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Testimony on National Institute of Child Health and Human Development's FY 1998 Budget by Dr. Duane Alexander
Director, National Institute of Child Health and Human Development
Accompanied by
Dr. Yvonne T. Maddox, Deputy Director, NICHD
Dr. Florence P. Hasseltine, Director, Center for Population Research, NICHD
Mr. Benjamin E. Fulton, Associate Director for Administration, NICHD
Ms. Donna S. Casady, Acting Budget Officer, NICHD
and
Dr. Harold Varmus, Director, NIH
Mr. Dennis P. Williams, Deputy Assistant Secretary, Budget, DHHS

U.S. Department of Health and Human Services

Before the House Appropriations Committee, Subcommittee on Labor, Health and Human Services, Education and Related Agencies
March 4, 1997


The NICHD is charged by Congress to conduct research on maternal and child health, the population sciences, and medical rehabilitation. The NICHD supports a wide range of research approaches to these areas, from the latest in molecular biology, through clinical trials, to epidemiologic surveys of various populations in our society, and development of new assistive technologies.

Last year, during the appropriations hearings, we reported good news of research advances and the awarding of the Nobel prize to two of our grantees. We will be continuing many of these themes this year, as these positive trends continue, including reduction in the infant mortality rate and high honors for NICHD-supported scientists.

For example, following on the four percent decline in infant mortality in 1994 that we reported last year, the trend continued with a 6 percent decline from 1994 to 1995, and the preliminary data for 1996 look equally encouraging. Since the NICHD was established in 1962, the U.S. infant mortality rate has declined by 70 percent. NICHD research advances have played a major role, particularly improvements in treating respiratory distress syndrome (RDS) and the "Back to Sleep" campaign aimed at reducing the risk of sudden infant death syndrome (SIDS).

The "Back to Sleep" campaign is based on NICHD research and, as its name implies, recommends that healthy infants be placed on their backs to sleep to reduce the risk of SIDS. Consequently, stomach sleeping has changed from 80 percent of babies to 25 percent, and deaths due to SIDS have fallen by more than 30 percent in the past three years. New Mexico, with an intensive Back to Sleep campaign, had a reduction of over 60 percent in SIDS deaths. Prior to the campaign, about 5,000 babies were lost each year to SIDS. Now, 1,600 fewer babies annually die of SIDS. The NICHD "Back to Sleep" campaign is being intensified in FY 97 and 98 with the goal of having 95 percent of babies sleeping on their backs and cutting SIDS deaths by more than half.

Many premature infants have trouble breathing. NICHD research previously revealed that such infants lack surfactant, a surface factor that keeps the inside of the lungs from sticking together and makes breathing easier. The development and administration of surfactant has markedly reduced deaths due to RDS and saves about $90 million a year in medical costs. Another new therapy, using inhaled nitric oxide, has just been shown by NICHD research to rescue many infants with a breathing problem called hypoxic respiratory failure and averts the need for a surgical procedure to oxygenate their blood. Inhaled nitric oxide is also much less expensive than the riskier surgery that often caused loss of one of the carotid arteries.

A brief historical note dramatically illustrates the progress from NICHD research. In 1963, President Kennedy's son Patrick was born prematurely and died of respiratory distress syndrome. Despite all his advantages, his doctors and his parents could only watch helplessly as Patrick struggled to breathe, because the cause of RDS was not understood and there was no treatment. Now, with treatment with surfactant, new respirators, better isolettes, and advanced intravenous fluid therapy, premature babies have a far better chance to live. When Patrick was born, an infant with RDS at his weight and gestational age had a 95 percent chance of dying; today, an infant at that weight and age has a 95 percent chance of living.

This has been a year of unprecedented acknowledgment of research supported by NICHD. It began with the award of the 1995 Nobel Prize to two scientists, Drs. Eric Wieschaus and Edward Lewis, whose basic research on embryonic development had been funded by NICHD for decades. Research on early development is of vital importance in understanding birth defects, the leading cause of infant mortality, and is carried on intramurally as well as extramurally. Senior investigators working in the field of early development are being joined by oustanding younger scientists, assuring the future of such research. In 1996, Dr. Ali Hemmati-Brivanlou and Dr. Lee Niswander, two NICHD grantees working in the area of birth defects, were chosen by the White House Office of Science and Technology Policy to be awarded Presidential Early Career Awards for Scientists and Engineers. These Awards recognize scientists for their outstanding work and exceptional potential for scientific leadership.

The awards continued last fall, when two NICHD intramural scientists, John Robbins, MD, and Rachel Schneerson, MD, were honored by the 1996 Albert Lasker Clinical Medical Research Award for their landmark development of a polysaccharide-protein conjugate vaccine for Hemophilus influenzae type b (Hib). The Lasker awards are often referred to as the "American Nobel Prizes."

The awards are treasured, but the research that merited the awards provided the true benefit to the world. Drs. Robbins and Schneerson joined the NICHD in 1970. At that time, the leading cause of acquired mental retardation in the United States was brain damage from meningitis caused by Hib. Even with effective antibiotic treatment of Hib infection, 5 to 10 percent of those who contracted it died, and about 30 percent had residual central nervous system damage, including mental retardation, deafness or seizures. Before the Hib conjugate vaccine was licensed and entered into routine use, there were 15,000 to 20,000 Hib cases yearly in the U.S. Now there are fewer than 100 cases a year. Where the vaccine is used, Hib meningitis has virtually disappeared.

