Genome-Based Repositioning: Bioinformatics Delivers

The field of bioinformatics, generally described as the use of computer-based data processing applied to problems in the life/medical sciences has now delivered a promising set of results: expression profiles (a biochemical fingerprint of what is happening in the body) were compared between known diseases and known drugs in order to identify "cousins" - where both drug and disease have similar effects on the body. This was a random matching pool, looking to identify drugs which might have previously undiscovered relationship - i.e., potential use - for other medical conditions. In this report, scientists at Stanford reported some previously unrecognized connections between, for example, topiramate, an anticonvulsant drug, now shown to have a parallel biochemical signature with inflammatory bowel disease (IBD), suggesting a potential new application for the drug. Original papers here and here. The repackaging of old drugs for new uses is also referred to as repositioning (or repurposing). Legal issues? New uses for old drugs are potentially patentable, but the drugs themselves are likely to be either coming off patent or already in the public domain, so the focus turns to method patent claims, which recite a way of using a drug in a process (e.g., therapeutic application). From a regulatory perspective, the drugs may have already passed through the FDA-required clinical approval process, so safety profiles are established. But efficacy would still need to be studied (do they work?). From the view of R&D, here's an approach to drug discovery that doesn't require finding new chemicals, but simply (sort of) enlarging the profile of drugs that are already known.