Sawamoto N et al., Nature 2025 (phase I/II trial of iPS cell-derived dopaminergic cells in Parkinson's disease)

PMID 40240591

Phase I/II trial in Kyoto: 7 patients aged 50 to 69 received bilateral grafts of dopaminergic progenitors from allogeneic induced pluripotent stem cells and were followed for 24 months. No serious adverse events (73 mild to moderate); no graft overgrowth or tumours on MRI; dyskinesia increased with medication doses kept unchanged. In 6 patients assessed for efficacy, open-label without a sham group, the authors reported motor-score improvement off medication in 4 and on medication in 5; putaminal dopamine uptake on PET rose by 44.7%.