Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD
Author
Summary, in English
Cell replacement therapies for Parkinson's disease (PD) based on transplantation of pluripotent stem cell-derived dopaminergic neurons are now entering clinical trials. Here, we present quality, safety, and efficacy data supporting the first-in-human STEM-PD phase I/IIa clinical trial along with the trial design. The STEM-PD product was manufactured under GMP and quality tested in vitro and in vivo to meet regulatory requirements. Importantly, no adverse effects were observed upon testing of the product in a 39-week rat GLP safety study for toxicity, tumorigenicity, and biodistribution, and a non-GLP efficacy study confirmed that the transplanted cells mediated full functional recovery in a pre-clinical rat model of PD. We further observed highly comparable efficacy results between two different GMP batches, verifying that the product can be serially manufactured. A fully in vivo-tested batch of STEM-PD is now being used in a clinical trial of 8 patients with moderate PD, initiated in 2022.
Department/s
- StemTherapy: National Initiative on Stem Cells for Regenerative Therapy
- WCMM-Wallenberg Centre for Molecular Medicine
- MultiPark: Multidisciplinary research on neurodegenerative diseases
- Department of Experimental Medical Science
- Stem Cell Center
- Developmental and Regenerative Neurobiology
- Department of Clinical Sciences, Lund
Publishing year
2023
Language
English
Pages
1299-1314
Publication/Series
Cell Stem Cell
Volume
30
Issue
10
Document type
Article
Publisher
Cell Press
Topic
- Neurology
Keywords
- ATMP
- clinical trial
- dopamine
- minipig
- neurosurgery
- neurosurgical
- Parkinson's
- pluripotent
- regulatory
- stem cell therapy
- transplantation
- SDG 3 - Good Health and Well-being
Status
Published
Project
- STEM-PD study
Research group
- Developmental and Regenerative Neurobiology
ISBN/ISSN/Other
- ISSN: 1934-5909