The Children's Hospital of Philadelphia has a long and distinguished tradition of research that has spanned more than 80 years and positioned the Hospital as a world-renowned pediatric research center. The many research breakthroughs at Children's Hospital have improved the lives of countless children not only in the Philadelphia region, but throughout the world.
Among the historical research breakthroughs that have made Children's Hospital an international pioneer in pediatric medicine are vaccines against measles, mumps and rubella, the development of a balloon catheter for use in cardiology, and the generation of methods for changing sickle-shaped red blood cells. By fostering collaborations between clinical and basic scientists the Hospital's traditional “bench to bedside” philosophy has resulted in an array of other major scientific achievements.
Building upon its solid foundation in both clinical and laboratory investigation, the Hospital and its Research Institute continue on the course to pediatric research preeminence.
Research today at Children's Hospital reflects the institution's commitment to improve child health and concentrates on basic, translational and clinical research on issues of importance and relevance to child health. With more than 500 investigators, research staff in the thousands and externally sponsored awards exceeding $141 million, The Children's Hospital of Philadelphia Research Institute continues its groundbreaking research on diabetes, neonatal seizures, childhood cancer, hemophilia, pediatric heart disease, cystic fibrosis, nutrition disorders, hypercholesterolemia, mental retardation, AIDS, sickle cell disease and numerous other diseases and disorders that affect children. Among its distinguished investigators are members of the Institute of Medicine, investigators of the Howard Hughes Medical Institute, Pew Scholars in Biomedical Sciences, and NIH Merit Scholars.
The Children’s Hospital of Philadelphia Research Institute engages in numerous activities and research programs to fulfill its mission of advancing the health of children by turning scientific discovery into medical innovation. It is through such a mission that the Research Institute is the preeminent institution in the world dedicated to translational research for children.
RESEARCH OVERVIEW
In addition to the traditional academic structure of the clinical departments, research at The Children's Hospital of Philadelphia Research Institute is organized by scientific discipline. Our goal is to foster interdisciplinary collaboration and innovation. We use two main organizational units to accomplish this goal: Research Affinity Groups and Centers of Emphasis.
Investigators throughout the Institution benefit from the services provided by the core facilities, which provide investigators with sophisticated research equipment, knowledgeable advisors and experienced support staff.
RESEARCH AFFINITY GROUPS
Pledging to be a leader in pediatric research, The Children's Hospital of Philadelphia Research Institute has witnessed phenomenal growth over the last decade. This growth is evident not only in the number of laboratory and clinical investigators conducting innovative research but also in the unique and cutting-edge programs that are the hallmark of research at Children's Hospital.
As part of the Hospital's overall strategic plan, the Research Institute continues its expansion, with the aim of achieving a level of visibility that will help the Institute achieve research preeminence.
Recognizing the challenges that often accompany growth, the Research Institute looked for a unique way to manage the needs of its investigators, foster interdisciplinary collaboration and better position the institute to compete for decreasing federal research funding. The solution: Research Affinity Groups.
An organizing principle of The Children's Hospital of Philadelphia Research Institute, the Research Affinity Group concept reflects the increasingly interdisciplinary and multi-disciplinary nature of many key research questions. In addition, the affinity group structure aligns with the emerging national consensus that multidisciplinary centers are essential to advancing the nation's research agenda.
The Institute designed the Research Affinity Group structure to build on areas of existing strength, identify new and important research areas, and explore important, broad and interdisciplinary scientific questions that may have an impact on the health of children.
CHOP researchers now collaborate across disciplines more easily to address issues of central importance to children's health. This collaboration has the potential to link multi-talented investigators with common research interests who are widely dispersed through the Institute.
The Institute currently has nine Research Affinity Groups, encompassing the areas of fetal biology and therapy; genes, genomics and pediatric disease; health and behavior; metabolism, nutrition and physical development; mitochondria; neuroscience; normal and malignant hematopoiesis; proteins; and vaccines and immunotherapies.
