Sharing With Congress How High-Tech Drug Screening Speeds Research

One reason it takes so long to develop a drug that serves as either a treatment or a cure for a specific disease is that it often takes researchers years, even decades, to screen the innumerable compounds that go into making the drug. Or at least it used to. These days, thanks to recent advances in computer, robotic and data-processing technologies, such tests can take only weeks, as long as researchers have the right resources, which can be very costly and difficult to access.

He’ll also note that, thanks to the National Institutes of Health, accessing both the equipment and compounds needed to carry out such tests is not nearly as difficult as it once was.

“Years ago, because technology and access were limited, this part of the process took much more time,” says Stephen Rose, Ph.D., chief research officer at the Foundation. “Now, once promising compounds are identified—in the blink of an eye, so to speak—they’re tested in animal studies, which help researchers better understand the compounds’ safety, efficacy and clinical potential.”

High-throughput technology is simple in theory, although it can be technically challenging to implement. In a gridlike plate containing divots, or “wells,” a variety of compounds are mixed with biological materials—proteins or cells, for instance—to check for positive or negative reactions. These measurements, or "assays," can be very sophisticated in design. What the latest technology enables researchers to do, via an automated process that involves the incubation and analysis of multiple plates simultaneously, is set in motion the screening of tens of thousands of compounds daily.

And, as Dr. Zack will inform members of Congress, such heavy loads can be handled by the National Institute of Health’s Chemical Genomics Center (NCGC), which works with those researchers whose applications warrant the center’s aid. Dr. Zack is collaborating with NCGC scientists in a public-private partnership aimed at identifying drugs that have the potential to save and restore vision for people affected by devastating retinal diseases, such as retinitis pigmentosa (RP) and age-related macular degeneration (AMD), the leading cause of blindness in Americans 50 and older. In just weeks, Dr. Zack and his NCGC colleagues were able to identify several molecules with vision-saving potential, and they are now working to move them into animal and human studies for further analysis. Without NCGC’s expertise and technology, that same effort would have taken several years.

Speeding up the drug-development process is not only humane; it’s cost-effective. Rare retinal degenerative diseases—including RP, Usher syndrome (combined deafness and blindness), and Stargardt disease (juvenile macular degeneration)—afflict 200,000 Americans. More common retinal diseases, such as AMD, affect more than 10 million Americans. NIH’s National Eye Institute estimates an annual cost of $68 billion in the United States from vision impairment and eye disease.