Mclean Hospital

Video: Autologous iPSC Based Cell Therapy for Parkinson’s Disease

Available with English captions.

Penelope J. Hallett, PhD, McLean Hospital – McLean Forum Lecture

In This Presentation

Parkinson’s disease (PD) is a progressive, degenerative disease characterized by tremor, bradykinesia, rigidity, and postural instability.

At the time of diagnosis about 60% of the midbrain dopamine (mDA) neurons have already degenerated, resulting in a depletion of roughly 70% of striatal dopamine (DA) levels and synapses. Symptomatic treatment (e.g., with L-dopa) can initially restore dopamine levels and motor function, but with time often lead to side effects like dyskinesia.

Cell replacement therapy with mDA neurons provides cellular and synaptic repair in the parkinsonian brain and addresses both the motor symptoms of PD as well as levodopa-induced dyskinesias. Fetal cell transplantation work shows that in PD patients, transplanted mDA neurons remain healthy and can provide remarkable therapeutic benefit for decades.

While fetal cell transplantations are not scalable for a larger patient population and require immunosuppression, induced pluripotent stem cells (iPSCs) are a promising alternative cell source. iPSCs generated from PD patients can be differentiated into midbrain dopaminergic cells, frozen, and used for autologous transplantation.

January 14, 2021

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