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Study: New approach helps ID possible novel uses for existing drugs

​​​​​April 8, 2015​

Genetics research highlights thousands of possible drug-disease connections

Scott Hebbring, Ph.D.Finding new uses for existing drugs has become increasingly important for treating diseases and a Marshfield Clinic-led research team has proven a new way to speed up the process, according to a study published today in Nature Biotechnology.

Drug repurposing is the process of discovering new uses for existing drugs. The process is becoming increasingly important in drug development as success rates for new drugs in clinical trials decrease and costs increase. Critical to drug repurposing is initially identifying candidate drug-disease relationships.

Using a type of genetic study called a phenome-wide association study, researchers identified thousands of potential disease and drug relationships. This could serve as an early clue when looking for drugs that may effectively treat diseases they’re not currently prescribed for.

“This is a proof of principle study demonstrating phenome-wide association data may be rapidly applied to developing or repurposing existing drugs,” said Scott Hebbring, Ph.D., principal investigator and research scientist, Marshfield Clinic Research Institute​​’s Center for Human Genetics. “Unlike other genetic-based approaches, a phenome-wide association study allows us to look at thousands of diseases at once, rather than just one disease at a time.”

A team of researchers, using the phenome-wide approach, identified more than 14,800 examples of where a drug might be used to treat an unrelated disease. They did this by cross-referencing drug-disease pairs identified by phenome-wide data with existing medical literature to find examples where a disease and drug were mentioned in the same article.

“While we didn’t provide context for these relationships, we hope these novel findings offer researchers clues that allow them to prioritize which potential relationships to study first,” Hebbring said. “That, in turn, could help us more quickly find new uses for current drugs.”

They also found more than 38,000 novel drug-disease relationships not yet studied, according to the medical literature. These results suggest phenome-wide association study data may bring to light many additional diseases that may be treated with already developed drugs.

To read the full study, go to Nature Biotechnology.

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Marshfield Clinic Research Institute (MCRI), a division of Marshfield Clinic, was founded in 1959. It’s the largest private medical research institute in Wisconsin. MCRI consists of research centers in clinical research, agricultural health and safety, epidemiology, human genetics and biomedical informatics. Marshfield Clinic investigators publish extensively in peer-reviewed medical and scientific journals addressing a wide range of diseases and other health issues including cancer, heart disease, diabetes, eye disease, infectious disease, neurological disease, pediatrics, radiology, vaccine effectiveness, women's health, agricultural safety and genetics.

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