Combining CRISPR-Cas9 and TCR exchange to generate a safe and efficient cord blood-derived T cell product for pediatric relapsed AML.

CD8-Positive T-Lymphocytes Cell Engineering Hematologic Neoplasms Pediatrics T cell Receptor - TCR

Journal

Journal for immunotherapy of cancer
ISSN: 2051-1426
Titre abrégé: J Immunother Cancer
Pays: England
ID NLM: 101620585

Informations de publication

Date de publication:
05 Apr 2024
Historique:
accepted: 18 03 2024
medline: 6 4 2024
pubmed: 6 4 2024
entrez: 5 4 2024
Statut: epublish

Résumé

Hematopoietic cell transplantation (HCT) is an effective treatment for pediatric patients with high-risk, refractory, or relapsed acute myeloid leukemia (AML). However, a large proportion of transplanted patients eventually die due to relapse. To improve overall survival, we propose a combined strategy based on cord blood (CB)-HCT with the application of AML-specific T cell receptor (TCR)-engineered T cell therapy derived from the same CB graft. We produced CB-CD8 The gene editing and transduction procedures achieved high efficiency, with up to 95% of cells lacking eTCR and over 70% of T cells expressing rWT1-TCR. WT1-TCR-engineered T cells lacking the expression of their eTCR (eTCR In summary, we show the feasibility of developing a potent CB-derived CD8

Sections du résumé

BACKGROUND BACKGROUND
Hematopoietic cell transplantation (HCT) is an effective treatment for pediatric patients with high-risk, refractory, or relapsed acute myeloid leukemia (AML). However, a large proportion of transplanted patients eventually die due to relapse. To improve overall survival, we propose a combined strategy based on cord blood (CB)-HCT with the application of AML-specific T cell receptor (TCR)-engineered T cell therapy derived from the same CB graft.
METHODS METHODS
We produced CB-CD8
RESULTS RESULTS
The gene editing and transduction procedures achieved high efficiency, with up to 95% of cells lacking eTCR and over 70% of T cells expressing rWT1-TCR. WT1-TCR-engineered T cells lacking the expression of their eTCR (eTCR
CONCLUSIONS CONCLUSIONS
In summary, we show the feasibility of developing a potent CB-derived CD8

Identifiants

pubmed: 38580329
pii: jitc-2023-008174
doi: 10.1136/jitc-2023-008174
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Informations de copyright

© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

Déclaration de conflit d'intérêts

Competing interests: JK reports grants from Gadeta, Novartis, and Miltenyi Biotec and is the inventor of patents dealing with γδT cell-related aspects, as well as the cofounder and shareholder of Gadeta. ZS is an inventor of patents dealing with γδT cell-related aspects.

Auteurs

Vania Lo Presti (V)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.
Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.

Angelo Meringa (A)

Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.

Ester Dunnebach (E)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.

Alice van Velzen (A)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.

Aida Valera Moreira (AV)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.

Ronald W Stam (RW)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.

Rishi S Kotecha (RS)

Department of Clinical Haematology, Oncology, Blood and Marrow Transplantation, Perth Children's Hospital, Perth, Western Australia, Australia.
University of Western Australia, Perth, Western Australia, Australia.

Anja Krippner-Heidenreich (A)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.

Olaf T Heidenreich (OT)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.

Maud Plantinga (M)

Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.

Annelisa Cornel (A)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.
Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.

Zsolt Sebestyen (Z)

Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.

Jurgen Kuball (J)

Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.

Niek P van Til (NP)

Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.
Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam and Amsterdam Neuroscience, Cellular & Molecular Mechanisms, Amsterdam, The Netherlands.
Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands.

S Nierkens (S)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands S.Nierkens-2@prinsesmaximacentrum.nl.
Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.

Classifications MeSH