Phase I/II trial of iPS-cell-derived dopaminergic cells for Parkinson's disease

The replacement of lost DA neurons with hfVM in patients with PD began in the 1980s22,23. Although PS cells are expected to be an alternative donor cell source to hfVM, the safety and efficacy of iPS-cell-derived DA progenitors remains unclear. This first clinical trial using iPS cells confirmed that iPS-cell-derived DA progenitors can survive without forming tumours and produce DA in the putamen of patients with PD. Moreover, there were no serious adverse events or GIDs. Four out of six patients showed improvement in MDS-UPDRS part III OFF at 24 months after transplantation, suggesting that grafted cells functioned as DA neurons. No serious adverse events were reported for all seven patients subjected to safety assessment. The graft size measured with MRI T2-weighted images gradually increased over the 24 months. However, no tumorigenic overgrowth was identified, as evidenced by the absence of 18F-FLT uptake, a marker of cellular proliferation, which was indirectly supported by resul ... Read full article.