News and Publications

The Institute for Childhood and Neglected Diseases

Steve Kay, Ph.D.

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 TSRI campus, the ICND is a focus group within TSRI 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 TSRI 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 35,000­45,000 human genes. This formidable challenge requires complex multidisciplinary approaches that 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 the 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 systems.

ICND researchers have already been recognized by the international scientific community during the first 2 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.	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 Mueller, 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