What companies do have is an enormous backlog of stalled drug compounds, or "dead drugs," as they are sometimes called. Nine of 10 drugs that enter human trials are "discontinued," as the industry delicately puts it, sometimes for safety reasons, but often after they fail to reach just one or two crucial clinical endpoints. Uncounted thousands of such compounds have been shelved at pharmaceutical companies large and small. Most never return to the clinic.
Companies that are willing to talk about these drugs at all say they would love to repurpose them—to look for new clinical targets, or even to chemically modify compounds in order to reduce safety hazards or increase efficacy. But, to judge by company practices, most drug developers see shelving inactive drug candidates as a solid business decision. Ignoring the once promising compounds frees up R&D resources for drugs that are still in the pipeline.
"We let the compound tell us what it is," says Mark Gessler, Gene Logic's CEO. "If the compound is active, we can find out what it does." Using genomics and other technologies, scientists at Gene Logic have developed a battery of tests to check the activity of compounds against bio-markers and gene targets for more than 400 diseases. No molecule is chemically altered to increase its chances of hitting a therapeutic target—whether the clinical trial's original one or a new one. No drug is re-engineered. Instead, the company seeks to discover, with systematic biological screens and assays, how—if at all—a drug interacts with a broad range of biological systems. At Gene Logic, this is known as "interrogating" a compound. Literally asking it how it reacts biologically.
The idea is to do systematically in the laboratory for many compounds what happens serendipitously during clinical trials and practice for a few. Viagra (sildenafil), Botox (botulinum), and even thalidomide, for instance, have become successful drugs when they proved effective for indications their creators did not anticipate. Why shouldn't unanticipated applications rescue compounds that never made it out of the clinic?