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

Research output: Contribution to journal › Journal article › Research › peer-review

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Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD. / Parmar, Malin; Novo Nordisk Cell Therapy R&D ; Christiansen, Josefine Rågård (Member of author collaboration).

In: Cell Stem Cell, Vol. 30, No. 10, 2023, p. 1299-1314.e9.

Research output: Contribution to journal › Journal article › Research › peer-review

Harvard

Parmar, M, Novo Nordisk Cell Therapy R&D & Christiansen, JR 2023, 'Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD', Cell Stem Cell, vol. 30, no. 10, pp. 1299-1314.e9. https://doi.org/10.1016/j.stem.2023.08.014

APA

Parmar, M., Novo Nordisk Cell Therapy R&D, & Christiansen, J. R. (2023). Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD. Cell Stem Cell, 30(10), 1299-1314.e9. https://doi.org/10.1016/j.stem.2023.08.014

Vancouver

Parmar M, Novo Nordisk Cell Therapy R&D, Christiansen JR. Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD. Cell Stem Cell. 2023;30(10):1299-1314.e9. https://doi.org/10.1016/j.stem.2023.08.014

Author

Parmar, Malin ; Novo Nordisk Cell Therapy R&D ; Christiansen, Josefine Rågård. / Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD. In: Cell Stem Cell. 2023 ; Vol. 30, No. 10. pp. 1299-1314.e9.

Bibtex

@article{90227fae2a874e8ca0bb8351a5f93a15,
title = "Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD",
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, clinical trial, dopamine, minipig, neurosurgery, neurosurgical, Parkinson's, pluripotent, regulatory, stem cell therapy, transplantation",
author = "Agnete Kirkeby and Jenny Nelander and Hoban, {Deirdre B.} and Nina Rogelius and Hj{\'a}lmar Bjartmarz and Petter Storm and Alessandro Fiorenzano and Adler, {Andrew F.} and Shelby Vale and Janitha Mudannayake and Yu Zhang and Tiago Cardoso and Bengt Mattsson and Landau, {Anne M.} and Glud, {Andreas N.} and S{\o}rensen, {Jens C.} and Lillethorup, {Thea P.} and Mark Lowdell and Carla Carvalho and Owen Bain and {van Vliet}, Trinette and Olle Lindvall and Anders Bj{\"o}rklund and Bronwen Harry and Emma Cutting and H{\aa}kan Widner and Gesine Paul and Barker, {Roger A.} and Malin Parmar and {Novo Nordisk Cell Therapy R&D} and Christiansen, {Josefine R{\aa}g{\aa}rd}",
note = "Publisher Copyright: {\textcopyright} 2023 The Authors",
year = "2023",
doi = "10.1016/j.stem.2023.08.014",
language = "English",
volume = "30",
pages = "1299--1314.e9",
journal = "Cell Stem Cell",
issn = "1934-5909",
publisher = "Cell Press",
number = "10",

}

RIS

TY - JOUR

T1 - Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD

AU - Kirkeby, Agnete

AU - Nelander, Jenny

AU - Hoban, Deirdre B.

AU - Rogelius, Nina

AU - Bjartmarz, Hjálmar

AU - Storm, Petter

AU - Fiorenzano, Alessandro

AU - Adler, Andrew F.

AU - Vale, Shelby

AU - Mudannayake, Janitha

AU - Zhang, Yu

AU - Cardoso, Tiago

AU - Mattsson, Bengt

AU - Landau, Anne M.

AU - Glud, Andreas N.

AU - Sørensen, Jens C.

AU - Lillethorup, Thea P.

AU - Lowdell, Mark

AU - Carvalho, Carla

AU - Bain, Owen

AU - van Vliet, Trinette

AU - Lindvall, Olle

AU - Björklund, Anders

AU - Harry, Bronwen

AU - Cutting, Emma

AU - Widner, Håkan

AU - Paul, Gesine

AU - Barker, Roger A.

AU - Parmar, Malin

AU - Novo Nordisk Cell Therapy R&D

A2 - Christiansen, Josefine Rågård

N1 - Publisher Copyright: © 2023 The Authors

PY - 2023

Y1 - 2023

N2 - 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.

AB - 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.

KW - ATMP

KW - clinical trial

KW - dopamine

KW - minipig

KW - neurosurgery

KW - neurosurgical

KW - Parkinson's

KW - pluripotent

KW - regulatory

KW - stem cell therapy

KW - transplantation

U2 - 10.1016/j.stem.2023.08.014

DO - 10.1016/j.stem.2023.08.014

M3 - Journal article

C2 - 37802036

AN - SCOPUS:85174454209

VL - 30

SP - 1299-1314.e9

JO - Cell Stem Cell

JF - Cell Stem Cell

SN - 1934-5909

IS - 10

ER -

ID: 371289695