Scientific Report 2007
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 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.
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.
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
is another condition that affects children everywhere; about 1% of American children
3 to 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 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 conditions—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 abnormalities but also the interactions between the genes
in living model systems.
have already been recognized by the international scientific community during the
first 6 years of the institute's existence. In December 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 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
|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 Alzheimer's 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