Developing a Human ES Cell Derived Dopamine Neuron Source for Cell Therapy in Parkinson's Disease

Parkinson's disease (PD) is the second most common neurodegenerative disorder and is estimated to affect 4.1-4.6 million patients world-wide, a number predicted to more than double by 2030. A fundamental characteristic of PD is progressive, severe and irreversible loss of specific dopamine-producing neurons in the midbrain, ultimately resulting in disabling motor dysfunction. Multiple therapies have been developed for PD but none can restore the function of the lost cells. Cell transplantation has been considered a promising therapy but it has been plagued by multiple challenges including the absence of an appropriate cell source. Our team has developed highly efficient strategies to obtain dopamine neurons from human embryonic stem cells. In recent publications, we demonstrated that these cells can survive in rodents and monkeys suffering from Parkinsonism and can reverse motor symptoms of the disease. In addition, they have an excellent safety profile with no evidence of tumor or excessive growth in any of the animals tested. Our multidisciplinary consortium has the overarching goal of developing an optimized, clinical-grade source of human DA neurons for cell therapy in PD. We anticipate that by the end of the project period, we will be ready to submit for FDA approval of a safety trial in Parkinson patients. We have assembled an outstanding group of scientists, neurologists, surgeons, industry leaders, ethicists, trial experts and patient advocates who will dedicate their efforts towards the achievement of this goal. Visit the Consortium's website here.