New Uses for Existing Medicines
In a novel approach, researchers used computers and genomic data to find new applications for existing FDA-approved drugs. The accomplishment represents a major step forward in drug discovery.
The research team created a computer program to compare the expression profiles of about 164 drugs and 100 diseases. The program searched through the thousands of possible drug-disease combinations to find drugs and diseases whose gene expression patterns essentially cancelled each other out. The work was supported by NIH’s National Institute of General Medical Sciences (NIGMS), National Cancer Institute (NCI) and National Library of Medicine (NLM). The results appeared in 2 articles on August 17, 2011, in Science Translational Medicine.
The approach pointed to potential drug-disease relationships for 53 of the 100 diseases examined. The program associated each of the 164 drugs with at least one disease. It predicted drug-disease pairs that are already in the market, validating the approach. For example, it matched prednisolone, a well known corticosteroid, with Crohn’s disease, for which it is already a standard therapy.
The researchers selected 2 candidate drug-disease pairs for further testing. They found that cimetidine, which is prescribed for heartburn, successfully inhibited the growth of human lung tumors both in the laboratory and when implanted in mouse. They also found that topiramate, an epilepsy drug, effectively decreased symptoms in rodents with inflammatory bowel disease.
"This work is still in an early stage, but it is promising proofs of principle for a creative, fast and affordable approach to discovering new uses for drugs we already have in our therapeutic arsenal," says Dr. Rochelle M. Long, Director of the NIH Pharmacogenomics Research Network.