Projects per year
Abstract
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.
Original language | English |
---|---|
Pages (from-to) | 1299-1314 |
Number of pages | 26 |
Journal | Cell Stem Cell |
Volume | 30 |
Issue number | 10 |
DOIs | |
Publication status | Published - 2023 Oct 5 |
Subject classification (UKÄ)
- Neurology
Free keywords
- ATMP
- clinical trial
- dopamine
- minipig
- neurosurgery
- neurosurgical
- Parkinson's
- pluripotent
- regulatory
- stem cell therapy
- transplantation
Fingerprint
Dive into the research topics of 'Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD'. Together they form a unique fingerprint.Projects
- 1 Active
-
STEM-PD study
Parmar, M. (PI), Kirkeby, A. (Researcher), Barker, R. (Researcher), Paul-Visse, G. (Researcher), Widner, H. (Researcher), Bjartmarz, H. (Researcher), Nelander, J. (Researcher), Van Vliet, T. (Researcher) & Piccini, P. (Researcher)
The Swedish Parkinson Foundation, Parkinsonfonden, Swedish Research Council
2022/11/01 → …
Project: Research