NeuroStemcell is formed to create a world-leading consortium that can take stem cell based therapies for Parkinson´s disease (PD) and Huntington´s disease (HD) to the clinic. The consortium brings 13 together elite European research teams and 3 SMEs from 6 EU member countries representing the broad range of expertise necessary to reach this goal, including stem cell specialists, developmental neurobiologists, scientists and clinicians with expertise in animal models of PD and HD and in vivo imaging, with the goal to develop safe and validated cells and clinical grade reagents to be used in clinical trials and eventually also in drug discovery. The regulatory, ethical and societal issues associated with the use of stem cells for therapy will be carefully considered as science progresses from bench to bedside.

PD and HD are ideal candidate diseases for restorative stem cell-based therapies. In both diseases the pathology is slowly progressive and characterized by the preferential loss of one type of neuron, i.e., the mesencephalic dopamine (mesDA) neurons in PD and the GABAergic medium sized spiny neurons in HD. The cell replacement strategy aims at substituting the lost mesDA and GABA neurons, respectively, by implantation of new functional cells. Although other cell types are ultimately affected, experimental evidence obtained in rodent and primate models of PD and HD, as well as the experience gained from clinical trials using grafts of fetal mesDA and striatal GABAergic progenitors, indicate that effective restorative therapies may be possible to achieve by neural transplantation in these two diseases. Further development of this approach, however, will critically depend on the development of alternative sources of therapeutically effective cells derived from stem cells.

NeuroStemcell is focused on the identification and systematic comparison of progenitor cell lines with the most favourable characteristics for mesDA and striatal GABAergic neuronal differentiation, generated either directly from human embryonic stem (ES) cells, from Neural Stem (NS) cells derived from ES cells or fetal brain, from induced Pluripotent Stem (iPS) cells or from in vitro short-term expanded neural progenitors from ventral midbrain grown as neurospheres (VMN, Ventral Midbrain Neurospheres)4, and perform rigorous and systematic testing of the most prominent candidate cells in appropriate animals models. The consortium will engage in parallel into a number of educational activities and promote the development of resources for patient groups, regulators and lay public.