Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD
Författare
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.
Avdelning/ar
- StemTherapy: National Initiative on Stem Cells for Regenerative Therapy
- WCMM- Wallenberg center för molekylär medicinsk forskning
- MultiPark: Multidisciplinary research on neurodegenerative diseases
- Institutionen för experimentell medicinsk vetenskap
- Stamcellscentrum (SCC)
- Utvecklings- och regenerativ neurobiologi
- Institutionen för kliniska vetenskaper, Lund
Publiceringsår
2023
Språk
Engelska
Sidor
1299-1314
Publikation/Tidskrift/Serie
Cell Stem Cell
Volym
30
Avvikelse
10
Dokumenttyp
Artikel i vetenskaplig tidskrift
Förlag
Cell Press
Ämne
- Neurology
Nyckelord
- ATMP
- clinical trial
- dopamine
- minipig
- neurosurgery
- neurosurgical
- Parkinson's
- pluripotent
- regulatory
- stem cell therapy
- transplantation
- SDG 3 - Good Health and Well-being
Aktiv
Published
Projekt
- STEM-PD-studien
Forskningsgrupp
- Developmental and Regenerative Neurobiology
ISBN/ISSN/Övrigt
- ISSN: 1934-5909