Drug Discovery for Mitochondrial Disease

In a recent WCMR Science Seminar, we heard from Dr Iffath Ghouri who talked to us about her research that aims to identify compounds that could improve mitochondrial function as part of the drug development pathway for mitochondrial disease. Read on to find out more.

There is currently no cure for mitochondrial disease. Available treatments involve alleviating the symptoms caused by dysfunctional mitochondria and supportive therapy to improve patient quality of life. Together with other members of the Treatment Team based at the WCMR, we aim to identify compounds that have the potential for development to treat mitochondrial disease by improving mitochondrial function.

We use a high-throughput screening assay that allows us to check the effect of thousands of drugs (available through compound libraries) on mitochondria. In this way, we are able to narrow down thousands of compounds to the most promising few. These so called “hit” compounds undergo further analysis through various different mitochondrial assays and using a range of concentrations. The compounds that perform best on these assays can be refined and optimised further by modifying the structure of the drug to hopefully make them more effective.

Using this process, we have identified lead compounds which we hope have the potential for further therapeutic development. We are working with collaborators to produce derivatives of these molecules, with the aim of improving the drugs’ effects on rescuing mitochondrial function. If we identify one or more molecule which performs well on the assays and does not have toxic effects at therapeutic concentrations, we hope to proceed to the next stage of drug development.

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