A New Commitment to Personalized Medicine

The Rockefeller University

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In 2001, mapping the human genome was a $100 million project—and an astonishingly successful one. Today, an individual's genome can be sequenced for as little as $3,500. The availability of rapid, inexpensive sequencing technology is already leading to unforeseen findings in basic biology and unprecedented breakthroughs in clinical research. In order to realize this technology's full potential, The Rockefeller University is preparing to establish a new Center for Genomic Medicine.

The current genomics revolution was seeded at Rockefeller in 1944, when medical scientists Oswald Avery, Colin MacLeod, and Maclyn McCarty discovered that DNA is the chemical that transmits hereditary information. Just 70 years later, we are close to the day when information gleaned from a patient's own DNA sequence will be integrated into the planning of his or her medical care. Clearly, the dawn of the era of personalized medicine has arrived.

The new Center for Genomic Medicine will accelerate the pace of discovery in biomedicine by making the techniques and approaches of today's genetic research available to Rockefeller faculty members representing a broad range of disciplines in the life sciences. Investigators heading basic science laboratories are interested in expanding their current research programs by adding capabilities in human genetics. For these scientists and many others in the university's 73 laboratories, the new Center will be the vehicle that helps them take their most significant discoveries into the realm of human biology and, eventually, enables them to see their findings applied in the clinic.

In establishing the Center, the University will build on its considerable strengths in human genetics. The current faculty includes: Jeffrey Friedman, recipient of an Albert Lasker Award, who is noted for his discovery of the weight-regulating hormone leptin; Jean-Laurent Casanova, who conducts paradigm-changing research on the role of heredity in infectious disease; and Agata Smogorzewska, who studies the genes involved in DNA repair and how failures in these mechanisms contribute to cancer. Rockefeller is now in the process of recruiting additional world-class human geneticists to join the Center.

"The Center will provide unprecedented opportunities to establish the clinical significance of DNA variants."

For Friedman, who identified leptin in the mid-1990s through an arduous gene-mapping approach that he now describes as "mind-numbing," today's speedy and powerful sequencing technologies offer a world of exciting possibilities. He says that the new Center will provide "unprecedented opportunities to establish the clinical significance of DNA variants—genetic differences large and small that will be found within and across human populations. We'll develop an understanding of the variants that cause disease and those that underlie the normal characteristics that make every human being unique."

The ability to decipher all 3 billion units of a person's DNA is just one element of a research program that strives to discover new genetic diseases and identify unknown, clinically relevant subtypes within categories of illness that are now understood only in the broadest sense. The Rockefeller University Hospital, which has pioneered advanced approaches to clinical investigation since its opening in 1910, can play a critical role in achieving these goals. The hospital has unique assets for phenotyping, the rigorous clinical characterization of individuals and families affected by disease. The quality and organization of collected patient information will play a key role in any analytic method designed to extract clinically relevant insights from DNA sequence data.

The Rockefeller University will also take full advantage of its close association with the New York Genome Center (NYGC), which was created two years ago by a dozen area institutions on a collaborative model that allows members to draw on resources and expertise for experimental design, sequencing, and genomic bioinformatics—the development and application of computational tools to interpret sequence and clinical data.

Robert Darnell, a professor at Rockefeller and the founding president of the New York Genome Center, notes that genetic studies undertaken by Rockefeller scientists and other NYGC members will "tap into the ethnic diversity of New York." This not only means better science; such a strategy also represents an "egalitarian approach that is crucial to the future of medicine."

Rockefeller President Marc Tessier-Lavigne adds that genomics will also serve to "accelerate drug discovery by helping pharmaceutical developers identify the patients who stand to benefit most from new therapies."

The Center for Genomic Medicine at Rockefeller will be a productive participant in a city-wide initiative that is bringing the best minds in academic bioscience to New York. The Center will also build and support partnerships between academia and the pharmaceutical/biotech sector, further assisting in efforts to enhance New York's standing as a science capital. As DNA sequencing unlocks the secrets of both health and disease, The Rockefeller University—a leading light in New York for well over a century—will continue to make essential contributions in this golden age of biomedical research.

Photo: Jeffrey M. Friedman (right), committee chair for The Center for Genomic Medicine, and a colleague.