Scientists turn drug-disease match makers

By Dr. Ananya Mandal, MDAug 18 2011

In a new medical breakthrough American researchers have devised a drug-disease matchmaking program that mines databases for potentially useful new treatment combinations and have actually turned up two so far, said a study on Wednesday.

They found that a a drug commonly used to alleviate ulcers has shown promise against lung cancer and an epilepsy medicine for halting seizures could also work against inflammatory bowel disease. The research, led by Stanford University scientists and funded by the National Institutes of Health, is published in the journal Science Translational Medicine.

Rochelle Long, director of the NIH Pharmacogenomics Research Network said, “This work is still at an early stage, but it is a promising proof of principle for a creative, fast and affordable approach to discovering new uses for drugs we already have in our therapeutic arsenal.” The technique could also save money, since it costs about one billion dollars to develop a new drug these days, Long added.

The researchers used an NIH database that is available to the public, called the NIH National Center for Biotechnology Information Gene Expression Omnibus, which contains thousands of genomic studies from around the world. The database compiles information on how gene activity changes during the course of disease and in response to drug treatment.

Narrowing down their focus to a range of 100 diseases and 164 drugs, the researchers created a program that scoured the database for “drugs and diseases whose gene expression patterns essentially cancelled each other out,” said the study. “For example, if a disease increased the activity of certain genes, the program tried to match it with one or more drugs that decreased the activity of those genes.”

They found that an anti-ulcer treatment known as cimetidine was a good match for lung cancer, and a seizure drug topiramate would work against inflammatory bowel disease. They tested cimetidine on human lung cancer cells - some in a lab and others that were transplanted into mice. Both approaches slowed the cancer cells' growth. The anti-convulsant drug was administered to rats with bowel disease and was shown to reduce the associated symptoms of diarrhea, ulcers and colon damage. This is not the first time scientists have found that a drug has other uses.

It takes an average 15 years and about $1 billion to bring a single new drug to the market, researchers said in one of the reports. So-called repurposing of old drugs can cut that cost because drug makers wouldn’t have to do the toxicity tests required in early development. The study authors said they hope their findings will help line up funding sources, allowing them to use this new approach to match certain compounds with the right clinical trials.