Stanford scientists find promising Parkinson's treatment through enzyme inhibition

In a groundbreaking study at Stanford Medicine, researchers investigated the role of the LRRK2 enzyme in Parkinson's disease, revealing a novel treatment approach. The research was published in the journal Science Signaling and involved mice genetically modified to have overactive LRRK2 enzymes, mimicking symptoms of early Parkinson's disease. The lead author, Suzanne Pfeffer, PhD, and her team utilized the MLi-2 LRRK2 kinase inhibitor on these mice for a duration of three months. Initial observations showed no change in the brain structure or function of dopamine neurons. However, after the full treatment period, significant restoration of neuron structure and communication was observed in the treated mice, aligning them closer to normal neurological function. These promising findings suggest that targeting the overactive LRRK2 could be a viable strategy in stabilizing or potentially reversing symptoms of Parkinson's disease. Researchers stress the importance of early intervention, as symptoms frequently emerge long after the onset of the disease. Identifying patients who are at risk or at an early stage may be crucial for optimizing treatment success. Despite the study's limitations, as it is based on mouse models and not yet tested in humans, there is optimism that similar pathways may exist in human patients with Parkinson's. The researchers plan further investigations to determine if this treatment could be beneficial for other variations of Parkinson's disease and possibly for other neurodegenerative conditions. The study was financially supported by The Michael J. Fox Foundation for Parkinson's Research, further highlighting the urgency and potential impact of these findings.