Drug discovery toward successful cell transplantation therapy for Parkinson's disease using human pluripotent stem cells

Abstract

Cell transplantation therapy using human pluripotent stem cell (PSC)–derived midbrain dopaminergic (mDA) neurons is soon expected to be available for patients with Parkinson’s disease (PD). Highly efficient and reproducible protocols for the induction of mDA neurons for clinical application have already been reported, and the therapeutic potential and safety of these cells have been studied in parkinsonian animal models as preclinical trials. However, a new strategy that improves the survival and functional quality of the grafted mDA neurons is needed to achieve maximal efficacy of the cell transplantation therapy. One strategy would definitively be to adapt the brain’s microenvironment with the use of small compounds, such as soluble factors and clinical drugs, in addition to current pharmacotherapies for PD. In this mini review, we focus on recent findings regarding the induction of mDA neurons from human PSCs toward clinical application and on a complementary strategy of drug treatment toward more efficient cell transplantation therapy for PD patients. Keywords: Parkinson’s disease; human pluripotent stem cells; cell transplantation therapy; drug discovery; clinical application (Published: 9 August 2016) Citation: Advances in Regenerative Biology 2016, 3 : 31772 - http://dx.doi.org/10.3402/arb.v3.31772