Using Model Systems to Find Drugs to Repurpose for Rare Diseases

The use of model system, such as fruit flies and worms, to screen existing drugs for their potential to treat rare genetic diseases offers a relatively fast and economic method to find candidates for repurposing. The success at screening 4,000 compounds in a worm model of the neurodegenerative disease ALS to identify a candidate that is now in human clinical testing gave rise to Modelis, a Canadian company that is now repeating the exercise in other rare diseases. We spoke to James Doyle, CEO of Modelis, about model system, how the company creates genetic avatars of patients; and how it works with rare disease drug developers, patient organizations, and patients to identify candidates for repurposing.