UM Researcher to Play Prominent Role in NIH Effort to Develop New Medicines

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A University of Miami Miller School of Medicine researcher is taking a prominent role in a high-priority National Institutes of Health research consortium designed to identify promising new targets for drug development.

Map of United States showing different research locations
University of Miami Miller School of Medicine researchers will collaborate with other leading institutions in this NIH initiative.

It’s part genomics, part big data, and all about driving discoveries that could someday translate to new treatments for cancer, neurological diseases, addiction, and many other conditions, said Stephan Schürer, Ph.D., Program Director, Drug Discovery, University of Miami Center for Computational Science, and associate professor of molecular and cellular pharmacology at the Miller School.

The NIH’s ‘Illuminating the Druggable Genome’ (IDG) initiative is expected to generate a lot of scientific information from several investigators at leading institutions nationwide. Schürer has been appointed to the IDG’s Resource Dissemination and Outreach Center to organize the incoming data and coordinate all the project's resources.

To increase their odds for success, IDG researchers will be strategic. They are looking at promising but understudied members of protein families already associated with productive drug development: the G-protein coupled receptors, ion channels, and protein kinases. The idea is that proteins closely related to known drug targets will be more promising candidates for future drug development.

“There are already examples of small molecules that bind to members of the same families,” Schürer said. “The Data and Resource Generation Centers are developing scalable technologies to quickly interrogate those protein families and identify new chemicals that can be developed into chemical probes.”

Chemical probes are important tools used to study the function of proteins in cells and animal models. They’re important because pharmaceutical companies also want to be strategic in terms of where they focus their drug development efforts.

“One of the barriers for a pharmaceutical company or a biotech company to develop a drug for a particular target is target validation," Schürer said. “Target validation essentially demonstrates that a target is relevant to a disease … and that the target can be modulated with a small molecule [drug].

“Drug companies basically won’t touch it unless a protein target is firmly established as druggable, meaning it can be engaged with a small molecule and there is a significant pharmacological effect.”

Standardizing data, making sure it is accessible to researchers, organizing the vast available data for these protein families and developing scalable technologies to ramp up the IDG project are further goals. Schürer was part of the Knowledge Management Center for three years during Phase I of the IDG initiative. The second phase is now starting and will run for the next six years.