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The Institute for Childhood and Neglected Diseases

The Scripps Research Institute established the Institute for Childhood and Neglected Diseases (ICND) to apply the new molecular understanding of biology to the goal of reducing and treating childhood and neglected diseases. Diseases of both categories often infect populations in developing countries where the health infrastructure may be too limited to support major research efforts.

Examples of such diseases include malaria, epilepsy, mental retardation, cystic fibrosis, chronic pain, and depression. The human and economic costs of these diseases is staggering. Malaria infects 300 million people a year, and the disease kills approximately one million a year, according to the World Health Organization. About 90 percent of these victims are in Africa, where the disease costs some $12 billion annually. The disease is especially devastating to children in Africa, where it is the leading cause of childhood mortality.

The Centers for Disease Control and Prevention (CDC) estimates that epilepsy and its associated seizures affect about 2.3 million Americans, and result in an estimated annual cost of $12.5 billion in medical costs and reduced earnings and production. Mental retardation is another condition affecting children everywhere—about one percent of American children ages 3 to 10 years of age have mental retardation, according to the CDC.

Housed in a state-of-the-art 54,000-square-foot laboratory building on the east side of TSRI’s campus, the ICND is an umbrella group within TSRI for young scientists who are working in areas relevant to the institute's 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 affecting 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 initiative. The Moores went on to contribute a valuable coin collection, as well as pledging an additional $5 million over five years.

Around the same time that these individuals were making donations in support of the ICND, a milestone scientific achievement was nearing completion. The Human Genome Project, which was finished around the same time the ICND opened its doors, has led to a deeper understanding of the mechanisms underlying human disease than ever before. But while the isolated identification of genes has yielded glimpses inside the machinery of the body, it has been difficult to look at genes in the larger context of how they interact with each other and with their surroundings in the cell and the body. Further, the regulatory mechanisms that have been discovered frequently 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 them 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 devise treatments for them. Scientists will undertake a systematic effort to study not only the genes themselves but the interactions between them in living systems. Over the long-term, this approach should lead to medical achievements that are unimaginable with current technologies.