Harnessing the MYB-dependent TAL1 5'super-enhancer for targeted therapy in T-ALL.


Journal

Molecular cancer
ISSN: 1476-4598
Titre abrégé: Mol Cancer
Pays: England
ID NLM: 101147698

Informations de publication

Date de publication:
18 01 2023
Historique:
received: 21 11 2022
accepted: 16 12 2022
entrez: 17 1 2023
pubmed: 18 1 2023
medline: 20 1 2023
Statut: epublish

Résumé

The acquisition of genetic abnormalities engendering oncogene dysregulation underpins cancer development. Certain proto-oncogenes possess several dysregulation mechanisms, yet how each mechanism impacts clinical outcome is unclear. Using T-cell acute lymphoblastic leukemia (T-ALL) as an example, we show that patients harboring 5'super-enhancer (5'SE) mutations of the TAL1 oncogene identifies a specific patient subgroup with poor prognosis irrespective of the level of oncogene dysregulation. Remarkably, the MYB dependent oncogenic 5'SE can be targeted using Mebendazole to induce MYB protein degradation and T-ALL cell death. Of note Mebendazole treatment demonstrated efficacy in vivo in T-ALL preclinical models. Our work provides proof of concept that within a specific oncogene driven cancer, the mechanism of oncogene dysregulation rather than the oncogene itself can identify clinically distinct patient subgroups and pave the way for future super-enhancer targeting therapy.

Identifiants

pubmed: 36650499
doi: 10.1186/s12943-022-01701-x
pii: 10.1186/s12943-022-01701-x
pmc: PMC9847025
doi:

Substances chimiques

Proto-Oncogene Proteins 0
T-Cell Acute Lymphocytic Leukemia Protein 1 0
Basic Helix-Loop-Helix Transcription Factors 0
Mebendazole 81G6I5V05I
TAL1 protein, human 135471-20-4

Types de publication

Letter Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

12

Informations de copyright

© 2023. The Author(s).

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Auteurs

Charlotte Smith (C)

Université de Paris Cité, Institut Necker Enfants-Malades INEM, Institut National de La Santé Et de La Recherche Médicale (Inserm), U1151, Paris, France.
Laboratory of Hematology, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants-Malades 75743, Paris, France.

Aurore Touzart (A)

Université de Paris Cité, Institut Necker Enfants-Malades INEM, Institut National de La Santé Et de La Recherche Médicale (Inserm), U1151, Paris, France.
Laboratory of Hematology, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants-Malades 75743, Paris, France.

Mathieu Simonin (M)

Université de Paris Cité, Institut Necker Enfants-Malades INEM, Institut National de La Santé Et de La Recherche Médicale (Inserm), U1151, Paris, France.
Laboratory of Hematology, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants-Malades 75743, Paris, France.

Christine Tran-Quang (C)

Institut Curie, Orsay, France.
CNRS UMR3348, Institut Curie, Orsay, France.
INSERM U1278, Centre Universitaire, Orsay, France.
PSL Research University, Paris, France.
University Paris-Saclay, 91400, Orsay, France.

Guillaume Hypolite (G)

Université de Paris Cité, Institut Necker Enfants-Malades INEM, Institut National de La Santé Et de La Recherche Médicale (Inserm), U1151, Paris, France.
Laboratory of Hematology, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants-Malades 75743, Paris, France.

Mehdi Latiri (M)

Université de Paris Cité, Institut Necker Enfants-Malades INEM, Institut National de La Santé Et de La Recherche Médicale (Inserm), U1151, Paris, France.
Laboratory of Hematology, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants-Malades 75743, Paris, France.

Guillaume P Andrieu (GP)

Université de Paris Cité, Institut Necker Enfants-Malades INEM, Institut National de La Santé Et de La Recherche Médicale (Inserm), U1151, Paris, France.
Laboratory of Hematology, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants-Malades 75743, Paris, France.

Estelle Balducci (E)

Université de Paris Cité, Institut Necker Enfants-Malades INEM, Institut National de La Santé Et de La Recherche Médicale (Inserm), U1151, Paris, France.
Laboratory of Hematology, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants-Malades 75743, Paris, France.

Marie-Émilie Dourthe (MÉ)

Université de Paris Cité, Institut Necker Enfants-Malades INEM, Institut National de La Santé Et de La Recherche Médicale (Inserm), U1151, Paris, France.
Laboratory of Hematology, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants-Malades 75743, Paris, France.
Department of Pediatric Hematology and Immunology, Assistance Publique-Hôpitaux de Paris (AP-HP), Robert Debré Hospital, Université de Paris Cité, Paris, France.

Ashish Goyal (A)

Division of Cancer Epigenomics, German Cancer Research Center (DKFZ), 69120, Heidelberg, Germany.

Françoise Huguet (F)

Centre Hospitalier Universitaire de Toulouse, Institut Universitaire du Cancer de Toulouse Oncopole, Laboratoire d'Hématologie, Toulouse, France.

Arnaud Petit (A)

Service d'Hématologie Et d'Oncologie Pédiatrique, AP-HP, Hôpital Armand Trousseau, Sorbonne Université, Paris, France.

Norbert Ifrah (N)

UFR Santé, Université Angers, PRES LUNAM, Centre Hospitalier-Universitaire (CHU) d'Angers, Service Des Maladies du Sang Et INSERM U892, 49933, Angers, France.

André Baruchel (A)

Department of Pediatric Hematology and Immunology, Assistance Publique-Hôpitaux de Paris (AP-HP), Robert Debré Hospital, Université de Paris Cité, Paris, France.

Hervé Dombret (H)

Université de Paris Cité, Institut Universitaire d'Hématologie, EA-3518, Assistance Publique-Hôpitaux de Paris, University Hospital Saint-Louis, Paris, France.

Elizabeth Macintyre (E)

Université de Paris Cité, Institut Necker Enfants-Malades INEM, Institut National de La Santé Et de La Recherche Médicale (Inserm), U1151, Paris, France.
Laboratory of Hematology, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants-Malades 75743, Paris, France.

Christoph Plass (C)

Division of Cancer Epigenomics, German Cancer Research Center (DKFZ), 69120, Heidelberg, Germany.
German Cancer Research Consortium (DKTK), 69120, Heidelberg, Germany.

Jacques Ghysdael (J)

Institut Curie, Orsay, France.
CNRS UMR3348, Institut Curie, Orsay, France.
INSERM U1278, Centre Universitaire, Orsay, France.
PSL Research University, Paris, France.
University Paris-Saclay, 91400, Orsay, France.

Nicolas Boissel (N)

Université de Paris Cité, Institut Universitaire d'Hématologie, EA-3518, Assistance Publique-Hôpitaux de Paris, University Hospital Saint-Louis, Paris, France.

Vahid Asnafi (V)

Université de Paris Cité, Institut Necker Enfants-Malades INEM, Institut National de La Santé Et de La Recherche Médicale (Inserm), U1151, Paris, France. vahid.asnafi@aphp.fr.
Laboratory of Hematology, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants-Malades 75743, Paris, France. vahid.asnafi@aphp.fr.

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Classifications MeSH