New Institute Within TSRI Focuses on Molecular Genetics of Disease

Researchers Target Illnesses of Children and Afflictions of People in Developing Nations

By Mika Ono

With the opening of a brand-new, 54,000-square-foot building on the east side of The Scripps Research Institute (TSRI) campus last month, the idea of the Institute for Childhood and Neglected Diseases (ICND) became a reality. The ICND will act as an umbrella group within TSRI for young scientists working in areas relevant to childhood diseases (such as mental disorders and cystic fibrosis) and diseases (such as malaria and schistosomiasis) that primarily afflict people in developing countries.

“The ICND represents a whole new effort for TSRI in the molecular genetics of disease,” says Cell Biology Professor Steve Kay, PhD, who has been organizing the initiative. "The ICND will focus on combining genetic analysis with the new tools of genomics under development by private industry. The potential for synergy is phenomenal."

Six faculty, four new to TSRI, will open the ICND:

•Assistant Professor Colin Fletcher (AB, Dartmouth College; PhD, The Rockefeller University), who investigates the genetics of the locomotor disorders—work relevant to childhood epilepsy and familial forms of migraines and ataxia. Fletcher arrives at TSRI from the National Cancer Institute's Frederick Cancer Research and Development Center.
• Associate Professor Mark Mayford (BS, PhD, University of Wisconsin, Madison), whose work focuses on the role of single genes and specific cells in the formation of memories—work relevant to mental retardation and learning disabilities. Mayford most recently headed a lab at the University of California, San Diego.
•Assistant Professor Ardem Patapoutian (BA, University of California, Los Angeles, PhD, California Institute of Technology), who works on pain and pain perception—a field relevant to most diseases. Patapoutian arrives at TSRI after a postdoctoral fellowship at the University of California, San Francisco.
• Assistant Professor Elizabeth Winzeler (BA, Lewis and Clark College; MS, Oregon State University; PhD, Stanford University), who is finding ways to select promising proteins to target in the malaria parasite. Winzeler comes to TSRI after a postdoctoral fellowship at Stanford University.
• Professor William Balch (BSc, Portland State University; PhD, University of Illinois), who works transport through the secretory pathway with disease models of cystic fibrosis and hereditary emphysema. Balch, who has been at TSRI since 1990, will continue to run his laboratory outside the ICND building.
•Professor Steve Kay (BSc, PhD, University of Bristol, United Kingdom), who studies circadian rhythms—a field relevant to sleep disorders, depression, and bipolar disorder. Kay has been at TSRI since 1996.

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 $5 million over five years. The Chase brothers also made a donation to the effort.

In 1999, Lerner, Kay, and Professor Peter Schultz met to begin detailing the scientific aspects of the ICND, which was conceived in three phases:
• Phase I, now essentially complete, puts in place investigators who explore the physiologic functions of genes;
• Phase II, currently underway, focuses on recruiting scientists with experience working with direct models of disease;
• Phase III, which is to launch in a year or more, brings in genomics-based human geneticists interested in working on both basic and clinical research.

Throughout all phases, collaborations with the The Genomics Institute of the Novartis Research Foundation (GNF) and other private institutions will be set up as needed.

"The ICND is a far-reaching project that is immensely appropriate for an institution of TSRI’s stature," comments Kay. "With the ICND, TSRI is making a significant commitment to translating the letters of the Human Genome Project into prose that will benefit mankind."

Assistant Professor Elizabeth Winzeler, whose research focuses on finding ways to select promising proteins to target in the malaria parasite, is one of six faculty to open the ICND.