Cures for new diseases may come from old medicines

Could a bottle of pills in your medicine cabinet aid in the fight against tomorrow's health problems? Scientists think they could.

Using publicly accessible databases, researchers have developed a method of predicting the unexpected benefits of existing drugs, and how they might be repurposed for treating seemingly unrelated diseases.

Just this year, a team of researchers discovered that a commonly prescribed arthritis medication was effective in the treatment of melanoma in animal models. It sounds incredible, but examples of science finding new uses for old drugs are numerous. ScienceNOW's Kai Kupferschmidt reports:

There are plenty of examples of drugs originally developed to treat one disease that turned out to help another: Acetylsalicylic acid (aspirin) is not just a pain killer but is also used to reduce the risk of heart attack. And when a blood pressure drug called sildenafil was discovered to have an unexpected side effect, it went on to become the erectile dysfunction blockbuster now known as Viagra. Such crossovers can save drug developers a lot of time and money. Developing a single new drug on average takes more than a decade and costs about $800 million. Existing drugs have known safety profiles and are approved for human use, so they can be rapidly evaluated for new indications.

Now Atul Butte, a bioinformatics expert from Stanford University, has published two studies in the latest issue of Science that show how combining information on the genetic effect of existing drugs with data on the genetic impact of various diseases provides an efficient method of finding new uses for old medications.

"Our hypothesis was, if a disease is characterized by certain changes in gene expression and if a drug causes the reverse changes, then that drug could have a therapeutic effect on the disease," says Butte.

Sounds pretty straight forward, right? We recently reported on the power of public databases and the wealth of information they contain. To demonstrate the power of his system, Butte used these same databases to search for existing therapeutic candidates for 100 different diseases. He found drug candidates for 53 of the diseases, many of them unexpected, including one that predicted that an epilepsy drug could be used to treat inflammatory bowel syndromes like Crohn's disease.

When they followed up by investigating the therapeutic effects of the epilepsy drug, Butte and his colleagues found it to have a significant effect in the rodent models they tested. But more important than this finding is the support it offers to the use of the team's refined method of using public molecular data in the discovery of new disease therapies.

Science (Article 1, Article 2) Via ScienceNOW

Top image via Rob Byron/Shutterstock