A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID.


Journal

Nature communications
ISSN: 2041-1723
Titre abrégé: Nat Commun
Pays: England
ID NLM: 101528555

Informations de publication

Date de publication:
30 Apr 2024
Historique:
received: 13 10 2023
accepted: 10 04 2024
medline: 1 5 2024
pubmed: 1 5 2024
entrez: 30 4 2024
Statut: epublish

Résumé

Hematopoietic stem cell gene therapy (GT) using a γ-retroviral vector (γ-RV) is an effective treatment for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency. Here, we describe a case of GT-related T-cell acute lymphoblastic leukemia (T-ALL) that developed 4.7 years after treatment. The patient underwent chemotherapy and haploidentical transplantation and is currently in remission. Blast cells contain a single vector insertion activating the LIM-only protein 2 (LMO2) proto-oncogene, confirmed by physical interaction, and low Adenosine Deaminase (ADA) activity resulting from methylation of viral promoter. The insertion is detected years before T-ALL in multiple lineages, suggesting that further hits occurred in a thymic progenitor. Blast cells contain known and novel somatic mutations as well as germline mutations which may have contributed to transformation. Before T-ALL onset, the insertion profile is similar to those of other ADA-deficient patients. The limited incidence of vector-related adverse events in ADA-deficiency compared to other γ-RV GT trials could be explained by differences in transgenes, background disease and patient's specific factors.

Identifiants

pubmed: 38688902
doi: 10.1038/s41467-024-47866-5
pii: 10.1038/s41467-024-47866-5
doi:

Substances chimiques

Adenosine Deaminase EC 3.5.4.4
Proto-Oncogene Mas 0
MAS1 protein, human 0

Types de publication

Journal Article Research Support, Non-U.S. Gov't Case Reports

Langues

eng

Sous-ensembles de citation

IM

Pagination

3662

Subventions

Organisme : Ministry of Health, Italy | Agenzia Italiana del Farmaco, Ministero della Salute (Italian Medicines Agency)
ID : GR-2018-12367860
Organisme : Fondazione Italiana per la Ricerca sul Cancro (Italian Foundation for Cancer Research)
ID : IG #22197
Organisme : Fondazione Italiana per la Ricerca sul Cancro (Italian Foundation for Cancer Research)
ID : 9962
Organisme : Fondazione Italiana per la Ricerca sul Cancro (Italian Foundation for Cancer Research)
ID : IG #21614
Organisme : Ministero dell'Istruzione, dell'Università e della Ricerca (Ministry of Education, University and Research)
ID : PRIN 2017WC8499_004
Organisme : Ministero dell'Istruzione, dell'Università e della Ricerca (Ministry of Education, University and Research)
ID : FOE 2019

Informations de copyright

© 2024. The Author(s).

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Auteurs

Daniela Cesana (D)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Maria Pia Cicalese (MP)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Università Vita-Salute San Raffaele, Milan, Italy.

Andrea Calabria (A)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Pietro Merli (P)

IRCCS Bambino Gesù Children's Hospital, Rome, Italy.

Roberta Caruso (R)

IRCCS Bambino Gesù Children's Hospital, Rome, Italy.

Monica Volpin (M)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Laura Rudilosso (L)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Maddalena Migliavacca (M)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Federica Barzaghi (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Claudia Fossati (C)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Francesco Gazzo (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Simone Pizzi (S)

Molecular Genetics and Functional Genomics, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.

Andrea Ciolfi (A)

Molecular Genetics and Functional Genomics, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.

Alessandro Bruselles (A)

Department of Oncology and Molecular Medicine, Istituto Superiore di Sanità, Rome, Italy.

Francesca Tucci (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Giulio Spinozzi (G)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Giulia Pais (G)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Fabrizio Benedicenti (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Matteo Barcella (M)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
National Research Council, Institute for Biomedical Technologies, Segrate, Italy.

Ivan Merelli (I)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
National Research Council, Institute for Biomedical Technologies, Segrate, Italy.

Pierangela Gallina (P)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Stefania Giannelli (S)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Francesca Dionisio (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Serena Scala (S)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Miriam Casiraghi (M)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Luisa Strocchio (L)

IRCCS Bambino Gesù Children's Hospital, Rome, Italy.

Luciana Vinti (L)

IRCCS Bambino Gesù Children's Hospital, Rome, Italy.

Lucia Pacillo (L)

Immune and Infectious Diseases Division, Research Unit of Primary Immunodeficiencies, Academic Department of Pediatrics, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.

Eleonora Draghi (E)

Immunogenetics, Leukemia Genomics and Immunobiology Unit, Division of Immunology, Transplantation and Infectious Diseases, Ospedale San Raffaele Scientific Institute, 20132, Milan, Italy.

Marcella Cesana (M)

Telethon Institute of Genetics and Medicine (TIGEM), Armenise/Harvard Laboratory of Integrative Genomics, Pozzuoli, Italy.
Department of Advanced Biomedical Sciences, University of Naples "Federico II", Naples, Italy.

Sara Riccardo (S)

Telethon Institute of Genetics and Medicine (TIGEM), Armenise/Harvard Laboratory of Integrative Genomics, Pozzuoli, Italy.
NEGEDIA S.r.l., Pozzuoli, Italy.

Chiara Colantuono (C)

Telethon Institute of Genetics and Medicine (TIGEM), Armenise/Harvard Laboratory of Integrative Genomics, Pozzuoli, Italy.
NEGEDIA S.r.l., Pozzuoli, Italy.

Emmanuelle Six (E)

Laboratory of Human Lympho-hematopoiesis, INSERM, Paris, France.

Marina Cavazzana (M)

Laboratory of Human Lympho-hematopoiesis, INSERM, Paris, France.

Filippo Carlucci (F)

Department of Medical Biotechnologies, University of Siena, Siena, Italy.

Manfred Schmidt (M)

Genewerk GmbH, Heidelberg, Germany.

Caterina Cancrini (C)

Immune and Infectious Diseases Division, Research Unit of Primary Immunodeficiencies, Academic Department of Pediatrics, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
Department of Systems Medicine University of Rome Tor Vergata, Rome, Italy.

Fabio Ciceri (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Università Vita-Salute San Raffaele, Milan, Italy.
Haematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy.

Luca Vago (L)

Università Vita-Salute San Raffaele, Milan, Italy.
Immunogenetics, Leukemia Genomics and Immunobiology Unit, Division of Immunology, Transplantation and Infectious Diseases, Ospedale San Raffaele Scientific Institute, 20132, Milan, Italy.
Haematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy.

Davide Cacchiarelli (D)

Telethon Institute of Genetics and Medicine (TIGEM), Armenise/Harvard Laboratory of Integrative Genomics, Pozzuoli, Italy.
Department of Translational Medicine, University of Naples "Federico II", Naples, Italy.
School for Advanced Studies, Genomics and Experimental Medicine Program, University of Naples "Federico II", Naples, Italy.

Bernhard Gentner (B)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Haematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy.

Luigi Naldini (L)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Università Vita-Salute San Raffaele, Milan, Italy.

Marco Tartaglia (M)

Molecular Genetics and Functional Genomics, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.

Eugenio Montini (E)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Franco Locatelli (F)

Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy.
Università Cattolica del Sacro Cuore, Rome, Italy.

Alessandro Aiuti (A)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy. aiuti.alessandro@hsr.it.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy. aiuti.alessandro@hsr.it.
Università Vita-Salute San Raffaele, Milan, Italy. aiuti.alessandro@hsr.it.

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