Parkinson’s Disease (PD) is a neurodegenerative disease affecting millions of predominantly elderly individuals worldwide. Despite intensive efforts devoted to drug discovery, the disease remains incurable.
Pathologically, PD is characterised by the loss of a rather selective population of dopamine-producing neurons in a circumscribed region of the brain, which makes cell-based therapy a feasible option.
Proof-of-concept studies to support this approach have been conducted as early as the 1970s. This was followed by several high-profile clinical trials using foetal mesencephalic tissue as a source for transplantation, which gave mixed results. More recently, investigators have turned to induced pluripotent stem cell (iPS) technology to derive dopaminergic neurons for grafting. Notably, human trials for PD with iPS-derived cells are currently ongoing in Japan. As with every clinical trial, hope and risk are interwoven in tandem.