Scientific Report 2006
The Institute for Childhood and Neglected Diseases
Institute for Childhood and Neglected Diseases (ICND) was established to apply cutting-edge
research to understand the basic mechanisms underlying diseases of childhood and
orphan diseases that lack efficacious treatments. Diseases in both these categories
often affect populations in developing countries, where the health infrastructure
may be too poor to support major research efforts on these problems.
Examples of such diseases include malaria,
epilepsy, mental retardation, cystic fibrosis, chronic pain, and sleep disorders.
The human and economic costs of these diseases are staggering. According to the
World Health Organization, each year, the microorganism that causes malaria infects
300 million persons, and the disease kills approximately 1 million persons. About
90% of the people who have malaria are in Africa, where the annual costs associated
with the disease are $12 billion. Malaria is the leading cause of childhood mortality
in African countries.
Epilepsy is another widespread and costly
disease. It affects about 2.3 million persons in the United States and accounts
for $12.5 billion in medical costs and reduced productivity each year.
Mental retardation is another condition
that affects children everywhere; about 1% of American children 310 years
old are mentally retarded. During the 19951996 school year, about 600,000
6- to 21-year-olds with mental retardation in the United States received special
educational services, at a cost of about $3.3 billion.
Housed in a state-of-the-art, 54,000-square-foot
building on the east side of the Scripps Research campus, the ICND is a focus group
within Scripps Research for young scientists who are working in areas relevant to
the ICND mission. The concept of the ICND grew out of conversations in 1996 and
1997 among Scripps Research president Richard Lerner; John Moores, who was interested
in supporting research on illnesses that affect people in developing countries;
and the brothers Bernard and Marc Chase, who were interested in supporting research
on childhood diseases. John and Rebecca Moores, Bill Bauce, and other automobile
enthusiasts donated a number of vintage automobiles, which were auctioned to support
the ICND. The Moores went on to contribute a valuable coin collection, as well as
pledging $5 million to be awarded over 5 years.
The Human Genome Project has led to a
deeper understanding than ever before of the mechanisms underlying human disease.
The ability to study the draft mouse and human genomes in parallel is providing
an unprecedented opportunity to create a road map for assigning a physiologic function
to all of the 35,000-45,000 human genes. This formidable challenge requires complex
multidisciplinary approaches that allow scientists to create and implement the most
powerful research tools available. Investigators at the ICND use genomics, proteomics,
and advanced microscopic imaging technologies; develop many novel transgenic animal
models; and aggressively apply these technologies in an effort to understand the
mechanisms of action of a variety of diseases and conditionsmalaria, mental
retardation, neurodegenerative diseases, neuropathic pain, deafness, sleep disorders,
migraines, and epilepsy, for exampleand to devise treatments for these maladies.
ICND scientists plan to systematically study not only the genes associated with
these abnormalities but also the interactions between the genes in living model
ICND researchers have already been recognized
by the international scientific community during the first 5 years of the institutes
existence. In December of 2002, 3 of the highly prized Top Ten Breakthroughs of
the Year of Science magazine were results produced by ICND researchers working
on the malarial genome, mechanisms of pain perception, and processes important for
sleep disorders and seasonal depression. This remarkable level of achievement speaks
to the investment made in these research areas and bodes well for further rapid
progress in the near future.
Faculty Areas of
|Steve A. Kay, Ph.D.
||Molecular mechanisms of circadian rhythms and sleep disorders, genetics of anxiety, novel targets in neurodegenerative diseases
|William E. Balch, Ph.D.
||Protein trafficking and the molecular basis for the hereditary childhood disease cystic fibrosis
|Kristin Baldwin, Ph.D.
|| Molecular biology of the sense of smell, genetic mechanisms governing neural circuit development in the olfactory system and cortex
|Shelley Halpain, Ph.D.
|| Organization and function of the neuronal cytoskeleton, mechanisms underlying potential treatments for Alzheimers disease and repair of neuronal damage after trauma
|Mark Mayford, Ph.D.
||Molecular basis of cognitive function, including learning and memory disabilities and mental retardation
|Ulrich Müller, Ph.D.
|| Molecular cell biology of mechanosensory perception and childhood deafness
|Ardem Patapoutian, Ph.D.
|| Ion channels and receptors involved in nociception and neuropathic pain
|Elizabeth Winzeler, Ph.D.
||Functional genomic approaches to identifying targets in Plasmodium, the parasite that causes malaria