Dust off those old drugs. A Stanford University group has developed a computational method for finding potential new uses for previously approved drugs. And two papers published Wednesday in the journal Science Translational Medicine suggest the potential promise of this approach.
The development comes as the NIH is pushing so-called drug repositioning, or finding new uses for drugs developed for other diseases. The agency, which provided funding for the studies, says repositioning strips away some of the big expenses of development and offers new, affordable ways to treat illnesses. With the support of repositioning advocates, the Stanford group's technology has made progress in finding two repositioned drugs with some signs of efficacy in mouse models of inflammatory bowel disease (IBD) and lung cancer.
The group's computational approach involves algorithms that are applied to such data sources as molecular profiles of diseases and drug information to predict novel uses for meds. In the IBD study, the group let its computational method loose on public gene-expression data and information on 164 small-molecule drugs. The system discovered that the anticonvulsant topiramate might treat IBD, and mouse experiments confirmed that hypothesis. The ulcer drug cimetidine, which the system identified as a potential lung cancer treatment, showed activity against the tumors in mice.
Obviously, there need to be further studies to prove whether the drugs work in humans. Yet what impressed in the Stanford group's papers was the ability quickly analyze large volumes of data to develop hypotheses about new uses for compounds, Rochelle Long, of the National Institute of General Medical Sciences, told the Wall Street Journal.
Smelling a commercial opportunity, upstart NuMedii has formed to take the Stanford technology to the next level. Eric Schadt, a leader in using computational methods in drug research and the chief scientist at Pacific Biosciences ($PACB), is listed on NuMedii's website as a scientific advisor to the start-up.