There has been remarkable progress in our understanding of the development and function of the immune system in health and disease. The regulation of immunity impacts profoundly on many of the conditions that pediatricians treat. The objective of this program is to enable PAS attendees coming from diverse disciplines to understand the language of modern immunology and the translation of recent advances in basic science to the diagnosis and treatment of diseases of children. This course will address three areas: immunology of infectious diseases, immunology of auto-immunity and tumor immunology.

Opening remarks
E. Richard Stiehm, UCLA Children's Hospital, Los Angeles, CA

Opening remarks
Beverly J. Lange, Children's Hospital of Philadelphia, Philadelphia, PA

Immunology of Infectious Disease
Katherine F. Luzuriaga, University of Massachusetts Medical School, Worcester, MA

Immunology of Autoimmunity
Betty Diamond, Albert Einstein College of Medicine, Bronx, NY

Tumor Immunology
Robert H. Vonderheide, University of Pennsylvania, Philadelphia, PA

This session will review the development and clinical usefulness of blood/blood products and derivatives used in the treatment of patients with hematologic and oncologic diseases and the bleeding patient. The role of these novel and often modified products in selected patient groups will be discussed.

Introduction
Naomi L. C. Luban, Center for Cancer and Blood Disorders, Children's National Medical Center, Washington, DC

Transfusion Support of the HSCT Patient
Terry Gernsheimer, Puget Sound Blood Center, Seattle, WA

Transfusion Support of the Chronically Transfused Patient with Sickle Cell Anemia
Naomi L. C. Luban, Center for Cancer and Blood Disorders, Children's National Medical Center, Washington, DC

Novel Therapies for Acute Bleeding
Guy Young, Children's Hospital of Orange County, Orange County, CA

Pathogen Reduction—Risks, Benefits and Hidden Benefits for GVHD
Lawrence Corash, Cerus Corporation, Concord, CA

The pipeline of new therapies flowing from the bench to the bedside is extremely long and tortuous, with many "valves" that must be opened. Expertise is required in both basic and clinical research to conduct Investigational New Drug-directed early phase human clinical trials or to become skilled in the design and implementation of pre-clinical studies necessary to effectively progress basic observations into human trials. For the academician, the challenges can be legion. This session will begin with an example of the infrastructure required for an academic center to foster translational research. The symposium will then cover three of the major aspects of new drug development. First, we will explore the preclinical selection of appropriate targets and the models in which to test them. Second, we will explore the pharmaceutical pipeline to give participantsknowledge of new clinical agents. Third, we will learn about coordinating the early stage clinical trials with regulatory agencies. We will close with an academician¹s perspective of the end of the pipeline based on recent gene therapy trials for hemophilia.

Introduction to Translational Research: Opening the Valves of the Pharmaceutical Pipeline
Timothy Cripe, Cincinnati Children's Hospital Medical Center, Cincinnati, OH

Pediatric Malignancies Provide Unique Cancer Therapy Targets
Jeffrey Toretsky, Lombardi Comprehensive Cancer Center, Washington, DC

Exploiting Genetic Defects for Targeting Oncolytic Viruses to Pediatric Cancers
Timothy Cripe, Cincinnati Children's Hospital Medical Center, Cincinnati, OH

A Comparative and Preclinical Approach Toward Drug Development
Chand Khanna, National Cancer Institute, National Institutes of Health, Bethesda, MD

Developing Drugs in the Era of Targeted Therapy
Pamela S. Cohen, Novartis Pharmaceuticals Corporation, Florham, NJ

Ask Not What CTEP Can Do for You…Moving Agents Through the Pipeline
Barry Anderson, National Cancer Institute, National Institutes of Health, Rockville, MD

Special Delivery: Novel Approaches and Challenges to Gene Delivery
Mark Kay, Stanford University Medical Center, Stanford, CA

Many children living with chronic and life-threatening conditions experience pain and other distressing symptoms. Control of pain and symptoms is the foundation upon which competent palliative care is built. Yet children and families suffer when they encounter pediatricians and other professionals who are ill-prepared to offer them competent and compassionate palliative and end-of-life care. The Institute of Medicine (IOM) report on Improving Palliative and End-of-Life Care for Children and Their Families (2002) calls upon pediatric health professionals to address the needs of children and families for comprehensive palliative care services. In addition, routine assessment and management of pain is now a required component of patient care according to the Joint Commission for the Accreditation of Health Care Organizations (JCAHO). This session will outline basic tenets of pain and symptom management for children and adolescents, their implementation across care settings and consideration of the continued barriers to full implementation of these care standards.

