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Human embryonic stem cell-derived dopaminergic cells for Parkinson's disease : a phase 1/2 open-label trial

Paul, G LU orcid ; Bjartmarz, H LU ; Kirkeby, A LU ; Nelander, J LU orcid ; Smith, R LU ; Kayhanian, S ; Evans, A ; Harry, B ; Cutting, E and Fazal, S , et al. (2026) In Nature Medicine
Abstract

Parkinson's disease (PD) is characterized by progressive loss of nigral dopaminergic neurons, resulting in disabling motor symptoms. Intracerebral transplantation of stem cell-derived dopaminergic progenitors to replace lost endogenous dopaminergic neurons offers a new potentially restorative therapeutic approach for PD. Here we report the 12-month primary safety end point and interim efficacy outcomes from a phase 1/2, open-label, multicenter trial evaluating STEM-PD, a cryopreserved, off-the-shelf dopaminergic progenitor product derived from human pluripotent stem cells. Eight individuals with moderate PD underwent bilateral intraputaminal transplantation at two escalating doses (n = 4 per cohort), followed by 12 months of... (More)

Parkinson's disease (PD) is characterized by progressive loss of nigral dopaminergic neurons, resulting in disabling motor symptoms. Intracerebral transplantation of stem cell-derived dopaminergic progenitors to replace lost endogenous dopaminergic neurons offers a new potentially restorative therapeutic approach for PD. Here we report the 12-month primary safety end point and interim efficacy outcomes from a phase 1/2, open-label, multicenter trial evaluating STEM-PD, a cryopreserved, off-the-shelf dopaminergic progenitor product derived from human pluripotent stem cells. Eight individuals with moderate PD underwent bilateral intraputaminal transplantation at two escalating doses (n = 4 per cohort), followed by 12 months of immunosuppression. Seven participants completed 12-month follow-up; one participant died from a pulmonary infection. No serious adverse events were attributed to the cell product, no graft-induced dyskinesias were observed and serial magnetic resonance imaging showed no evidence of tumor formation. These findings support the feasibility and favorable safety profile of human pluripotent stem cell-derived dopaminergic progenitor transplantation in this early-phase study, with risks primarily associated with the immunosuppression regimen. Ongoing follow-up to 36 months will further evaluate durability, clinical outcomes and graft function. ClinicalTrials.gov identifier: NCT05635409 .

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organization
publishing date
type
Contribution to journal
publication status
epub
subject
in
Nature Medicine
publisher
Nature Publishing Group
external identifiers
  • scopus:105044354359
  • pmid:42426223
ISSN
1546-170X
DOI
10.1038/s41591-026-04525-0
project
STEM-PD study
language
English
LU publication?
yes
additional info
© 2026. The Author(s).
id
29ed3356-c37f-4418-a34a-af4108f8ee0f
date added to LUP
2026-08-03 12:45:38
date last changed
2026-08-04 14:41:02
@article{29ed3356-c37f-4418-a34a-af4108f8ee0f,
  abstract     = {{<p>Parkinson's disease (PD) is characterized by progressive loss of nigral dopaminergic neurons, resulting in disabling motor symptoms. Intracerebral transplantation of stem cell-derived dopaminergic progenitors to replace lost endogenous dopaminergic neurons offers a new potentially restorative therapeutic approach for PD. Here we report the 12-month primary safety end point and interim efficacy outcomes from a phase 1/2, open-label, multicenter trial evaluating STEM-PD, a cryopreserved, off-the-shelf dopaminergic progenitor product derived from human pluripotent stem cells. Eight individuals with moderate PD underwent bilateral intraputaminal transplantation at two escalating doses (n = 4 per cohort), followed by 12 months of immunosuppression. Seven participants completed 12-month follow-up; one participant died from a pulmonary infection. No serious adverse events were attributed to the cell product, no graft-induced dyskinesias were observed and serial magnetic resonance imaging showed no evidence of tumor formation. These findings support the feasibility and favorable safety profile of human pluripotent stem cell-derived dopaminergic progenitor transplantation in this early-phase study, with risks primarily associated with the immunosuppression regimen. Ongoing follow-up to 36 months will further evaluate durability, clinical outcomes and graft function. ClinicalTrials.gov identifier: NCT05635409 .</p>}},
  author       = {{Paul, G and Bjartmarz, H and Kirkeby, A and Nelander, J and Smith, R and Kayhanian, S and Evans, A and Harry, B and Cutting, E and Fazal, S and Lao-Kaim, N P and van Vliet, T and Ullén, S and Grubor, I and Hansson, O and Piccini, P and Lindvall, O and Björklund, A and Widner, H and Parmar, M and Barker, R A}},
  issn         = {{1546-170X}},
  language     = {{eng}},
  month        = {{07}},
  publisher    = {{Nature Publishing Group}},
  series       = {{Nature Medicine}},
  title        = {{Human embryonic stem cell-derived dopaminergic cells for Parkinson's disease : a phase 1/2 open-label trial}},
  url          = {{http://dx.doi.org/10.1038/s41591-026-04525-0}},
  doi          = {{10.1038/s41591-026-04525-0}},
  year         = {{2026}},
}