Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD

Agnete Kirkeby, Jenny Nelander, Deirdre B Hoban, Nina Rogelius, Hjálmar Bjartmarz, Novo Nordisk Cell Therapy R&D, Petter Storm, Alessandro Fiorenzano, Andrew F Adler, Shelby Vale, Janitha Mudannayake, Yu Zhang, Tiago Cardoso, Bengt Mattsson, Anne M Landau, Andreas N Glud, Jens C Sørensen, Thea P Lillethorup, Mark Lowdell, Carla CarvalhoOwen Bain, Trinette van Vliet, Olle Lindvall, Anders Björklund, Bronwen Harry, Emma Cutting, Håkan Widner, Gesine Paul, Roger A Barker, Malin Parmar

Publikation: Bidrag til tidsskrift/Konferencebidrag i tidsskrift /Bidrag til avisTidsskriftartikelForskningpeer review

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.

OriginalsprogEngelsk
TidsskriftCell Stem Cell
Vol/bind30
Nummer10
Sider (fra-til)1299-1314.e9
Antal sider16
ISSN1934-5909
DOI
StatusUdgivet - okt. 2023

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