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

a year ago

Parkinson’s disease (PD) is caused by the loss of dopamine neurons, leading to motor symptoms.
Initial cell therapies using fetal tissues showed promise but had complications and ethical concerns.
Pluripotent stem (PS) cells emerged as a promising alternative for developing safe and effective treatments.
A phase I/II trial at Kyoto University Hospital involved seven patients receiving bilateral transplantation of dopaminergic progenitors derived from induced PS (iPS) cells.
Primary outcomes focused on safety and adverse events, with no serious adverse events reported.
Secondary outcomes assessed motor symptom changes and dopamine production for 24 months, showing improvements in some patients.
Four out of six patients showed improvements in the Movement Disorder Society Unified Parkinson’s Disease Rating Scale part III OFF score.
Five patients showed improvements in the ON scores, with average changes of 9.5 (20.4%) and 4.3 points (35.7%) for OFF and ON scores, respectively.
Fluorine-18-l-dihydroxyphenylalanine (18F-DOPA) influx rate constant (Ki) values in the putamen increased by 44.7%, with higher increases in the high-dose group.
The trial demonstrated that allogeneic iPS-cell-derived dopaminergic progenitors survived, produced dopamine, and did not form tumors, suggesting safety and potential clinical benefits.

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