CENTERS of EMPHASIS
Children's Hospital makes a significant investment in translational research. This investment includes the creation of Centers of Emphasis, established to target effort and resources to areas of research that will have the greatest impact on translating basic research findings to medical innovations.
These centers are directly linked to the strategic plan of the Children's Hospital and are charged with fulfilling the specific purpose called for in the plan. Center directors are senior leaders in the Institute and have responsibility for recruitment and space allocation.
Center for Applied Genomics
The Center for Applied Genomics is one of the world's largest genotyping programs and the only center at a pediatric hospital to have access to state-of-the-art high-throughput genotyping technology at this scale. The program detects gene variations and links them to particular illnesses, and focuses on some of the most prevalent diseases of childhood — asthma, obesity, ADHD and diabetes, among others — as well as pediatric cancer, all of which are thought to involved the contributions of multiple, interacting genes.
The goals for the center, led by Hakon Hakonarson, M.D., Ph.D., are to generate new diagnostic tests for childhood diseases and use this diagnostic knowledge to guide physicians to the most appropriate therapies.
Center for Autism Research
The Center for Autism Research coordinates and supports research into the causes of the autism spectrum disorders (ASDs). The Center's programs of research are predicated on the belief that effective treatments will follow from a better understanding of causal mechanisms. Led by Robert T. Schultz, Ph.D., in collaboration with other Children's Hospital faculty, the CAR establishes a broad-based research program aimed at fundamental discoveries into causes of the ASDs.
The Center establishes programs of research focused on developmental, neurobiological and genetic mechanisms of the ASDs, with a particular emphasis on understanding the individual differences across the spectrum. Affiliated faculty also engage in research to evaluate the current standard of care for patients with an ASD and to test the effectiveness of promising new treatments.
Center for Biomedical Informatics
An innovative, interdisciplinary and academically focused group that facilitates the utilization of biomedical information, the Center for Biomedical Informatics provides the expertise and infrastructure needed to maximize the value of information relevant to all biomedical research, education and clinical activities occurring at Children's Hospital. This endeavor blends the disciplines of bioinformatics and clinical information, which themselves require excellence in and integration of various knowledge domains, including biology, medicine, statistics, mathematics, linguistics and computer science.
The aim of CBMi, led by Peter White, PhD, is to empower researchers, clinical staff, patients and families to most effectively utilize the ever-expanding totality of pediatric health information. In turn, these processes are expected to result in more effective pediatric healthcare interventions.
Center for Cellular and Molecular Therapeutics
The Center for Cellular and Molecular Therapeutics facilitates rapid translation of pre-clinical discoveries into clinical application. One of few such programs based at a pediatric institution, the center collaborates with other major programs to pursue new therapies for inherited and acquired disorders. Howard Hughes Medical Institute Investigator Katherine High, M.D., Division of Hematology leads the center, which also serves as an educational resource for investigators, clinicians, students, patient families and the general public.
The center has dedicated resources and personnel to help facilitate rapid translation. For example, the cGMP facility produces clinical-graded vectors in accordance with Food and Drug Administration regulations. In addition, given the complicated nature and the government's stringent regulations of cell and gene therapy, the center guides and assists investigators through the regulatory approval process.
Center for Childhood Cancer Research
The Center for Childhood Cancer Research, directed by John Maris, M.D., represents a highly integrated basic, translational and clinical research environment dedicated to eradicating the pain and suffering caused by cancer in children. This goal will be realized by bringing together the diverse talents of investigators in the Hospital's renowned multidisciplinary program in pediatric cancer research, patient care and genomics.
The Center's organization supports an environment where basic scientists interact with master clinicians around the central theme of improving cure rates through translational research initiatives. Recruitment of leading talent in areas that can facilitate this progress, spanning the laboratory and clinical research ends of the spectrum, enable the Center's mission. Key to the Center's success is translating the latest scientific findings obtained from cutting-edge basic research into innovative clinical trials designed to dramatically improve the cure rates for pediatric cancers while simultaneously eliminating long-term side effects.