Assessment and Management of Pain in Children and Adolescents
Neil L. Schechter, St. Francis Hospital and Medical Center, Hartford, CT

Reducing Barriers to Effective Pain and Symptom Management at the End of Life
Nancy Hutton, Johns Hopkins University School of Medicine, Baltimore, MD

Palliative Pain and Symptom Management in Pediatric Tertiary Care Settings
Joanne Wolfe, Dana-Farber Cancer Institute and Children's Hospital, Boston, MA

Pediatric Pain and Symptom Management in Home Care and Hospice
Speaker To Be Determined
 

Visit in February 2004 for the abstract program schedules.
 

This mini course will set the stage for several discussions of particular issues of major importance and interest. What is "preparedness" and what are the real risks of continuing terrorism in the United States? What is the current status of preparedness in the U.S. hospital and public health systems? How do children differ from adults in terms of response to weapons of mass destruction (chemical, biological and radiological)? How do these differences matter in disaster planning? Are the needs of children being incorporated in local, state and federal disaster plans? Smallpox, anthrax and other biological threats: Where do we stand? What do we do? Nuclear power plants, nuclear weapons, dirty bombs and potassium iodide: What do we know? The mental health consequences of terrorism: What have we learned since 9/11, how do we prepare children for an increasingly vulnerable world, building resiliency and sustaining a positive vision. The new pediatric agenda: What do we have to teach students, residents and pediatricians about the pediatric aspects of terrorism planning. Children and exposure to weapons of mass destruction: science and the essential research agenda.

Introduction
Paul H. Saenger, Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, NY

Welcome and Context
Irwin Redlener, National Center for Disaster Preparedness, Columbia University Mailman School of Public Health, New York, NY

Pediatric Preparedness for Terrorism and Disasters
David S. Markenson, Columbia University Mailman School of Public Health, New York, NY

Biological Weapons of Terror: What Pediatricians Need to Know
Theodore J. Cieslak, U.S. Army Research Institute of Infectious Diseases, Ft. Detrick, MD

Helping Children and Families Cope with Terrorism
David J. Schonfeld, Yale University School of Medicine, New Haven, CT

Radiologic Terrorism, Children and the Question of Potassium Iodide
Thomas P. Foley, University of Pittsburgh, Children's Hospital of Pittsburgh, Pittsburgh, PA

The program will highlight new developments in clinical management of patients with chronic granulomatous disease as well as an update on the progress of gene therapy of CDG stem cells. There will also be an update on the management and diagnosis of patients with the Shwachman-Diamond syndrome, and, finally, there will be an update on diagnosis, treatment and pathophysiology of severe chronic neutropenias.

Overview
Laurence A. Boxer, Mott Children's Hospital, University of Michigan, Ann Arbor, MI

Severe Chronic Neutropenia Pathogenesis and Treatment
David Dale, University of Washington, Seattle, WA

Shwachman-Diamond Syndrome Pathogenesis and Treatment
Melvin H. Freedman, Hospital for Sick Children, Toronto, Canada

Chronic Granuloma Disease, Pathogenesis, Management
Harry Malech, National Institutes of Health, Bethesda, MD

Visit in February 2004 for the poster program schedules.
 

Dr. Castle is a pediatric hematologist/oncologist and is chair of the Department of Pediatrics at the University of Michigan. She will discuss opportunities in academic pediatrics with special emphasis on careers in the subspecialties.

Academic Pediatrics: Challenges and Opportunities
Valerie P. Castle, C.S. Mott Children's Hospital, Ann Arbor, MI
 

Visit in February 2004 for the abstract program schedules.
 

TLRs (Toll-like receptors) are a family of transmembrane germ line coded pattern recognition receptors that bind structural motifs common to pathogenic organisms. These structural motifs include endotoxin, products of gram+ organisms, fungi and mycobacteria, as well as DNA and RNA structures common to bacteria and virus but not mammalian cells. The TLRs are expressed by diverse cell types. TLR signaling initiates the innate immune/inflammatory host response to pathogens and also initiates antigen processing for acquired immunity.

Moshe Arditi will review the recent progress in understanding how children respond to pathogens. Maria Abreau will explore how immune signaling is central to both the maintenance of normal gut function and how chronic GI disease may develop. Christopher Karp will then explore how immune signaling relates to the hygiene hypothesis regarding the striking increase in the prevalence of both allergic and autoimmune diseases in children in Westernized countries over recent decades. The goal is to provide an update about newly described mechanisms signaling inflammation/immunity that are central to multiple homeostatic and disease processes in children.

