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The Institute for Childhood and Neglected
Diseases
The
Institute for Childhood and Neglected Diseases (ICND) was established to apply cutting-edge
research to understand the basic mechanisms that underlie diseases of childhood and orphan diseases
that lack efficacious treatments. Diseases of both 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 3–10 years old have mental
retardation. During the 1995–1996 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 Mooreses
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—malaria, mental retardation, neurodegenerative
diseases, neuropathic pain, deafness, sleep disorders, migraines, and epilepsy, for example—and
to devise treatments for these maladies. ICND scientists plan to systematically study not only
the genes associated with these diseases but also the interactions between the genes in living
model systems.
ICND researchers have already been recognized
by the international scientific community during the first 3 years of the institute’s 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 Research |
| 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 |
| Shelley Halpain, Ph.D. |
Molecular
basis of cognitive function, including learning and memory disabilities and mental retardation |
| 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 |
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