Virtual repurposing can help discover new uses for existing drugs

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Virtual repurposing can help discover new uses for existing drugs

Virtual repurposing uses big data and genomics to discover connections between seemingly “unconnected” diseases that may lead to new treatments.

he process for discovering drugs tends to be based on the same kind of pigeonholing that doctors use to treat disease: Parkinson’s is one disease, Crohn’s is something completely separate. A new approach, sometimes called virtual repurposing, offers a way to discover unknown connections between “unconnected” diseases that may lead to new treatments.

Drug discovery usually begins with a promising molecule. It is then tested in animal models and, if there are signs of effectiveness, in human clinical trials. At every step along the way, the data emerging from this work must be analyzed. It’s a slow process that can take a decade or more and cost, on average, more than $2.6 billion.

But recent advances in data analysis are making it possible to turn the process on its head. Virtual repurposing begins with existing data. Using new capabilities in biomedical informatics and machine learning, researchers comb through massive quantities of data in search of common genetic links between diseases that, based on symptoms, would seem to have nothing in common. If a shared genetic pathway is discovered, then it may be possible to treat one disease with medicines approved for the other.

My colleagues and I used this technique to uncover a possible link between Parkinson’s disease and the inflammatory bowel diseases, Crohn’s disease and ulcerative colitis.

We started by searching for common genetic mutations in a population at high risk for Crohn’s disease: Jews of Eastern European origin, known as Ashkenazi Jews. One gene we found, called leucine-rich repeat kinase 2 (LRRK2), was highly associated with Crohn’s disease, increasing the risk for the disease by 70 percent. That came as a surprise, because LRRK2 has been linked to Parkinson’s disease, a neurodegenerative disorder that appears to have little in common with IBD.

We confirmed the genetic link by finding a connection between the LRRK2 mutation and Crohn’s disease in patients with Parkinson’s disease in both Ashkenazi and non-Ashkenazi populations.

Read on: Virtual repurposing can help discover new uses for existing drugs

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