Toll Like Receptors—Bridging Innate and Adaptive Immunity
Moshe Arditi, Cedars-Sinai Medical Center, UCLA School of Medicine, Los Angeles, CA

TLR Signaling in the Gut in Health and Disease
Maria Abreu, Cedars-Sinai Medical Center / UCLA School of Medicine, Los Angeles, CA

Signaling the Hygiene Hypothesis
Christopher Karp, Cincinnati Children's Hospital Medical Center, Cincinnati, OH

Children who are victims of violent behavior or merely observers of violence may learn destructive or self-destructive patterns of behavior. Violence is a major public health problem. This symposium will focus on breaking the cycle of violence and will showcase speakers who are working on violence prevention in the pediatric emergency department, school and community. The speakers will demonstrate what can be done by physicians who see the importance of this issue and the ways in which we can make a difference.

Violence Prevention in Primary Care: Moving from Public Health to Private Practice
Robert D. Sege, Tufts-New England Medical Center, Boston, MA

Beyond Treat and Street: Violence Prevention in the Emergency Department
Joel Fein, The Children’s Hospital of Philadelphia, PA

Efforts in the Community
Sheryl A. Ryan, University of Rochester School of Medicine, Rochester, NY

Visit in February 2004 for the abstract program schedules.
 

Visit in February 2004 for the poster program schedules.
 

The National Children’s Study is a national prospective, longitudinal study of environmental effects, including physical, chemical, biological and psychosocial effects, on child health and development. The goal of the study is to improve the health and well-being of children. The study will examine these environmental effects on the health and development of more than 100,000 children across the United States, following them from before birth until age 21. The study is led by a consortium of federal agency partners: the U.S. Department of Health and Human Services, including the National Institute of Child Health and Human Development (NICHD); the National Institute of Environmental Health Sciences (NIEHS); the Centers for Disease Control and Prevention (CDC); and the U.S. Environmental Protection Agency (EPA). For additional information, visit the website at http://www.nationalchildrensstudy.gov/.

The National Children’s Study—An Overview
Duane Alexander, NICHD, National Institutes of Health, Bethesda, MD

The National Children’s Study—Methods
Peter C. Scheidt, National Institutes of Health, Bethesda, MD

Children’s Health and Environmental Exposures: The Most Important Unanswered but Answerable Questions
Michael Weitzman, The AAP Center for Child Health Research at the University of Rochester, Rochester, NY

Despite the dramatic increase in patient and graft survival following solid organ transplantation over the past decade, complex and potent immunosuppressive regimens have led to significant complications. In this multidisciplinary symposium, the pathophysiology and therapy of infections, lymphoproliferative disease and growth abnormalities following solid organ transplantation will be reviewed. In addition, the problem of non-compliance with complex immunosuppressive regimens—a major cause of organ loss/dysfunction post transplantation—will be reviewed.

Infections Following Solid Organ Transplantation
Michael Green, University of Pittsburgh School of Medicine, Pittsburgh, PA

Post-transplant Lymphoproliferative Disease: Pathophysiology and Treatment
Vikas R. Dharnidharka, University of Florida Health Science Center, Gainesville, FL

Growth Disorders Following Solid Organ Transplantation
Leona Cuttler, Case Western Reserve University, Cleveland, OH

Compliance with Complex Medical Regimens Post-transplantation: Anticipatory Therapies
Robert Wells, Children's Hospital Central California, Fresno, CA

This symposium will cover new methods to detect fungal infections and indications for new drugs. CMV detection and treatment outcome will be discussed in the post-HSCT patient. Finally, other viral infections that can cause significant morbidity in the immunocompromised patient, such as HHV6 and adenovirus, will be discussed.

Introduction
Nancy Bunin, Children's Hospital of Philadelphia, Philadelphia, PA

New Approaches to Diagnosis and Treatment of Invasive Fungal Infections in Immunocompromised Pediatric Patients
Thomas Walsh, National Cancer Institute, Bethesda, MD

New Developments in the Diagnosis and Management of CMV and Adenovirus Infection in Stem Cell Transplant Recipients
Michael Boeckh, Fred Hutchinson Cancer Research Center, Seattle, WA

HHV6 Infections in Immunocompromised Patients
Danielle Zerr, Children's Hospital and Regional Medical Center, Seattle, WA

The application of imaging technologies to solving questions in biology and medicine is revolutionizing medicine by accelerating analyses in situ and in vivo and providing new perspectives on biological processes as diverse as development, neoplasia and injury repair. In this plenary session, three internationally recognized speakers will focus on developmental processes and discuss how these new imaging technologies are providing dynamic insights into the genetic and epigenetic mechanisms that underpin hematopoiesis and vascular development.

