There is currently no cure for Mitochondrial Disease and so a major theme of the Wellcome Centre is to develop treatments for patients with these conditions. The research theme aims to identify novel drugs that can improve mitochondrial function using our unique collection of patient samples that make up the Mitochondrial Biobank. This exceptional bioresource, which include tissues and cell lines from patients obtained for research or excess to diagnostic procedures, allows our preclinical research to be performed to the highest standard using the most relevant models of Mitochondrial Disease.
Drug development within the Centre is currently done using two different approaches. We are collaborating with a number of pharmaceutical companies to test large numbers of different compounds and identify those that can affect the mitochondria and how they function. We are also working with smaller, specialised companies to test compounds that have already been shown to affect mitochondrial function using cell-based models of Mitochondrial Disease.
By utilising both approaches to drug development, it is hoped that these preclinical studies will lead to potential drug trials for our patients. The Mitochondrial Patient Cohort will allow us to identify eligible patients from all over the UK to take part in such clinical trials in the future. We believe that we are closer than ever to finding effective treatments for Mitochondrial Disease and as such, improving the lives of our patients.