This investigation is a Phase II prospective trial designed to assess the safety and efficacy of autologous transplantation of human neural stem cell-derived dopaminergic cells into the affected striatal structures of patients suffering from Parkinson's Disease. Movement disorders neurologists blinded to the side of implantation will evaluate clinical efficacy with motor score measurements. Functional metabolism will be evaluated with [18F] Dopa PET imaging studies. The primary claim is that neural stem cell derived dopaminergic cell transplantation is a safe procedure and produces clinical benefits.

This study will involve 15 patients diagnosed with Advanced Parkinson's Disease. Following an initial needle biopsy-harvesting of neural stem cells and a 6 to 9 months expansion process, cells will be characterized and differentiated prior to unilateral injection in the putamen. There is an estimated 500,000 dopaminergic neurons in the substantia nigra of a normal individual. In the brain of a Parkinson's patient, the dopamine neuronal loss has reached over 80% when the first symptoms appear. The current study will also investigate dose escalation beween four groups of patients.

Clinical Hold

This FDA-approved study has been placed on hold pending completion of a cell processing facility meeting strict cGMP standards, including monitoring of processes, optimization of the isolation and expansion techniques and animal studies of cell migration and phenotypes after long term transplantation. NeuroGeneration is continuing its studies in human patients of harvesting, propagation and differentiation of adult human neural stem cells.