The Benefits of Having a National Institute of Child Health and Human Development

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AAP, American Academy of Pediatrics, Hib, Haemophilus influenzae type b, NICHD, National Institute of Child Health and Human Development, NICUs, Neonatal intensive care units, NIH, National Institutes of Health, PSDP, Pediatric Scientist Development Program, SIDS, Sudden infant death syndrome

Acting on the request of President John Kennedy, Congress, in October 1962, passed legislation establishing a new Institute at the National Institutes of Health (NIH), the National Institute of Child Health and Human Development (NICHD). This legislation implemented the visionary proposal first put forward by Robert E. Cooke, MD, Chair of Pediatrics at Johns Hopkins, to fill a void in the NIH research spectrum by focusing on developmental processes and how they were affected by genes, disease, or environment, with a focus on the health impact on mothers and children. This action provided the opportunity to use the unparalleled resources of the federal government, provided by the American people, to advance a great cause: Improving the health and lives of mothers and children, in this country and worldwide, through research. I would like to share with you some of what has come from a part of this investment.

One of the keys to research success is training. Without a sound base of trained, skilled investigators, your field has no chance in the highly competitive world of winning research support. Nearly 25 years ago, the scientific leadership of Pediatrics asked me to support the convening of a meeting to plan a way to assure sound training of the scientific leadership for the pediatrics of the future. What came from that meeting at NICHD was the Pediatric Scientist Development Program (PSDP), funded by a new K12 grant from NICHD but contributed to by numerous pediatric organizations, including the American Academy of Pediatrics (AAP), Society for Pediatric Research, Association of Medical School Pediatric Department Chairs, and American Pediatric Society. It was highly selective in who entered the program and intensely mentored to ensure success in training in top-notch labs and entering an academic career. That PSDP grant continues today, growing and expanding under the nurturing leadership of Dr Peggy Hostetter. Because of its success but need for larger numbers, NICHD added a companion program, the Child Health Research Career Development Program. It was also supported by NICHD K12 grants, awarded to and located in 20 pediatric departments, and markedly augmented the numbers of pediatric scientists trained in the flagship PSDP.

Seeing the need for better organized and conducted clinical trials, the NICHD initiated two Networks for clinical trials in Obstetrics and Neonatology. These competitive, cooperative agreements support a standing group of maternal-fetal medicine units and neonatal intensive care units to design and conduct clinical trials under common protocols they develop and have provided the data to stop ineffective practices or expand effective ones. They have become so popular and effective that the concept has been extended to clinical trial networks in pediatric and obstetric pharmacology, pediatric intensive care, adolescent AIDS, and global maternal and child health.

Research conferences are an effective tool NICHD has used to translate research to practice or identify needed research. One outstanding example was the Consensus Development Conference on Antenatal Corticosteroids to enhance lung development in infants threatening to deliver prematurely. NICHD believed this was an underutilized therapy. The conference panel concluded that the data strongly supported that position and recommended routine use to enhance lung maturity in premature infants and prevent respiratory distress syndrome. The American College of Obstetricians and Gynecologists quickly concurred, and, within 3 years, antenatal corticosteroid use increased from 15% of candidate pregnancies to 85%, with a concomitant marked decrease in respiratory distress syndrome and preemie deaths.

Another way to translate research to practice is through public information campaigns. The prime example here is the Back to Sleep campaign to reduce sudden infant death syndrome (SIDS). Here, NICHD partnered with the AAP, assisting the expert committee appointed by the Academy in reviewing the research literature on sleep position and SIDS, and, when the AAP recommendation to change to back sleeping had only a small effect in 2 years, aggressively publicizing the AAP recommendation that babies should be put on their backs to sleep to reduce SIDS risk. NICHD called it the Back to Sleep campaign and, in 5 years, back-sleeping increased markedly and SIDS deaths declined by more than 50%.

Although vaccines for children are not an NICHD primary responsibility, the presence of Drs John Robbins and Rachel Schneerson in our intramural program has produced major vaccines for children. By introducing the concept of conjugation (binding a weak sugar antigen to a strong protein carrier antigen to increase immunogenicity), they developed the Haemophilus influenzae type b (Hib) conjugate vaccine. This vaccine, licensed by the Food and Drug Administration in 1987, quickly achieved universal use and has virtually eliminated Hib meningitis in the United States. It used to affect 15 000 to 20 000 children a year and was this country’s leading cause of acquired mental retardation.