Center for Developmental Biology
Disorders and illnesses of childhood are unique from those in adults as a result of development. Thus, understanding pathobiology within the context of development is essential for the diagnosis, management, cure and prevention of pediatric diseases. The goal of the Center for Developmental Biology is to increase our understanding of these developmental issues in order to support the Hospital's mission of being the leader in caring for children.
The center employs basic research focused on elucidating the mechanisms of developmental processes. The interdepartmental and interdivisional nature of the center augments and elevates ongoing research efforts at Children's Hospital. Led by Jeffrey Golden, M.D., the center aspires to implement a "bench to bedside and back to the bench" paradigm inherent when developmental biology research is tightly linked to the ongoing care of children. In addition to providing leadership in developmental research, the center provides training for the next generation of pediatric physician-scientists, and will help Children's Hospital to maintain a leadership role in defining healthcare practice and policy.
Center for Injury Research and Prevention
The Center for Injury Research and Prevention is a comprehensive pediatric trauma research facility dedicated exclusively to addressing injury, the leading cause of death for children and adolescents. The center advances the safety of children, adolescents and young adults through research into the origin and nature of childhood injuries, and develops effective interventions that will prevent the recurrence of those injuries. The center's cornerstone programs, Partners for Child Passenger Safety and the Young Driver Research Initiative, are ongoing research partnerships with State Farm® focused on mitigating motor vehicle crash injuries and death to children and young drivers.
Led by Flaura K. Winston, M.D., Ph.D., and Dennis R. Durbin, M.D., M.S.C.E., the center uses a multidisciplinary approach, drawing on a team of scientists from the fields of emergency medicine, pediatric trauma, pediatric and adolescent development, epidemiology and biostatistics, bioengineering, computational engineering, psychology, behavioral development, communications and health education. These different points of view form the foundation of a methodology in which research into the pre-event, event and post-event causes of injury and trauma can lead to action and ultimately make an impact on our nation's youth. The center's findings are not only published in peer-reviewed medical and scientific journals but are translated into recommendations for parents, educators, policymakers and product manufacturers by the center's outreach and advocacy professionals.
Center for Pediatric Clinical Effectiveness
The mission of the Center for Pediatric Clinical is to discover and disseminate knowledge about best practices in the management of pediatric disease. Led by Ron Keren, M.D., M.P.H, the center provides infrastructure for training in and performance of clinical effectiveness research — research aimed at understanding the best ways to prevent, diagnose and treat diseases in children.
The center builds on the existing research expertise and infrastructure at Children's Hospital to create an environment and opportunities for the exchange of ideas among clinical effectiveness researchers, facilitate the performance of clinical effectiveness research through a pilot grant program and assistance with projects that use existing national and local databases, and educate the next generation of clinical effectiveness researchers in the methods of clinical epidemiology. In addition, the center aims to partner with other Hospital organizations to improve the care of our patients and disseminate research findings that define the most effective healthcare for children.
PolicyLab
The PolicyLab Center of Emphasis ensures that Children's Hospital's research findings make a direct impact on local and national systems of pediatric healthcare. The center employs an innovative and comprehensive strategy of interdisciplinary research, program evaluation, and policy work to facilitate policy and program interventions that improve the health and well-being of the most vulnerable children.
By consulting with policymakers, foundation experts, and other key stakeholders who are directly involved in advocacy and implementation, PolicyLab is able to design research approaches that guide policy reform by building or refuting a base of evidence. These interactions also allow the center to identify practice innovations that require systematic investigation. The center is led by David Rubin, M.D., M.S.C.E.; Susmita Pati, M.D., M.P.H.; and Kathleen Noonan, J.D., all of the Division of General Pediatrics.
