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Approved research

Data-driven identification of a drug's disease-modifying potential outside its main indication area

Principal Investigator: Professor Michael Krauthammer
Approved Research ID: 54717
Approval date: January 15th 2020

Lay summary

The main aim of this study is the analysis of UK Biobank data to identify new uses for existing drugs. This may help patients without sensible drug options to get drug treatment without the need for lengthy and costly drug development. In this manner, a drug may potentially be repurposed for other uses. When drugs are used for treating a specific disease or symptom, patients may experience additional unintended drug effects, referred to as drug side effects, which could be minor, such as a skin rash or more severe, such as gastrointestinal bleeding. In some cases, those effects might be considered beneficial and exploited for treatment. An example is a drug that is unexpectedly observed to cause low blood sugar. Interestingly, in patients suffering from diabetes, such a drug may actually lead to improvement of the disease. To achieve our aim, we will proceed und use UK Biobank data as follows: based on the medications that patients have taken over time, and their medical history, we are trying to better understand additional unexpected consequences of those medications. We will then confirm these consequences by looking deeper at a person's genomic profile, and associated clinical manifestations and blood values. Our final goal ist to generate a list of existing drugs and link them to potentially novel clinical applications. An additional outcome of our study is that physicians might be in a better position to understand the consequences of drug prescriptions and thus make more informative decisions for the treatment and care of their patients. In the following 24 months, we will use UK Biobank data for both establishing and validating the list of potential new drug uses, using the described analytical approaches.