of staff at Lucile Packard Children's Hospital. ''We will study genetic and environmental influences on the body. We will study chromosomes and genes that the children have, how the genes are turned on or off, what proteins are made, how they may be altered and determine whether proteins and other substances change their location as a result of a disease process.''
The $700,000 gift is the first investment of its kind by the hospital in the medical school. The hospital's twin goals are financial and programmatic sustainability and medical and technological pre-eminence. ''Over the past several years, Packard Children's Hospital has invested in programs that have made it sustainable; because of this, the hospital is now able to invest in innovation such as the biotechnology effort,'' said Christopher G. Dawes, Packard's president and chief executive.
Philip Pizzo, MD, dean of the School of Medicine, notes that finding ways to translate basic knowledge into practical applications for patients - so-called translational medicine - is a cornerstone of the school's strategic plan, "Translating Discoveries."
"There has been a vast amount of knowledge that has emerged in recent years from the field of biotechnology," said Pizzo, the Carl and Elizabeth Naumann Professor of Pediatrics and of Microbiology and Immunology. "This gift will enable us to apply some of that knowledge to greatly improve the health and well-being of children. As a pediatrician, I very much appreciate the opportunity to work with Packard on this very important project."
The hospital and School of Medicine have recruited James W. Schilling, PhD, to serve as senior scientist and director of the Children's Biotechnology Core, a joint hospital and medical school appointment. Shilling comes from Sugen, Inc., now Pfizer, where he has served as principal scientist and director of protein chemistry.
Alan Krensky, MD, headed the national search for the senior scientist, a position that he views as ''connecting clinicians, clinician scientists and basic scientists'' and helping guide the effort to bring research from the laboratory to the patients and bring the knowledge gained from the patient experience back to the lab.
''To do this in a setting of a children's hospital on the campus of a major technology university is state-of-the-art,'' said Krensky, who is chief of the division of immunology and transplantation biology in the Department of Pediatrics at the School of Medicine. ''There are several great children's hospitals and several great technology universities, but no one offers this on a single campus as we can. We have both institutions, and now they're being connected.''
The biotechnology effort at Packard Children's Hospital and the Stanford University School of Medicine has begun focusing on important areas in pediatrics:
* Acute myelogenous leukemia. With current treatments, half of children are cured and half will die of their disease. ''By studying the genetics, genomics and proteomics of leukemic cells,'' said Cohen, who is also chairman of the Bio-X Interdisciplinary Initiatives Program, ''we hope to be able to identify those children who we can safely and effectively treat with what we have and those who need different treatments.''
* Kawasaki disease: Using comparative proteomics, researchers hope to develop a better tool for early diagnosis of this inflammatory disease, which affects young children and can result in cardiac problems. * Diabetes: Approximately half of certain relatives of children diagnosed with Type 1 (autoimmune) diabetes will develop the disease themselves within five years. ''We will be studying the serum of these individuals to see if we can predict those individuals who will develop diabetes,'' Cohen said.
* Necrotizing enterocolitis: Premature babies are at risk of developing this illness, which causes extensive ulceration of the intestines. Researchers at Packard Children's Hospital and the school of medicine will measure the proteins in infants' plasma before and after feeding to see if they can determine what changes are associated with the disease development.
''These investigations are at the forefront of pediatrics,'' Cohen said. ''Through the use of biotechnology, we are helping develop the 21st Century diagnostics and therapeutics for childhood illnesses.''
About Lucile Packard Children's Hospital
Lucile Packard Children's Hospital at Stanford is a 240-bed hospital devoted entirely to the care of children and expectant mothers. Providing pediatric medical and surgical services associated with Stanford University Medical Center, Packard offers patients locally, regionally and nationally the full range of health care programs and services - from preventive and routinecare to the diagnosis and treatment of serious illness and injury.
http://www.lpch.org/
About Stanford University School of Medicine
The mission of the School of Medicine is to be a premier research-intensive medical school that improves health through leadership and through a collaborative approach to discovery and innovation in patient care, education and research. http://www.med.stanford.edu/school/