DEPARTMENT OF HEALTH AND HUMAN SERVICES
Dr. Duane Alexander
Director, National Institute of Child Health and Human Development
Fiscal Year 2001 President's Budget Request
for the National Institute of Child Health and Human Development
Mr. Chairman and Members of the Committee:
I am pleased to present the FY 2001 President's budget request for the National Institute of Child Health and Human Development (NICHD). The request of $810.5 million reflects an increase of $40.8 million over the comparable FY 2000 appropriation. Including the estimated allocation for AIDS research, total support requested for NICHD is $904.7 million, an increase of $45.4 million over the FY 2000 appropriation. Funds for the NICHD efforts in AIDS research are included within the Office of AIDS Research budget request.
The NIH budget request includes the performance information required by the Government Performance and Results Act (GPRA) of 1993. Prominent in the performance data is NIH's first performance report which compares our FY 1999 results to the goals in our FY 1999 performance plan. As our performance measures mature and performance trends emerge, the GPRA data will serve as indicators to support the identification of strategies and objectives to continuously improve programs across the NIH and the Department.
The NICHD seeks to assure that every individual is born healthy and wanted, that women suffer no adverse consequence from the reproductive process, and that all children have the opportunity to fulfill their potential for a healthy and productive life unhampered by disease or disability. In pursuit of this mission, the NICHD conducts and supports laboratory, clinical, and epidemiological research on the reproductive, neurobiologic, developmental, and behavioral processes that determine and maintain the health of children, adults, families, and populations.
Since this Institute was established 37 years ago, a major portion of our research has been devoted to better understanding the causes, treatments, and prevention of mental retardation. One by one, in large part as a result of the support for research from this Committee, causes of mental retardation are being eliminated:
- phenylketonuria or PKU - eliminated;
- congenital hypothyroidism - eliminated;
- hemophilus influenzae type b meningitis or Hib - eliminated;
- measles encephalitis - nearly eliminated;
- congenital rubella syndrome - nearly eliminated;
- bilirubin encephalopathy - nearly eliminated
Our progress toward understanding and eliminating the causes of mental retardation has continued during the last year. In a highly significant advance, Dr. Huda Zoghbi, an NICHD grantee in our Mental Retardation Research Center at Baylor, identified the gene responsible for Rett Syndrome, a mysterious condition that causes seemingly normal infant girls to lose their ability to walk and to develop symptoms of severe mental retardation. After years of exploration, researchers discovered the genetic difference between girls with Rett Syndrome and unaffected children. Girls with Rett Syndrome have a defective gene on one of their two X chromosomes. These girls have some normally functioning copies of the gene, so their symptoms are not immediately apparent at birth. However, between 6 and 18 months of age, these girls begin to exhibit the symptoms of Rett Syndrome when the function of the normal single gene is insufficient to meet the growing child' s needs. [This discovery also sheds light on why only females are affected by the syndrome. Males with the Rett Syndrome gene possess only the mutant version of the gene because they have only one X chromosome. Presumably, male fetuses with the gene for Rett Syndrome die before birth or soon thereafter because they do not have a back-up copy of the normal gene.] The gene that is abnormal in Rett syndrome, called MECP 2, controls the function of several other genes, so when it is defective, multiple other genes, including some that are essential for brain development and function, operate improperly. Based on this exciting discovery, the NICHD is encouraging investigators to try to find pharmacologic agents that can substitute for the control mechanism, and thereby reverse or prevent the progression of Rett Syndrome.
Another significant finding we reported this year may provide a way to reduce the risk of mental retardation for children born to women who have hypothyroidism during their pregnancy. We have known for many years that congenital hypothyroidism in children is associated with a lower IQ and we have eliminated that problem by screening all newborn infants. Now NICHD research has demonstrated that children born to mothers who have untreated hypothyroidism during pregnancy scored lower on IQ tests than children of healthy mothers, with 19 percent in the borderline or retarded range. However, when mothers with hypothyroidism were being treated for the condition, their children's IQ scores were virtually identical to those of children born to healthy mothers. This study suggests that screening women for hypothyroidism before or early in pregnancy may provide a way to prevent mental retardation. A protocol is in preparation to test this possibility in the 14 ob-gyn departments that are part of the NICHD Maternal-Fetal Medicine Network. This network is also studying ways to reduce the incidence of low birth weight, another significant cause of mental retardation.
NICHD has also provided important testing for a proposed new treatment of autism. In recent months a number of reports suggested a potential benefit to using secretin in the treatment of autistic children. We were intensely interested in these reports, but they contained no scientific data to assess the degree or duration of potential benefits. For this reason, NICHD launched a series of placebo-controlled studies to investigate potential benefits and risks of using secretin to treat autism. In results from the first of these studies, NICHD researchers found that treatment with the synthetic version of secretin offered no more benefit for children with autism than did treatment with placebo. Additional studies will seek to determine if secretin may be effective when given at various doses or on more than one occasion. We are also investigating whether secretin benefits autistic children with a particular group of symptoms and whether biological secretin is more effective than the synthetic version.
Another area in which the NICHD has both a deep concern and a deep commitment is the elimination of health disparities among minority populations. The Institute is developing a comprehensive and coordinated research plan for eliminating health disparities among racial and ethnic minorities. Our plan will address infant mortality, reproductive health, medical rehabilitation, and child and adolescent health. I would like to highlight some of the initiatives which illustrate our strong commitment to eliminating health disparities.
