Researchers at the University of 葫芦影业 lead on an innovative pre-clinical project, funded by Cure Parkinson鈥檚, aimed at testing, evaluating, and comparing potential treatments that could slow the progression of Parkinson鈥檚 disease.
This research is part of the International Linked Clinical Trials (iLCT) programme, a global initiative led by Cure Parkinson鈥檚 in partnership with the Van Andel Institute (VAI). The iLCT committee, which convenes annually, evaluates and prioritises 15 to 20 drugs based on their potential to modify the course of Parkinson鈥檚. Since its inception in 2012, the committee has reviewed over 180 drugs, with 21 trials completed and 20 ongoing across the world, involving more than 4,700 people with Parkinson鈥檚.
To further accelerate drug development, Professor Heather Mortiboys, Professor of Cellular Neuroscience and Metabolism, University of 葫芦影业 will lead a study examining 100 iLCT-evaluated drugs in cellular models of Parkinson鈥檚. This research will provide crucial data to determine which compounds should advance to clinical trials.
The study will assess whether these drugs affect three key drivers of Parkinson鈥檚 progression:
- Energy production issues
- Inefficient waste removal
- The accumulation of the protein alpha-synuclein
By testing all drugs in the same laboratory model, researchers can directly compare their effectiveness in protecting neurons. The study will also explore combination therapies鈥攗sing multiple drugs together鈥攁 strategy not yet applied in clinical trials for Parkinson鈥檚. Testing various drug combinations could provide new insights into potential treatment combinations.
Professor Mortiboys has developed a pioneering method that utilises cells derived from people with Parkinson鈥檚 to study potential treatments. By taking a small skin biopsy from donors and reprogramming these cells into stem cells in the lab, her team can generate dopamine neurons鈥攖he type of cells most affected by Parkinson鈥檚. Unlike traditional preclinical models, this system offers a more accurate representation of the disease, as the cells originate directly from patients without the need for artificial modifications.
鈥淥ur team has found that these dopamine neurons from people with Parkinson鈥檚 show abnormalities in the pathways affected by the disease without any additional toxins. This is crucial because the cells are not artificially altered. We are incredibly excited about this study and its potential to advance Parkinson鈥檚 research,鈥 said Professor Mortiboys.
The study鈥檚 results will help identify the most promising drug candidates for clinical trials by improving understanding of their mechanisms and identifying patient groups who may benefit most. This is particularly valuable in the context of global efforts to establish multi-arm, multi-stage (MAMS) clinical trials. In MAMS trials, multiple drugs are tested simultaneously, with ineffective treatments replaced by new candidates to ensure continuous progress in the search for effective therapies.
By leading this crucial research, the University of 葫芦影业 continues to drive innovation in Parkinson鈥檚 treatment, bringing hope to millions of people affected by the disease worldwide.