Dr Sara Mole, who has a Sparks-funded project, has recently begun a large programme of work aimed at developing new therapies for Batten disease. Dr Mole led a consortium of researchers from 8 different countries to secure a €6M grant from the prestigious EU Horizon 2020 innovation grant programme to allow them to carry out this work.
Batten disease is a group of rare, fatal neuro-degenerative conditions, which affects cells in the brain gradually, causing a loss of physical and mental abilities. It is thought that around 150 individuals are living with a form of Batten disease in the UK. Dr Mole’s project – BATCure – will be focussed on three specific types of Batten disease called CLN3, CLN6 and CLN7 disease. She and the other consortium members will be working to develop gene therapies as well as look for small molecule drugs that could slow or halt progression of the disease. It is hoped that information gained during the project will also advance understanding of what goes wrong in the cells of those affected by these types of Batten disease. The consortium will also be working to ensure that the Batten disease community is ready for clinical trials as soon as any potential drugs are ready to be tested and Dr Mole and her colleagues are very much hoping to have at least one candidate drug to take forward at the end of the 3 year BATCure grant.
Dr Mole’s current Sparks grant is aimed at investigating potential new therapies for CLN3 and CLN10. She is using a yeast-based drug screen to test large numbers of small molecule drugs quickly, for their potential to prevent progression of Batten disease. Any promising potential drugs for CLN3 disease from the Sparks project will be further investigated as part of the BATCure grant allowing them to progress to clinical trial if they show promise.
This work has the potential to have a huge impact on this rare disease for which there are currently no cures and limited treatment options. We will be following the progress of the consortium with interest and look forward to seeing the benefits for families affected by Batten disease.