NIH has announced a new collaborative initiative to improve human health by exploring poorly understood genes that have the potential to be modified by medicines. The effort is part of an NIH Common Fund three-year pilot project called Illuminating the Druggable Genome (IDG). For the initial phase of the program, NIH has allocated $5.8 million to eight institutions and for intramural resources.

As many as 3,000 genes express proteins that could have their activities altered by medicines, according to predictions based on genomic information. Yet only about 10% of these druggable genes are targeted by FDA-approved drugs. The IDG program is designed to address this gap by supporting research of understudied genes in four important druggable gene families: nuclear receptors, ion channels, protein kinases and G-protein coupled receptors, often called GPCRs.

Initially researchers will delve into these uncharacterized genes and share what they learn on a public resource that will enable the larger scientific community to build on the findings with both basic research and clinical translation. They also will work to develop ways to rapidly identify and describe the genes they explore, creating a common language that can be applied across experimental systems, from individual cells to complex biological models.

The pilot awards include a grant to establish a Knowledge Management Center, led by the University of New Mexico in Albuquerque in collaboration with Icahn School of Medicine at Mt. Sinai in New York, with support from other institutions. Seven additional grants will support development of technology to understand functions of members of the four protein families.

The institutions receiving these grants are:

• University of North Carolina, Chapel Hill (two grants)
• Massachusetts General Hospital, Boston
• University of California, San Francisco
• Yale University, New Haven, Conn.
• J. David Gladstone Institutes, San Francisco, Calif.
• Baylor College of Medicine, Houston

“We have a gap in the drug development pipeline between what gene activities we know could be modified by medication and what currently is targeted. By focusing on understudied genes, we hope to find potential targets for medications to treat or cure some of our most burdensome diseases—and then share what we learn so that all can build on this knowledge,” said James M. Anderson, M.D., Ph.D., director of the division of program coordination, planning and strategic initiatives, which oversees the NIH Common Fund.

The awards are primarily funded through the Common Fund, which supports high-impact pioneering research across the NIH. Other support comes from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), the National Center for Advancing Translational Sciences (NCATS), the National Institute of Mental Health, the National Cancer Institute and the National Institute of Neurological Disorders and Stroke.