Researchers at the University of Sheffield lead innovative advance drug testing for Parkinson’s treatment

Researchers at the University of Sheffield, supported by Cure Parkinson’s, are leading a preclinical study to evaluate potential treatments that could slow Parkinson’s disease progression. This research is part of the International Linked Clinical Trials (iLCT) programme, a global initiative led by Cure Parkinson’s in partnership with the Van Andel Institute. Each year, the iLCT committee reviews and prioritizes 15 to 20 drugs with the potential to modify Parkinson’s. Since 2012, they have assessed over 180 drugs, leading to 21 completed clinical trials and 20 ongoing studies involving more than 4,700 people worldwide. To accelerate drug development, Professor Heather Mortiboys from the University of Sheffield is leading a study to test 100 iLCT-evaluated drugs using cellular models of Parkinson’s. The study focuses on three key disease mechanisms: energy production issues, inefficient waste removal, and the buildup of the protein alpha-synuclein. By testing all drugs in the same lab model, researchers can directly compare their effectiveness and explore combination therapies—an approach not yet used in clinical trials for Parkinson’s. Professor Mortiboys has developed a unique method using patient-derived cells. Skin cells from donors are reprogrammed into stem cells and transformed into dopamine-producing neurons, which naturally exhibit Parkinson’s-related abnormalities without artificial modifications. “This study has the potential to make a significant impact by identifying the most promising drug candidates and improving our understanding of their mechanisms,” she said. The findings will help determine which treatments should move to clinical trials and may support multi-arm, multi-stage (MAMS) trials, where multiple drugs are tested simultaneously for continuous progress. Through this research, the University of Sheffield is making headway to advance Parkinson’s treatment.