Congressional Briefing Highlights NIH’s Drug Discovery Technology

Featured speaker Donald Zack, M.D., Ph.D. (Wilmer Eye Institute, Johns Hopkins University)

Finding a vision-saving drug can take years, even decades, because scientists need to screen tens or hundreds of thousands of compounds before they find one that might work. But thanks to advanced screening technology and a research collaboration involving the Foundation Fighting Blindness (FFB), the National Institutes of Health’s (NIH) Chemical Genomics Center (NCGC), and Johns Hopkins University (JHU), the search for drugs to combat retinal diseases has been accelerated. The NCGC is a component of the Molecular Libraries Program destined for inclusion into the NIH’s proposed National Center for Advancing Translational Sciences (NCATS).

At a June 9 Capitol Hill briefing hosted by the Alliance for Eye and Vision Research’s (AEVR) Decade of Vision 2010-2020 Initiative and FFB, Donald Zack, M.D., Ph.D., a clinician-scientist at the Wilmer Eye Institute at JHU, spoke to 70 Congressional staffers about how the NCGC’s high-throughput drug screening systems have greatly accelerated his search for retinal disease treatments. In a research project funded in part by FFB, Dr. Zack and his NCGC colleagues were able to identify several molecules with vision-saving potential in a period of weeks. Without NCGC’s expertise and technology, that same effort would have taken several years.

"Years ago, because technology and access were limited, this part of the process took exponentially more time," says FFB Chief Research Officer Stephen Rose, Ph.D., who hosted the event with AEVR Executive Director James Jorkasky. "Now, new screening technology enables us to more quickly identify promising compounds and move them into animal studies, which help researchers better understand the compounds’ safety, efficacy, and clinical potential."

Dr. Rose added that the availability of advanced high-throughput screening systems is limited, because of their expense. "Most research institutions can’t afford screening systems that are as powerful and sophisticated as NCGC’s. To have such a resource available to FFB-funded researchers greatly accelerates our drive for vision-saving treatments," says Dr. Rose.

High-throughput systems combine robotics and computers to quickly screen large numbers of compounds. In grid-like plates containing divots, or wells, a variety of compounds are mixed with biological materials — proteins or cells, for instance — to determine if they promote cell health and function. What the latest technology enables researchers to do, via an automated process that involves the incubation and analysis of multiple plates simultaneously, is screen tens of thousands of compounds daily.

As Dr. Zack stressed, speeding up the drug-development process not only increases the quality of life of our citizens, it is also cost-effective. Rare retinal degenerative diseases — including retinitis pigmentosa (RP), Usher syndrome (combined deafness and blindness), and Stargardt disease (juvenile macular degeneration) — affect 200,000 Americans. More common retinal diseases, such as age-related macular degeneration (AMD), affect more than 10 million Americans. Dr. Zack’s research has implications for understanding the pathogenesis of AMD, RP, and glaucoma (the second leading cause of preventable blindness), enabling him to develop new paradigms for the diagnosis, prevention, and treatment of these blinding eye conditions. This is important, as the National Eye Institute (NEI) estimates that vision impairment and eye disease cost the United States economy more than $68 billion a year.

Left to right: Dr. Zack with briefing hosts Foundation Fighting Blindness (FFB) Chief Research Officer Stephen Rose, Ph.D. (center) and AEVR Executive Director James Jorkasky	Dr. Zack speaks with Association for Research in Vision and Ophthalmology (ARVO) scientist Bobbie Ann Austin, Ph.D.
Left to right: Dr. Rose, FFB’s Ben Shaberman, Dr. Zack, and National Eye Institute (NEI) Senior Advisor for Translational Research Matthew McMahon, Ph.D.