Disrupting tRNA modifications to target mitochondrial vulnerabilities in drug-resistant leukemia cells

C Cornelius Pauli (7Department of Medicine V, Hematology, Oncology and Rheumatology, University of Heidelberg, Heidelberg, Germany) M Michael Kienhöfer (1Division of Mechanisms Regulating Gene Expression, German Cancer Research Center, Heidelberg, Germany) M Maximilian Felix Blank (2Medical Faculty Heidelberg and Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg, Germany) O Oguzhan Begik C Christian Rohde (1Department of Internal Medicine V, Hematology, Oncology, and Rheumatology, University Hospital Heidelberg, Heidelberg, Germany) S Sarah Miriam Naomi Zimmermann (3Molecular Medicine Partnership Unit European Molecular Biology Laboratory–University Hospital Heidelberg, European Molecular Biology Laboratory, Heidelberg, Germany) L Laura Werner (2Medical Faculty Heidelberg and Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg, Germany) D Daniel Heid (2Medical Faculty Heidelberg and Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg, Germany) F Fu Xu K Katharina Weidenauer (2Medical Faculty Heidelberg and Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg, Germany) S Sylvain Delaunay N Nadja Krall (1Division of Mechanisms Regulating Gene Expression, German Cancer Research Center, Heidelberg, Germany) K Katrin Trunk (1Division of Mechanisms Regulating Gene Expression, German Cancer Research Center, Heidelberg, Germany) D Duoduo Zhao (2Medical Faculty Heidelberg and Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg, Germany) F Fengbiao Zhou (2Medical Faculty Heidelberg and Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg, Germany) L Laia Llovera (5Epitranscriptomics and RNA Dynamics, Centre for Genomic Regulation, The Barcelona Institute of Science and Technology, Barcelona, Spain) A Alexane Ollivier (5Epitranscriptomics and RNA Dynamics, Centre for Genomic Regulation, The Barcelona Institute of Science and Technology, Barcelona, Spain) A Anke Heit-Mondrzyk (1Division of Mechanisms Regulating Gene Expression, German Cancer Research Center, Heidelberg, Germany) U Uwe Platzbecker C Claudia Baldus (7Department of Hematology, Kiel University Hospital, Kiel, Germany) H Hubert Serve M Martin Bornhäuser C Cathrine Broberg Vågbø (10Proteomics and Modomics Experimental Core Facility, Norwegian University of Science and Technology and St. Olavs Hospital, Trondheim, Norway) S Salvador Aznar Benitah J Jeroen Krijgsveld E Eva Maria Novoa C Carsten Müller-Tidow M Michaela Frye (1Division of Mechanisms Regulating Gene Expression, German Cancer Research Center, Heidelberg, Germany)

Abstract

Abstract Dysregulated RNA modifications contribute to cancer progression and therapy resistance, yet the underlying mechanism often remains unknown. Here, we perform CRISPR-based synthetic lethality screens to systematically explore the role of RNA modifications in mediating resistance to antileukemic drugs. We identify the tRNA methyltransferase 5 (TRMT5)–mediated formation of N1-methylguanosine (m1G) in the transfer RNA (tRNA) anticodon loop as essential for mediating drug tolerance to cytarabine and venetoclax (Ven) in acute myeloid leukemia (AML). TRMT5 methylates nearly all mitochondrial and nuclear tRNAs with a guanosine at position 37, but its role in promoting drug tolerance specifically depends on its mitochondrial function. TRMT5 is essential for the dynamic upregulation of mitochondrial messenger RNA translation and oxidative phosphorylation, which are critical for sustaining drug tolerance in leukemia cells. This mitochondrial dependency correlates with therapy outcomes in patients with leukemia: lower expression of electron transport chain genes is linked to poorer outcomes in a cohort of nearly 100 patients with AML undergoing first induction therapy. Finally, we demonstrate that targeted depletion of the TRMT5 protein using a conditional degron, in conjunction with cytarabine and Ven treatment, synergistically induces cell death in drug-tolerant AML cells. Thus, our study reveals TRMT5 as a promising drug target for therapy-resistant leukemia.

Article Details

Journal Blood
Volume / Issue Vol. 146, Issue 20
Published November 13, 2025
Pages 2443-2456
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (28)

C

Cornelius Pauli

7Department of Medicine V, Hematology, Oncology and Rheumatology, University of Heidelberg, Heidelberg, Germany

M

Michael Kienhöfer

1Division of Mechanisms Regulating Gene Expression, German Cancer Research Center, Heidelberg, Germany

M

Maximilian Felix Blank

2Medical Faculty Heidelberg and Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg, Germany

O

Oguzhan Begik

C

Christian Rohde

1Department of Internal Medicine V, Hematology, Oncology, and Rheumatology, University Hospital Heidelberg, Heidelberg, Germany

S

Sarah Miriam Naomi Zimmermann

3Molecular Medicine Partnership Unit European Molecular Biology Laboratory–University Hospital Heidelberg, European Molecular Biology Laboratory, Heidelberg, Germany

L

Laura Werner

2Medical Faculty Heidelberg and Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg, Germany

D

Daniel Heid

2Medical Faculty Heidelberg and Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg, Germany

F

Fu Xu

K

Katharina Weidenauer

2Medical Faculty Heidelberg and Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg, Germany

S

Sylvain Delaunay

N

Nadja Krall

1Division of Mechanisms Regulating Gene Expression, German Cancer Research Center, Heidelberg, Germany

K

Katrin Trunk

1Division of Mechanisms Regulating Gene Expression, German Cancer Research Center, Heidelberg, Germany

D

Duoduo Zhao

2Medical Faculty Heidelberg and Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg, Germany

F

Fengbiao Zhou

2Medical Faculty Heidelberg and Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg, Germany

L

Laia Llovera

5Epitranscriptomics and RNA Dynamics, Centre for Genomic Regulation, The Barcelona Institute of Science and Technology, Barcelona, Spain

A

Alexane Ollivier

5Epitranscriptomics and RNA Dynamics, Centre for Genomic Regulation, The Barcelona Institute of Science and Technology, Barcelona, Spain

A

Anke Heit-Mondrzyk

1Division of Mechanisms Regulating Gene Expression, German Cancer Research Center, Heidelberg, Germany

U

Uwe Platzbecker

C

Claudia Baldus

7Department of Hematology, Kiel University Hospital, Kiel, Germany

H

Hubert Serve

M

Martin Bornhäuser

C

Cathrine Broberg Vågbø

10Proteomics and Modomics Experimental Core Facility, Norwegian University of Science and Technology and St. Olavs Hospital, Trondheim, Norway

S

Salvador Aznar Benitah

J

Jeroen Krijgsveld

E

Eva Maria Novoa

C

Carsten Müller-Tidow

M

Michaela Frye

1Division of Mechanisms Regulating Gene Expression, German Cancer Research Center, Heidelberg, Germany