Evidence map›Paper›PMID 39952239›Full record

ArticleCell stem cell2025

Pre-clinical safety and efficacy of human induced pluripotent stem cell-derived products for autologous cell therapy in Parkinson's disease.

Jeha Jeon, Young Cha, Yean Ju Hong, In-Hee Lee, Heejin Jang, Sanghyeok Ko, Serhiy Naumenko, Minseon Kim, Hannah L Ryu, Zenith Shrestha and 12 more

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PubMed Full text at PMC Publisher
In one paragraph

Article in Cell stem cell, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

18 citing papers in PubMed.

  1. Article
  2. Review
  3. Selective CaStem cell reports · 2026
    Article
  4. Review
  5. Article
  6. Article
  7. Review
  8. Review
  9. Review
  10. Article
  11. Review
  12. Review
  13. Review
  14. Article
  15. Article
  16. Wrap it up: myelination of transplanted neurons for repair.Frontiers in cellular neuroscience · 2025
    Review
  17. Review
  18. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

22 authors.

Jeha JeonMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Program in Neuroscience, Harvard Medical School, Belmont, MA 02478, USA.
Young ChaMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Program in Neuroscience, Harvard Medical School, Belmont, MA 02478, USA.
Yean Ju HongMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Program in Neuroscience, Harvard Medical School, Belmont, MA 02478, USA.
In-Hee LeeDepartment of Pediatrics, Harvard Medical School, Boston, MA 02115, USA; Computational Health Informatics Program, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA.
Heejin JangMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Program in Neuroscience, Harvard Medical School, Belmont, MA 02478, USA.
Sanghyeok KoMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Program in Neuroscience, Harvard Medical School, Belmont, MA 02478, USA.
Serhiy NaumenkoDepartment of Biostatistics, Harvard Chan School of Public Health, Boston, MA 02215, USA.
Minseon KimMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Program in Neuroscience, Harvard Medical School, Belmont, MA 02478, USA.
Hannah L RyuMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Program in Neuroscience, Harvard Medical School, Belmont, MA 02478, USA.
Zenith ShresthaMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Program in Neuroscience, Harvard Medical School, Belmont, MA 02478, USA.
Nayeon LeeMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Program in Neuroscience, Harvard Medical School, Belmont, MA 02478, USA.
Tae-Yoon ParkMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Program in Neuroscience, Harvard Medical School, Belmont, MA 02478, USA.
HoeWon ParkMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Department of Biological Sciences, Korea Advanced Institute of Science and Technology (KAIST), Daejeon, South Korea.
Seo-Hyun KimMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Department of Biological Sciences, Korea Advanced Institute of Science and Technology (KAIST), Daejeon, South Korea.
Ki-Jun YoonDepartment of Biological Sciences, Korea Advanced Institute of Science and Technology (KAIST), Daejeon, South Korea.
Bin SongDepartment of Neurosurgery, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA.
Jeffrey SchweitzerDepartment of Neurosurgery, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA.
Todd M HerringtonDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA.
Sek Won KongDepartment of Pediatrics, Harvard Medical School, Boston, MA 02115, USA; Computational Health Informatics Program, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA.
Bob CarterDepartment of Neurosurgery, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA. Electronic address: bcarter@mgh.harvard.edu.
Pierre LeblancMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Program in Neuroscience, Harvard Medical School, Belmont, MA 02478, USA. Electronic address: pleblanc@mclean.harvard.edu.
Kwang-Soo KimMolecular Neurobiology Laboratory, McLean Hospital and Department of Psychiatry, Harvard Medical School, Belmont, MA 02478, USA; Program in Neuroscience, Harvard Medical School, Belmont, MA 02478, USA; Department of Neurosurgery, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA; Harvard Stem Cell Institute, Harvard Medical School, Cambridge, MA 02138, USA. Electronic address: kskim@mclean.harvard.edu.

Funding

Human iPSC-Based Personalized Cell Therapy of PDR01NS129188 · NINDS · MCLEAN HOSPITAL · PI Kwang-Soo Kim, Sek Won Kong · 2023 to 2026
$2.8M
Crosstalk Between Nurr1 and Risk Factors of Parkinson's Disease and its Regulation by Nurr1's LigandsR01NS127391 · NINDS · MCLEAN HOSPITAL · PI Kwang-Soo Kim · 2023 to 2026
$1.8M
Using phasic stimulation to explore the subthalamic nucleus - cortical decision-making network in Parkinson's diseaseK23NS099380 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI HERRINGTON, TODD · 2017 to 2021
$1.0M
Crosstalk between Nurr1 and risk factors of Parkinson's disease and its regulation by Nurr1's ligandsR56NS127391 · NINDS · MCLEAN HOSPITAL · PI KIM, KWANG-SOO · 2022 to 2022
$526k
NINDS NIH HHS K23 NS099380NINDS NIH HHS R01 NS127391NINDS NIH HHS R01 NS129188NINDS NIH HHS R56 NS127391
6 · The paper itself

Abstract

Human induced pluripotent stem cell (hiPSC)-derived midbrain dopaminergic cells (mDACs) represent a promising source for autologous cell therapy in Parkinson's disease (PD), but standardized regulatory criteria are essential for clinical translation. In this pre-clinical study, we generated multiple clinical-grade hiPSC lines from freshly biopsied fibroblasts of four sporadic PD patients using episomal reprogramming and differentiated them into mDACs using a refined 21-day protocol. Rigorous evaluations included whole-genome/exome sequencing, RNA sequencing, and in vivo studies, including a 39-week Good Laboratory Practice-compliant mouse safety study. While mDACs from all lines met safety criteria, mDACs from one patient failed to improve rodent behavioral outcomes, underscoring inter-individual variability. Importantly, in vitro assessments did not reliably predict in vivo efficacy, identifying dopaminergic fiber density as a key efficacy criterion. These findings support comprehensive quality control guidelines for autologous cell therapy and pave the way for a clinical trial with eight sporadic PD patients, scheduled to commence in 2025.

Indexed as

Cell- and Tissue-Based TherapyInduced Pluripotent Stem CellsParkinson DiseaseAgedAnimalsCell DifferentiationDopaminergic NeuronsFemaleHumansMaleMiceMiddle AgedTransplantation, Autologousautologous cell therapydopaminergic fiber densitygenome integrityhuman induced pluripotent stem cellsinter-individual variabilitymidbrain dopaminergic cellsParkinson's diseasephase 1 clinical trialpre-clinical studysafety and efficacy

Identifiers

PMID39952239
PMCPMC11980241

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.