NeuroStemcell has the following ten major goals, and corresponding expected outcomes
To develop protocols for production of transplantable mesDA and striatal GABAergic progenitors from human ES and NS cell lines, building on recent developments in stem cell biology;
To carry out pre-clinical testing of stem cell-derived mesDA and striatal GABAergic progenitors in rodent models of PD and HD with the goal to generate efficient and safe candidate cells for clinical use;
To identify procedures and safety systems that will allow complete elimination of proliferative and/or tumour-forming cells from the graft cell preparations;
To apply in vivo imaging tools for non-invasive monitoring of the survival and growth of the grafted cells, as well as tools to reveal adverse immune/inflammatory reactions to the graft;
To develop criteria, procedures and protocols for reproducible, safe and large-scale manufacture of stem cells and their progeny to clinical grade standard;
To establish a banked stock of validated, safety-qualified and traceable cells for clinical use;
To develop clinical protocols to be used in phase-I trials in patients with PD and HD;
To establish ethical and safety criteria to guide implementation of stem cell therapies in the clinic;
To develop an interface with bio-industry and devise a product-specific regulatory strategy that is compatible with a future marketing authorization;
To provide accessible information to patient organizations and lay public about the advancement in the field and future critical steps to be fulfilled;
