First Clinical Report on the Treatment of Parkinson's Disease with Fetal Midbrain Precursor Cells

Background Because human fetal ventral mesencephalic tissue grafts provide promising results in ameliorating Parkinson's disease–implicated motor dysfunctions, human fetal midbrain‐derived dopamine neuronal precursor cells are considered good candidates for cell‐based therapy for Parkinson'...

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Bibliographic Details
Published in:	Movement disorders Vol. 38; no. 4; pp. 589 - 603
Main Authors:	Kim, Joopyoung, Inbo, Han, Kim, Hyun Sook, Kim, WonChan, Jang, Su Jin, Min, Kyunghoon, Kim, Sang Heum, Bae, Sang‐Hun, Jeong, Yun‐Hwa, Kim, Borah, Kim, Chul, Schwarz, Sigrid C., Schwarz, Johannes, Cho, Kyung Gi, Chung, Sang‐Sup, Moon, Jisook
Format:	Journal Article
Language:	English
Published:	Hoboken, USA John Wiley & Sons, Inc 01-04-2023 Wiley Subscription Services, Inc
Subjects:	Arm clinical trial Cognition Computed tomography Dopamine Dopamine transporter Dyskinesia efficacy Fetuses human fetal mesencephalic dopamine neural precursor cells Humans Mesencephalon Mesencephalon - diagnostic imaging Movement disorders Neural Stem Cells Neurodegenerative diseases Parkinson Disease - drug therapy Parkinson Disease - therapy Parkinson's disease Patients Positron emission tomography Prospective Studies Safety Tomography Tomography, X-Ray Computed Transplantation clinical trial Parkinson's disease human fetal mesencephalic dopamine neural precursor cells safety efficacy
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Summary:	Background Because human fetal ventral mesencephalic tissue grafts provide promising results in ameliorating Parkinson's disease–implicated motor dysfunctions, human fetal midbrain‐derived dopamine neuronal precursor cells are considered good candidates for cell‐based therapy for Parkinson's disease in that large quantities of cells can be supplied through a good manufacturing practice–compliant system. Objective We conducted a prospective, phase I/IIa, dose‐escalation, open‐label “first‐in‐human” clinical trial with fetal neural precursor cells to assess their safety and therapeutic efficacy in patients with idiopathic Parkinson's disease. Methods Fifteen patients were assigned to receive three different doses of cells (4 × 106, 12 × 106, and 40 × 106 cells) and completed a 12‐month follow‐up. The primary outcome was safety, by measuring the presence of grade 3 or higher cells according to National Cancer Institute guidelines and any contaminated cells. Secondary outcomes assessed motor and neurocognitive function, as well as the level of dopamine transporters, by positron emission tomography–computed tomography. Results Although a pronation‐supination and hand/arm movement performance was remarkably enhanced in all three groups (all P < 0.05), the medium‐ and high‐dose‐treated groups exhibited significant improvement in Unified Parkinson's Disease Rating Scale Part III only up to 26.16% and 40%, respectively, at 12 months after transplantation without any serious clinical complications or graft‐induced dyskinesia in all patients. However, the motor improvements did not correlate with increase in the dopamine transporter on positron emission tomography images. Conclusions Our results primarily demonstrate the safety and plausible dose‐dependent efficacy of human fetal midbrain‐derived dopamine neuronal precursor cells for idiopathic Parkinson's disease. © 2023 International Parkinson and Movement Disorder Society.
Bibliography:	The authors report no conflict of interest. Relevant conflicts of interest/financial disclosures This research was supported by the Bio & Medical Technology Development Program of the NRF funded by the Korean government, MSIP (NRF‐2019M3A9H1103765). Funding agencies ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23
ISSN:	0885-3185 1531-8257
DOI:	10.1002/mds.29316