Results from the Human Genome Project revealed that the human genome contains 20,000 to 25,000 genes. A gene contains (encodes) the information that each cell uses to make (express) a protein, which is essential for the body to function properly. Abnormal protein expression is associated with many human diseases, which makes proteins key targets for therapeutic agents.
Approximately 3,000 genes are considered part of the “druggable genome,” a set of genes encoding proteins that scientists can or predict they can modulate using experimental small molecule compounds. Yet only about 10 percent of these genes encode proteins that have been targeted successfully by an approved drug. Therefore, a large number of proteins remain for scientists to explore as potential therapeutic targets. The vast majority of the druggable genome encodes four key protein families: G-protein-coupled receptors, nuclear receptors, ion channels and kinases. Researchers lack crucial knowledge about the function of many proteins from these families and their roles in health and disease. Better understanding of how these proteins work could shed light on new avenues of investigation for basic science and therapeutic discovery.
A New Area of Discovery
To improve scientific understanding of the four understudied protein families, NIH launched an effort called Illuminating the Druggable Genome (IDG) in 2013, with the first awards made in 2014. IDG is a three-year pilot program sponsored by the NIH Common Fund and designed to test a two-pronged approach for exploring the druggable genome. One objective involves the creation of a centralized information repository, and the other focuses on developing new technology platforms to study the protein families.
NCATS Director Christopher P. Austin, M.D., co-chairs NIH’s IDG Working Group with Griffin P. Rodgers, M.D., M.A.C.P., director of the National Institute of Diabetes and Digestive and Kidney Diseases. The group includes representatives from the National Institute of Mental Health, the National Cancer Institute, the National Institute of Neurological Disorders and Stroke, and other Institutes and offices at NIH.
By expanding the potential therapeutic space through the IDG program, NIH is clearing a path for more efficient disease-related research and more effective treatments for patients.
For more information about IDG, visit the NIH Common Fund website.