Robbins’ and Schneerson’s conjugate technology has been used to develop effective vaccines for Pneumococcus, Meningococcus, and typhoid fever, as well, and they continue working on new vaccines.

The NICHD has also targeted newly emerging, specific concerns for children’s well-being. For example, when the AIDS epidemic began, NICHD scientists quickly recognized that children were infected, too. NICHD set up a new Pediatric, Adolescent, and Maternal AIDS Branch, specifically to address HIV infection in children and pregnant women, launched treatment trials in infected children that made antiretroviral drugs available quickly after they were approved for adults and tested these drugs to prevent maternal to child transmission of HIV. The initial trial in the United States produced a reduction of HIV transmission from 27% to 7%, and subsequent studies have brought the US transmission rate down to 1% to 2% and the rates in Asia and Africa to 4% to 10%.

Infant and young child day care was another concern addressed by NICHD research. With more than half of the mothers of infants and young children joining the work force, questions were raised about the effects on child development and family relationships from this major social change. NICHD tackled this issue by establishing a collaborative, 10-site, common protocol study of early child care and youth development. The reassuring results provided at ages 3 years, 5 years, 7 years, and later provided relief for young parents unsure about whether they were harming their child and guidance for programs to provide better quality care.

NICHD also identified newborn screening for genetic or metabolic diseases as an under-utilized and unevenly utilized technology and brought Dr Rodney Howell to NICHD to lead an effort to bring standardization and expansion to this field. There is now agreement on 30 disorders that should be universally screened for in every state, which is happening, and research is underway to develop screening techniques and treatments for more disorders.

Learning, in general, and learning to read in particular, are major milestones in child development. NICHD made major investments in studies of learning to read successfully and of learning disability and, under the leadership of Dr Reid Lyon, built a partnership with the Department of Education to co-fund and conduct clinical trials in the classroom of different approaches to teaching reading, simultaneously upgrading the quality of peer review and science in education research.

The Congress also assigned new responsibilities to NICHD. In 1990, Congress added a National Center for Medical Rehabilitation Research to NICHD, which has assured that the needs of children are addressed. At the AAP’s urging, research responsibilities for drug testing and evaluation in the Best Pharmaceuticals for Children Act were assigned to NICHD to provide guidance on generic drug dosage and safety for use in infants and children. Congress also assigned NICHD the leadership role to plan and conduct a National Children’s Study of Environmental Influences on Health and Development. Planning has been underway for what would be the largest and most expensive single research project the NIH has ever undertaken.

During its 47-year existence, the NICHD has invested $23 billion in research in pediatrics, obstetrician-gynecologist, and developmental and behavioral science, with a current budget of $1.3 billion a year. A measure of the benefit of having an NICHD is the impact of this research on people’s daily lives. Consider a newly married young couple today. If they want to postpone childbearing, their choice of contraceptive methods is enhanced by products and safety/effectiveness information from NICHD research. If they have difficulty conceiving, help is available from techniques developed with the assistance of NICHD research. The home pregnancy test kit they buy to check on their pregnancy status came directly from research in the NICHD intramural program. If problems develop in the pregnancy, improved treatments are available due to NICHD research. If parents are concerned about possible severe congenital anomalies or some genetic disorders, prenatal diagnosis assessed by NICHD research is available. Problems of newborn infants are addressed by improved resuscitation, respiratory care, intravenous fluids, and nutrition developed and evaluated by NICHD research. Every baby leaves the hospital with a bandage on its heel, where several drops of blood were obtained for newborn screening developed through NICHD research to detect and prevent harm from genetic or metabolic disorders. The first ride home is in a car safety seat promoted by NICHD research.

When their baby is put down to sleep for a nap or the night, it is placed on its back rather than its stomach to reduce its risk for SIDS, thanks to the NICHD-led Back to Sleep campaign. When their baby goes for its 2-month immunizations, the Hib vaccine it receives came from NICHD’s intramural labs, as did the conjugate technology for other vaccines. If the child goes to day care, the parents are comfortable with the knowledge that it is not harming and may be helping their child, thanks to NICHD research. When the child begins school, the mainstreaming of children with disabilities into regular classes and the teaching techniques for reading are a product of NICHD research.

These are but a few examples of the many contributions of NICHD research. They, along with many other discoveries, make a compelling case for concluding that there is not another institute at NIH whose research has had such a widespread and beneficial impact on people’s lives as the research from NICHD.