Introduction
Lisa M. Guay-Woodford, University of Alabama at Birmingham, Birmingham, AL

Dynamic Imaging of Fluid Forces in Developing Mouse Vasculature
Mary Dickinson, Beckman Institute–Caltech, Pasadena, CA

Microscopic Imaging of Angiogenesis
Donald M. McDonald, University of California, San Francisco, CA

Watching Hematopoietic Stem Cell Engraftment and Hematopoiesis in Living Animals
Christopher H. Contag, Stanford University School of Medicine, Stanford, CA

Questions from the audience
 

This program will focus on emerging trends in hematopoietic stem cell transplantation for children with primary immunodeficiencies. Topics to be covered will include the use of alternative donor sources such as unrelated cord blood, new approaches with lower intensity pre-transplant conditioning regimens and an update on transplant outcomes for some of the more common primary immunodeficiencies such as SCID, WAS and HLH.

Hematopoietic Stem Cell Transplant for Primary Immunodeficiency Disorders: Update of Outcomes and Unrelated Cord Blood
Alexandra H. Filipovich, Children's Hospital Medical Center, Cincinnati, OH

Introduction

Nonmyeloablative Stem Cell Transplant for Congenital Immunodeficiencies
Kanchana Rao, Great Ormand Street Hospital for Children, London, United Kingdom

Hematopoietic Stem Cell Transplant for Hemophagocytic Lymphohistiocytosis: Results from the Multi-institutional National Marrow Donor Program HLH Protocol
K. Scott Baker, University of Minnesota, Minneapolis, MN

Worldwide, more than 1,500 children per day become infected with HIV through mother-to-child transmission. Currently there are 2.7 million children living with HIV infection across the globe, >90% of whom reside in developing countries. While there have been enormous successes in the prevention and treatment of pediatric AIDS in the United States and Europe, it remains an open question as to how effectively these public health gains can be replicated in the poor countries of the world, which bear the greatest burden of disease. Efforts to develop an HIV vaccine appropriate for preventing infection among the world's children and adolescents are finally under way on a global scale. We will discuss these issues and accompanying controversies as they apply to the children of the developing world.

AIDS in Children—A Global Public Health Crisis
David L. Pugatch, Hasbro Children's Hospital and Brown Medical School, Providence, RI

Preventing Mother-to-Child Transmission of HIV in Developing Countries—Successes, Failures and Challenges
Catherine M. Wilfert, Elizabeth Glaser Pediatric AIDS Foundation, Santa Monica, CA and Washington, DC

HIV Treatment for Children—Can the Successes of Rich Countries Be Duplicated in Resource-Poor Settings?
Mark W. Kline, Baylor College of Medicine, Houston, TX

Finding an AIDS Vaccine That Works for the World's Children
Richard A. Koup, Vaccine Research Center, National Institutes of Health, Bethesda, MD

In 1929, a visionary band of 25 pediatric physician-scientists, spurred by their passion for science and their commitment to children, founded the Society for Pediatric Research. In the intervening 75 years, pediatric investigators have wrought a breathtaking suite of therapeutic breakthroughs—from the care of premature neonates, to effective immunoprotection from childhood disease, to novel therapeutic strategies for childhood leukemia. More recently, the completion of the Human Genome Project has identified new opportunities to delineate the impact of genetic variation on childhood disease expression.

Four leaders in Pediatric Research will celebrate the accomplishments of the past 75 years and share their perspectives about the challenges that lay before us.

Introduction
Gail J. Demmler, President, Society for Pediatric Research

The Problem of Prematurity: The Impact of Surfactant and the Challenge of Long-Term Morbidity
Samuel Hawgood, University of California Medical Center, San Francisco, CA

The Problem of Childhood Infectious Diseases: The Impact of Vaccine Development and the Challenge of Emerging Infections
Margaret K. Hostetter, Yale University School of Medicine, New Haven, CT

The Problem of Childhood Leukemia: The Impact of Combination Chemotherapy and the Challenge of Neuro-cognitive Morbidity
Alan L. Schwartz, Washington University Medical Center, St. Louis, MO

The Problem of Genetic Disease: The Impact of the Human Genome Project and the Challenge of Translation to Innovative Therapy
Edward R. B. McCabe, David Geffen School of Medicine at UCLA, Los Angeles, CA

The potential applications for using regenerated cells and tissues to treat injury and disease are unlimited. Early stem research concentrated on the hematopoietic stem cells of the bone marrow, but stem cells are now known to exist in most organs of the body. Furthermore, it may be possible to return mature, differentiated cells to a undifferentiated, stem-like state. This symposium will first provide an overview of non-hematopoietic stem cells, then focus on two rapidly-progressing areas of research—those of regenerating nervous tissue and liver.

Neural Stem Cells: Developmental Insights May Suggest Therapeutic Options
Evan Y. Snyder,

Hepatic Stem Cells and the Potential of Liver Repopulation for Cell Therapy
Sanjeev Gupta, Albert Einstein College of Medicine, Bronx, NY