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. 2023 Oct 5;30(10):1299-1314.e9.
doi: 10.1016/j.stem.2023.08.014.

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

Agnete Kirkeby  1 , Jenny Nelander  2 , Deirdre B Hoban  2 , Nina Rogelius  2 , Hjálmar Bjartmarz  3 ; Novo Nordisk Cell Therapy R&D  4 ; Petter Storm  2 , Alessandro Fiorenzano  2 , Andrew F Adler  2 , Shelby Vale  2 , Janitha Mudannayake  2 , Yu Zhang  5 , Tiago Cardoso  2 , Bengt Mattsson  2 , Anne M Landau  6 , Andreas N Glud  7 , Jens C Sørensen  7 , Thea P Lillethorup  6 , Mark Lowdell  8 , Carla Carvalho  8 , Owen Bain  8 , Trinette van Vliet  9 , Olle Lindvall  10 , Anders Björklund  2 , Bronwen Harry  11 , Emma Cutting  11 , Håkan Widner  9 , Gesine Paul  12 , Roger A Barker  13 , Malin Parmar  14
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Free article

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

Agnete Kirkeby et al. Cell Stem Cell. .
Free article

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.

Keywords: ATMP; Parkinson’s; clinical trial; dopamine; minipig; neurosurgery; neurosurgical; pluripotent; regulatory; stem cell therapy; transplantation.

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Conflict of interest statement

Declaration of interests M.P. is the owner of Parmar Cells AB. A.K. is the owner of Kirkeby Cell Therapy APS. M.P. and A.K. are co-inventors on patents WO2016162747A2/A3 and WO2019016113A1. M.P., A.K., R.A.B., H.W., H.B., A.B., E.C., D.B.H., G.P., and B.H. have performed paid consultancy for Novo Nordisk A/S, and members of NNCT R&D are current or previous employees of Novo Nordisk A/S. T.C., A.F.A., Y.Z., S.V., and D.B.H. performed the work as employees of Lund University but are currently employed by Novo Nordisk A/S (T.C., A.F.A., and S.V.), Takara Bio (Y.Z.), and D.B.H. at Eli Lilly and Company, where she is also a minor share holder. Novo Nordisk A/S is developing the STEM-PD product for commercial use.

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