4.2: Scientific Knowledge
Increase basic scientific knowledge to improve human health and human development. Other Information:
Basic research contributes significantly to personalized health care and to increasing understanding of human makeup and biological
processes. Current and future basic research projects in HHS focus on those areas with the greatest potential for reduction
in excess morbidity and mortality, including brain function, human development, asthma and other respiratory diseases, cancers,
dementia, influenza strain mapping, and antimicrobial resistance. The performance indicators for this strategic objective
highlight research efforts related to major diseases, including cardiovascular disease and Alzheimer’s, and imaging tools
for the early detection of diseases, including cancer. Brain Research: The rising public health impact of disorders of the
nervous system makes neuroscience one of the most important scientific frontiers for biomedical and behavioral research in
this century. Discoveries in the areas of pain, alcoholism, drug abuse, autism, schizophrenia, depression, and other mental
disorders are increasing dramatically. NIH will build on these discoveries by continuing to support research to better understand
the processes of the brain, including improving imaging technologies to be able to visualize brain processes as they happen.
The increased understanding of the nerve circuits will pave the way for improved diagnosis and treatment of common diseases
such as depression, stroke, and epilepsy and reduced burden on the Nation in terms of both suffering and health care costs.
NIH will also support the Autism Phenome Project, which will identify various clinical characteristics and subtypes of autism
to facilitate research on genetic and other potential causes of autism and to guide applied research related to treatment
approaches. Alzheimer’s Disease: Alzheimer’s disease, the most common cause of dementia among people older than 65, is one
of the most serious threats to the Nation’s health and economic well-being. Currently, 4.5 million Americans are affected
by the disease; that number is expected to almost triple by 2050. Those suffering from Alzheimer’s disease advance inexorably,
from early, mild forgetfulness to a severe loss of mental function and inability for self-care. Existing research suggests
that Alzheimer’s disease pathology begins to develop in the brain long before clinical symptoms yield a diagnosis. The ability
to make an accurate early diagnosis of Alzheimer’s disease would allow targeted intervention before cognitive loss becomes
significant. NIH is searching for valid, easily attainable biological markers that could help identify biological markers
for early disease. For example, NIH will support research to examine one promising approach that involves using coated gold
nanoparticles as bioprobes to measure the concentrations of substances that correlate with Alzheimer’s disease. NIH will also
continue to support the Alzheimer’s Disease Neuroimaging Initiative. The 5-year, 50-site project represents the most comprehensive
effort to date to develop neuroimaging and other biomarkers for the changes associated with mild cognitive impairments and
Alzheimer’s disease. The ongoing Genetics Initiative will also support the development of resources necessary for identifying
late-onset risk factor genes, associated environmental factors such as physical activity and diet, and their interactions.
Human Development: NIH is committed to funding a diverse portfolio of basic and translational research that addresses the
physical, psychological, psychobiological, language, behavioral, social-emotional, and educational development of children.
For example, the National Institute of Child Health and Human Development (NICHD) at NIH has taken a leadership role in advancing
scientific knowledge regarding the acquisition of reading and mathematics skills, related learning disabilities, and language
development and second language acquisition, as well as child maltreatment, childhood obesity, and the attainment of school
readiness skills. Additionally, understanding normative brain development and its relationship to cognitive, social emotional,
and behavioral development is important in finding the causes of myriad childhood disorders related to mental retardation,
mental illness, drug abuse, and pediatric neurological diseases, which can continue into adulthood. To define the healthy
ranges in brain growth and development patterns in children as they mature, NIH-funded researchers are creating the Nation’s
first database of Magnetic Resonance Imaging measurement of normal brain development over time in children and adolescents
in the United States. NIH is bringing together a diverse array of researchers to design and support a large scale longitudinal
study that uses state-of-the-art brain-imaging technologies and that collects clinical and behavioral data, which will be
used to develop analytical software tools. A special effort will be made to disseminate these data, and as a result, the scientific
community will have access to a Web-based, user friendly resource that integrates neuroanatomical and clinical/behavioral
data to examine brain-behavior relationships and relationships between physical maturation and brain development. Cancer Research:
NIH investment in cancer research is helping to make a real difference. In the United States, death rates from all cancers
combined dropped 1.1 percent per year from 1991 to 2001. Yet cancer remains a major public health problem with more than 1
million Americans per year diagnosed with some form of cancer. Despite significant progress, the cancer challenge remains
formidable, and NIH investment in basic cancer research remains critical. NIH will continue to support a broad range of basic
research to expand the understanding of cancer. Through the Cancer Genome Atlas Project, NIH will expand the capacity of the
cancer community to utilize information on cancer genes. NIH also will focus on a growing area of interest—understanding the
reaction of the body’s immune system to a developing tumor— because chronic inflammatory immune responses are known to exacerbate
certain cancers. Asthma: NIH supports a comprehensive asthma program to develop new approaches to prevent, treat, and control
asthma. Asthma exacerbations cause many of the negative effects of asthma, and management of asthma exacerbations accounts
for a large proportion of the estimated annual cost to the Nation’s economy. In contrast to the understanding of the origins
of asthma, little is known about the processes that occur during an acute episode; how worsening attacks are resolved; the
effect of attacks on future severity and frequency; and the long-term effects on lung physiology, function, and disease progression.
In order to develop new interventions to prevent and help resolve acute or worsening asthma episodes, NIH initiated a set
of basic, clinical, and translational studies to determine the molecular, cellular, and genetic causes of asthma exacerbations.
The long term goal is to identify and characterize two molecular pathways of potential clinical significance that may serve
as a basis for discovering new medications for preventing and treating the progression of this disease. The studies will address
diverse areas including the role of environmental triggers in enhancing airway hyper responsiveness; the relationship of environmental
factors to frequency and severity of asthma attacks; specific effects of initiating events on lung physiology and inflammation;
genetic approaches to individual susceptibility for worsening attacks; and the role specific immune and lung cells play in
asthma disease classification, chemistry, and physiology. Pandemic Influenza: HHS is working intensely against influenza.
The center of this work is the development of multiple vaccines against influenza virus. At the level of basic science, however,
NIH is collaborating with numerous public and private partners on an influenza sequencing project. This project will determine
the complete genetic sequences of thousands of influenza virus strains, providing the scientific community with data vital
to development of new vaccines, therapies, and diagnostics. Antimicrobial Resistance: Microbes once easily controlled by antimicrobial
drugs are causing infections that no longer respond to treatment with these drugs. In addition, new, serious, and unforeseen
infectious disease threats have emerged, including those posed by agents of bioterrorism. Because the existing repertoire
of antimicrobial medications may not provide an effective defense against newly emerging and resistant organisms and bioterrorism
agents in the future, there is a need to develop new treatments that may be effective against a range of pathogens. NIH is
working to develop a universal antibiotic, a drug effective against a wide spectrum of infectious diseases, to help address
these challenges. NIH also is expanding its capacity for medicinal and combinatorial chemistry, library and database resources,
and screening assays for use in identifying novel antimicrobial drugs.
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