Over the last two years, the NICHD awarded funds to 20 departments of obstetrics and gynecology to develop young investigators in the field and prepare the next generation of principal investigators. We also support 15 Reproductive Science Research Centers. In FY 2001, the NICHD will enhance these programs by funding a program of Specialized Centers for Research in Reproductive Medicine in Minority Institutions that pairs minority institutions with established research centers. The goal of this program is to increase the capacity of minority institutions and investigators to conduct cutting-edge research in the field of obstetrics and gynecology, focusing on problems particularly prevalent among minorities.
The Institute's national Back to Sleep campaign, which urges caretakers to place infants on their backs to sleep, has met with significant success. In the five years since the campaign was launched, deaths due to Sudden Infant Death Syndrome (SIDS) have dropped 38 percent. Despite this overall success, both the SIDS rate and the rate of stomach sleeping among African-Americans remain more than double that of white infants. To address this marked disparity, the NICHD invited the leaders from a number of national African-American organizations, as well as officials from Federal, state, and municipal governments, to join us in developing and implementing strategies for reducing SIDS in African-Americans. The group identified the need for culturally sensitive materials and programs designed by and for African-American communities. The NICHD is committed to carrying out this strategy. As a first step, NICHD is conducting research with African-American caretakers such as parents, grandparents, relatives, and child care workers to identify more effective ways to communicate the Back to Sleep message. One component is a transit ad, which will be used first in the DC Metro system, and eventually in other cities around the country. The Institute's goal is to eliminate the racial disparity in infant back sleeping position within three years and hopefully thereby eliminate the racial disparity in SIDS rates.
We are also exploring ways to improve reading skills in populations of culturally and linguistically diverse students. Three years ago, the NICHD began a reading instruction research program with nine DC public schools. The purpose of the program was to determine whether applying what we have learned in other reading programs could be applied successfully with regular teachers in regular classrooms. Data from the Early Intervention Project are still being collected and analyzed, but preliminary data show a pattern of remarkable improvements in reading ability. For instance, reading scores in schools that have historically been at the 10 th to 15 th percentile have improved to better than the 50 th percentile. Moreover, the entire class in intervention schools is now performing at the national average. In a related area, the NICHD and the Department of Education this year are jointly soliciting research proposals for systematically studying the most effective ways to teach reading English to children whose primary language is Spanish.
In previous years, I reported on the research that led to the remarkable reduction in the rate of HIV transmission from mother to infant during pregnancy and birth. NICHD and NIAID research have made another important contribution to reducing maternal HIV transmission this past year. Grantees discovered that the amount of HIV in a pregnant woman's blood, known as maternal HIV viral load, is the prime risk factor for transmitting the virus to the baby. By focusing treatment on reducing the viral load during pregnancy, the risk of HIV transmission from mother to infant can be further decreased.
In the developing world, where logistics and the cost of multiple drug therapy for HIV are often prohibitive, research reported last year showed that administering the antiviral drug nevirapine to the mother just before delivery and to the infant just after birth can reduce HIV transmission significantly. NICHD and NIAID are now conducting studies to evaluate whether nevirapine, administered during the time a mother is breast-feeding can reduce the rate of HIV transmission through breast milk.
Pediatric Trauma Rehabilitation
We also plan to expand research for children and teens in the area of trauma. Injury is the leading cause of death for children five to 18 years old; violence is the third leading cause of death for this age group. However, many clinical treatments for trauma are tailored exclusively to adults and fail to consider the long-term effects of these interventions on a developing child. The NICHD is planning a multi-disciplinary, collaborative program to address this issue, led by the Institute's National Center for Medical Rehabilitation Research. This program will allow us to develop and assess therapies specifically targeted to the physical, emotional, and social needs of children. As part of this program, we will start a collaborative pediatric injury and trauma clinical trials network. Concurrently, we will be examining ways to actually prevent the risky behaviors that often result in injury and trauma.
In 1998, the NICHD held a consensus conference on traumatic brain injury or TBI. The panel identified specific concerns that require further study regarding the impact of TBI on children. Brain injuries can have a profound impact on new learning and future physical and mental development of children. Based on the panel's recommendations, the NICHD will establish specialized research programs on treatment tailored directly to the needs of young children with traumatic brain injury.
In the area of behavioral research, the Institute is identifying some of the major influences on the health and health behavior choices of young people. Since 1994, the Institute has supported The National Longitudinal Study of Adolescent Health, also know as the Add Health Study. The study has provided new insights into the ways that peers, families, schools and neighborhoods can influence positive health outcomes, as well as negative outcomes, such as violent behavior, smoking, drinking, illegal drug use, and sexual behavior. Data from this survey informed and will continue to inform public policy. With the increased funding provided by this Committee in FY 2000, the Add Health study will collect additional data from the full original cohort. This study will help identify the major determinants of health and health behaviors during the transition from adolescence to early adulthood.
Mr. Chairman, the support from this Committee for the research of the National Institute of Child Health and Human Development has contributed to the elimination and near elimination of some of the major causes of childhood diseases and lifelong disabilities. We are proud of this progress but we know we still face many daunting yet exciting research challenges. In the years ahead, with your continued support, I am confident that we will return to this room and report back to you that we have eliminated some of the causes of learning disabilities, that we have eliminated some of the causes of infant mortality, that we have eliminated some of the life-long adverse consequences of child-bearing, and that we have contributed in a significant way to eliminating the health disparities that separate racial and ethnic communities. I will be pleased to answer any questions you have at this time.