One Lab’s Trash…

Drug failures yield new hope for neglected diseases.

A new drug’s journey from laboratory discovery to a pharmacy shelf takes about 14 years and costs upwards of $2 billion dollars. If that wasn’t staggering enough, consider this: more than 90 percent of drugs in development fail to make it from bench to bedside.

That means for every FDA-approved drug, pharmaceutical companies probably have 10 more—thousands altogether—collecting dust. Most are shelved because they’re ineffective at treating the intended disease; so while proven safe in humans, the best use has yet to been found. That’s why UC San Diego researchers are now taking a second (or third) look at many of these drugs and repurposing them to treat new diseases.

Web - Drug Repurposing
UC San Diego researchers are breathing new life into old drugs.

“Drug repurposing is a faster, cheaper and safer approach to finding new treatments for disease,” says James McKerrow, M.D., Ph.D. ’73, dean of the Skaggs School of Pharmacy and Pharmaceutical Sciences at UC San Diego. With the groundwork already laid, new therapies based on repurposed drugs could benefit patients much sooner than the typical 14 years—saving and improving more lives, and offering an efficient way to combat neglected diseases.

McKerrow recently received a National Institutes of Health award granting access to SAR114137, a drug the pharmaceutical company Sanofi initially developed to treat chronic pain. McKerrow’s team will now test the drug’s efficacy in treating Chagas disease, a neglected tropical disease and the leading cause of heart failure in Latin America.

McKerrow has a hunch that SAR114137 could work for Chagas disease because of its similarity to a previous drug that successfully treated infected animals, but couldn’t be validated in humans. Since SAR114137 has passed safety studies in humans, it’s already many steps ahead of its predecessors.

“Chagas affects more than 8 million people and is responsible for more than 10,000 deaths worldwide each year. Yet there are currently no FDA-approved therapies,” McKerrow says. “We call Chagas a ‘neglected’ disease because most pharmaceutical companies aren’t interested in developing new therapies for a disease that mainly affects poor communities. This is one area that’s especially likely to benefit from drug repurposing.”