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kaybw The Institute for Childhood and Neglected Diseases

Steve Kay, Ph.D.

The Institute for Childhood and Neglected Diseases (ICND) was established to apply the new molecular understanding of biology to reduce the prevalence of childhood and neglected diseases and to treat the patients who have them. 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 depression. 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 people, and the disease kills approximately 1 million people. 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 is epilepsy. According to estimates from the Centers for Disease Control and Prevention, epilepsy and its associated seizures affect about 2.3 million Americans and account for $12.5 billion in medical costs and reduced earnings and production each year.

Mental retardation is another condition that affects children everywhere; about 1% of American children 3 to 10 years old have mental retardation, according to the Centers for Disease Control and Prevention. 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 brand-new, state-of-the-art, 54,000-square-foot building on the east side of the TSRI campus, the ICND is an umbrella group within TSRI for young scientists who are working in areas relevant to the ICND focus on these sometimes widespread and often devastating diseases. 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. However, although the isolated identification of genes has yielded glimpses inside the machinery of the body, looking at genes in the larger context of how they interact with each other and with their surroundings in the cell and the body is difficult. Furthermore, the regulatory mechanisms that have been discovered often turn out to be small parts of larger, more complex cascades.

Investigators at the ICND use genomics and advanced imaging technologies, develop novel model animal systems, and apply the technologies and systems 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 themselves that are associated with these diseases but also the interactions between the genes in living systems. In the long-term, this approach should lead to medical achievements that are unimaginable with current technologies.



Copyright © 2004 TSRI.