Parkinson’s disease (PD) and dementia with Lewy bodies (DLB), collectively referred to as Lewy Body Diseases (LBDs), present progressive challenges. In response to the unmet clinical need to minimize the burden of disability and deaths resulting from LBDs, there is a growing demand for innovative therapies capable of slowing or preventing both PD and DLB.
Targeting GCase: A Promising Frontier
Recent drug development research has focused on the enzyme glucocerebrosidase (GCase), encoded for by the GBA1 gene. Reduced levels of GCase activity have been shown to increase PD risk and to accelerate the decline of PD patients. Trials of promising GCase-targeting therapies have recently been initiated, but work is needed to shift from recruiting the "average patient" to adopting a systematic precision medicine strategy to identify those patients most likely to benefit from GCase-targeting drugs.
Precision Medicine Approach: GRAND Project
The GCase-Responders Across Neurodegenerative Diseases (GRAND) project seeks to improve clinical trials in neurodegenerative diseases. Employing a multidisciplinary approach, we will deeply phenotype PD and DLB patients, integrating clinical, neuroimaging, and biochemical data. Machine learning will then enable a model for trial enrollment based on patients’ multimodal signatures of GCase dysfunction. The GRAND project collaborates with world-leading cohorts, encompassing >2000 individuals, fostering interdisciplinary collaboration between the University of Stavanger and Stavanger University Hospital. Our aim is to expedite the discovery of new treatments and redefine the landscape of neurodegenerative disease research.
Key funding sources
The GRAND project is supported by The University of Stavanger.