Chan Wook Park

1 Introduction Parkinson disease (PD) is recognized as a multi-systemic disorder characterized not only by classic motor features, but also by a broad spectrum of non-motor symptoms (NMSs), including rapid eye movement sleep behavior disorder (RBD), dysautonomia, cognitive impairment, and olfactory dysfunction [1]. Among these, olfactory dysfunction is a highly prevalent NMS in PD that often predates motor symptom onset by years, sometimes over a decade [2-4].

Keywords cell transplantation dose-escalation trial dopaminergic differentiation dopamine progenitor dopamine neurons human embryonic stem cells Parkinson’s disease stem-cell based therapy Introduction Parkinson’s disease (PD) is a progressive neurodegenerative disorder characterized by the selective loss of dopaminergic (DA) neurons in the substantia nigra pars compacta, leading to striatal dopamine depletion and the emergence of motor symptoms.1 Although pharmacological treatments and deep...

Highlights • We generated high-purity mDA progenitors from clinical-grade hESCs on a large scale • We validated the safety and efficacy of mDA progenitors in vitro and in vivo • mDA transplantation improved behavior in PD rat models dose-dependently • A phase 1/2a clinical trial (SIT) for PD cell therapy with 12 patients is underway Summary Human embryonic stem cell (hESC)-derived midbrain dopaminergic (mDA) cell transplantation is a promising therapeutic strategy for Parkinson’s disease (PD).