Drs. Robbins and Schneerson have also developed an improved vaccine for pertussis (whooping cough), as well as investigational vaccines against typhoid fever, shigellosis (dysentery), E. coli 0157 (a life-threatening bacterial contaminant of food), antibiotic resistant pneumococcus, and tuberculosis. Shigella, the most common cause of dysentery in children, is responsible for about 650,000 deaths worldwide per year. The FY 1998 budget request will support trials of these vaccines.

Completing the cycle of the world's leading scientific awards, Dr. Ryuzo Yanagimachi, an NICHD grantee for 29 years at the University of Hawaii, was awarded Japan's equivalent of the Nobel prize, the 1996 International Prize for Biology. Dr. Yanagimachi's research has been of immense fundamental importance and has laid the foundation for human infertility treatment. Building in part on his research, the NICHD Infertility Research Centers Program, authorized by the NIH Revitalization Act of 1993, has completed several clinical studies on the causes of infertility. One study identified subtle changes in the reproductive hormone profile, particularly in levels of the hormone prolactin, that are related to fertility and infertility. Another study on male infertility has identified a defect in gonadotropin that may explain why a critical step in stimulating fertility fails to occur.

Not all our research has been honored with such distinguished prizes, but much of it has already made a difference in people's lives, or has the potential to do so. One dramatic achievement has been the development of effective treatment for the inherited disease cystinosis, a rare metabolic disease. NICHD scientists identified the basic defect in the disease, developed a drug to treat it, and tested it in a clinical trial. Prior to this treatment, affected children were severely stunted in growth, went blind from cystine crystals deposited in their eyes, lost their kidney function, and died by age 10 unless they received a kidney transplant. Today, with early diagnosis and drug treatment, the development and lives of the children are entirely normal.

In the category of yet-to-be-realized benefits, basic studies by our scientists of the developing nervous system have demonstrated that glial cells produce a highly potent protein, called activity-dependent neurotrophic factor (ADNF). ADNF is one thousand times more potent than any other known growth factor, preventing neuronal cell death at unprecedentedly low concentrations, and can protect brain cells in vitro from the toxic effects of the HIV envelope protein GP120 and from beta amyloid peptide, which is deposited in excess in the brains of Alzheimer's disease patients. It appears that the protective effects can be provided by a small peptide component of ADNF. We will be vigorously pursuing the potential therapeutic uses of this ADNF peptide with funds in the FY98 request.

NICHD is leading an expanded research effort on autism, a neurologically based condition that probably has a genetic basis. Most people with autism require assistance throughout their lives to help manage their disabilities, including repetitive behavior and severe difficulties in communication and social interaction. A conference on autism was held in 1995, followed by a solicitation for research grant applications focused on neurobiology and genetics. Funding these applications will markedly expand autism research and bring new scientists into the field.

Abnormalities of brain development can result in children having difficulties in learning. Almost one-half of all the students receiving special education have learning disabilities. NICHD researchers using non-invasive neuroimaging methods have mapped a number of critical brain systems that are involved in the ability to read, and have found that dyslexic individuals manifest significant differences in one or more of these neural systems. Screening procedures have been developed for early identification of reading disabilities. Longitudinal studies using a new NICHD-developed phonics approach with children at risk for reading disabilities (about 20 percent of the total school population) in ordinary classrooms in California and Texas have resulted in approximately 97 percent of this group learning to read. To reach the President's goal of each child learning to read by grade three, such research must continue and be integrated into the classroom. Funds requested for FY98 will support this effort.

At any time, any of us can suffer an injury or an illness that changes our entire life. Christopher Reeve has turned his own tragedy into a remarkable stimulus for research. There is no question that the research needs are great: approximately 250,000 people in the United States have spinal cord injuries, and about 10,000 more are injured each year. NICHD supports numerous studies of rehabilitation for spinal cord injury. One of these studies demonstrated that theophylline can help stimulate the breathing of patients like Mr. Reeve and lessen their dependence on ventilators.

Beyond spinal cord injury, an estimated 35 to 43 million Americans have conditions that result in a limitation of life activities. Thanks to NICHD research, a new generation of assistive technology is becoming available. Novel imaging techniques facilitate the rapid fabrication of prosthetic and orthotic devices that are customized to fit individual users. An advanced, portable oxygen source will help school children with breathing problems, and an electronically controlled knee-lock will improve lower limb bracing to increase the mobility of people with congenital spinal defects, muscular dystrophy, or spinal cord injury. Funds in the FY98 request will continue this work.

NICHD studies issues related not only to individuals, but to families and populations as well. NICHD leads a government-wide initiative to improve federal statistics and research on fatherhood; and to develop policies to assist and encourage men in their role as fathers. As more women join the workforce, the issue of child care increases in importance. Last year, early results of the NICHD Study of Early Child Care showed that, in and of itself, child care by non-maternal figures does not adversely affect the mother-child relationship. This study continues to examine other important issues in the first seven years of life. A major study of adolescent health is concluding and will soon be available to help educators, health care planners and policy makers to understand the social changes and structures influencing teenagers and their needs.

Mr. Chairman, the fiscal year 1998 President's Budget request for NICHD is $582,032,000. I will be pleased to answer any questions.


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