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NIH Launches New Program to Find Potential Drug Targets

Researchers will explore poorly understood genes

The National Institutes of Health (NIH) has announced a new collaborative initiative to improve human health by exploring poorly understood genes that have the potential to be modified by medications. The effort is part of an NIH Common Fund 3-year pilot project called Illuminating the Druggable Genome (IDG). For the initial phase of the program, the NIH has awarded $5.8 million to eight institutions.

As many as 3,000 genes express proteins that could have their activities altered by medicines, according to predictions based on genomic information, and yet only about 10% of these so-called “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.

Initially, researchers will investigate these uncharacterized genes and share what they learn on a public resource that will allow the larger scientific community to build on the findings with both basic research and clinical translation. The researchers will also 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.

“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,” said James M. Anderson, MD, PhD, director of the Division of Program Coordination, Planning, and Strategic Initiatives, which oversees the NIH Common Fund. “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.”

Sources: NIH; July 31, 2014; and IDG; December 16, 2013.

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