National University of Ireland, Galway
Adult stem cells as vehicles for delivery of neurotrophic factors to the degenerating brain
Irish Research Council
This project will address a vital need in the treatment of Parkinsons disease (PD) which is the lack of an effective neuroprotective therapy. When a patient presents with the initial symptoms of PD there is already degeneration of up to 60% of the affected brain cells and current therapies offer no protection against further cell loss. The most promising neuroprotective therapy that has been identified for PD is a protective protein called glial cell line-derived neurotrophic factor (GDNF). However the effectiveness of GDNF in human trials has been hindered by issues relating to its delivery. Based on the vast amount of evidence in favour of the neuroprotective efficacy of GDNF, it is clear that identification of an improved delivery system for clinical application is worthy of pursuit.
This project aims to develop a novel GDNF delivery system for Parkinsons disease by genetically engineering adult bone marrow-derived stem cells to produce GDNF. These engineered cells will be transplanted into the Parkinsonian brain, and their ability to protect against the development of movement impairments and neuropathology will be assessed in models of the disease. If we can show that these stem cells can provide a long-term supply of GDNF to the brain, and prevent the development and progression of the disease, then this will highlight the potential of this approach as a novel therapy for Parkinsons disease.
Given that the incidence, and consequent personal, societal and economic costs, of neurodegenerative diseases, including Parkinsons disease, is set to escalate with the aging population, it is essential that novel disease-modifying, neuroprotective therapies are developed to treat these devastating conditions sooner rather than later.