CLINICAL RESEARCH
There are hundreds of clinical research trials underway at The Children’s Hospital of Philadelphia. These programs, aimed at providing new or improving existing treatments for a myriad of pediatric diseases and conditions, are a key component to the overall research program at the Research Institute. Such research is needed to ensure that the rapid advances in biomedical sciences can be efficiently moved into patient-oriented research in a scientifically rigorous, pediatric centric manner. To answer the questions and address some concerns parents may have about enrolling their children in clinical research studies, the Research Institute recently released the “Parents’ Guide to Research” Web site that provides valuable information on clinical research so educated decisions on participation can be made. The guide, located at http://www.research.chop.edu/research/parents_guide_to_research/, includes general information about clinical research, the oversight of clinical research at Children’s Hospital and resources from other valuable Web sites that contain information that may be of interest to those interested in taking part in clinical research.
DISCOVERIES to the MARKETPLACE
The Children's Hospital of Philadelphia is a world-class academic medical center devoted to the healthcare of children where federally sponsored research grants and contracts, philanthropic donations, and corporate funding contribute to advancements in basic and clinical research.
The Office of Technology Transfer (OTT), one of the critical administrative units within The Children's Hospital of Philadelphia Research Institute, actively and aggressively seeks industry partners for research collaborations and licensing opportunities. These industry relationships are mutually beneficial because they facilitate the commercialization of research and inventions to advance the healthcare of children.
Two important licensed technologies reflect CHOP’s eminence in pediatric vaccine development: RotaTeq, Merck’s vaccine against rotavirus was developed at CHOP and protects millions of children from a common gastroenteritis. Synflorix, GSK’s pneumococcal vaccine was launched in Europe in 2009. This vaccine is created using techniques developed at CHOP. It protects children from acquiring pneumococcal infections, including those that occur in the middle ear.
CHOP generally is awarded eight to ten US patents each year and OTT typically receives fifty to sixty new invention disclosures each year.
EDUCATION
As one of the nation's leading pediatric research institutions, The Children's Hospital of Philadelphia Research Institute has a responsibility to train the next generation of researchers. This commitment to training serves not only to support and promote the research enterprise at Children's Hospital, but also to enhance the professional development of investigators, trainees and staff to ensure that the overall research community consists of highly trained and responsible researchers.
The Children's Hospital of Philadelphia Research Institute is dedicated to research and training at all levels. Our research is enriched by including scientific perspectives from investigators at the height of their careers and those who are exploring research as their future career. There are opportunities at the CHOP Research Institute for students in high school, undergraduates, graduate students, postdoctoral fellows and medical fellows as well as faculty-level investigators.
While much training on how to conduct research takes place within the research groups, the Office of Responsible Research Training, a key administrative unit within the Research Institute, provides numerous learning opportunities for investigators and research staff. These opportunities include Responsible Conduct of Research, human subjects education, clinical research coordinator certification, and principal investigator training, among others.
In addition, Children's Hospital is the recipient of several highly competitive training grants that offer additional opportunities in areas relevant to advancing pediatric research.
BUILDING ON THE STRONG FOUNDATION OF CUTTING-EDGE RESEARCH
The Research Institute’s reputation as the preeminent institution conducting translational research for children received a significant boost when it recently opened the doors to its new state-of-the-art, environmentally responsible research facility.
Launched with an initial gift of $25 million from Ruth M. and Tristram C. Colket, Jr., the 12-story structure houses high-profile, cutting-edge programs for advancing research in pediatric diseases, with additional programs scheduled to move in over the coming months.
The $504 million project encompasses 700,000 square feet--four new laboratory floors, administration and conference space, and a two-story ground floor housing a lobby and cafeteria. There are an additional four stories below grade consisting of infrastructure and laboratory support space.
The Colket Translational Research Building will enable scientists across a variety of disciplines, all dedicated to developing treatments for a specific pediatric disease, to work side-by-side. Physical proximity will foster close collaboration, thus providing more opportunities for new ideas and expedited results. In keeping with the building's research focus, many of the current floors feature state-of-the-art laboratories, which can be reconfigured easily as teams grow, and their research evolves. The building will expand along with Children's Hospital's research program; it can accommodate 12 additional floors to reach 24 stories.
For more information, go to www.chop.edu/research.
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