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@ARTICLE{Fetsch:305544,
author = {V. Fetsch and L. F. Schwöbel and E. Ozyerli-Goknar and
A.-V. Stell and M. Punta and T. Plenge and T. Klaus and M.
K. Gupta and G. Andrieux and K. Shoumariyeh and S. Pfeiffer
and E. Corrales and L. Schlenke and H. Baniadam and S. M.
Brandl and M. Andreis and M. Remen and A. Hartmann and K.
Grueter$^*$ and M. Zwick and N. Köhler and M. Kuban$^*$ and
E. Metzger and C. Rummelt and J. Duyster and M. Börries and
M. Hofmann and J. Färber and L. M. Braun and A. Zähringer
and M. Lübbert and C. Toffalori and L. Vago and F. H.
Heidel and S. Minguet and P. Apostolova and T. Feuchtinger
and K. Maas-Bauer and F. Blaeschke$^*$ and M. W. M. Kühn
and H. T. M. Timmers$^*$ and T. Wertheimer and F. Perner and
R. Zeiser},
title = {{M}enin inhibition enhances graft-versus-leukemia effects
by {T}-cell activation and endogenous retrovirus induction
in {AML}.},
journal = {Blood},
volume = {nn},
issn = {0006-4971},
address = {Washington, DC},
publisher = {American Society of Hematology},
reportid = {DKFZ-2025-02218},
pages = {nn},
year = {2025},
abstract = {Acute myeloid leukemia (AML) carrying chromosomal
rearrangements involving the lysine methyltransferase 2A
(KMT2A) gene frequently relapse after allogeneic
hematopoietic cell transplantation (allo-HCT).
Pharmacological blockade of the menin-KMT2A interaction
disrupts the assembly of oncogenic KMT2A complexes on
chromatin, thereby attenuating aberrant self-renewal and
inducing myeloid differentiation. We found that beyond this
anti-leukemic mechanism, menin-inhibition induced CIITA and
MHC-II expression in KMT2A-rearranged and NPM1-mutated AML
cells in vitro and in vivo. Increased MHC-II expression
sensitized AML cells to T-cell mediated elimination after
allo-HCT in mice. Menin-inhibition also increased MHC-II
expression on primary human AML cells and enhanced the
graft-versus-leukemia (GVL) effect in human xenograft
models. Mechanistically, menin-inhibition increased
expression of multiple human endogenous retroviruses (HERVs)
leading to consecutive interferon-stimulated gene (ISG)
upregulation and enhanced MHC-II expression. Additionally,
menin-inhibition directly promoted anti-tumor effector
functions of donor T-cells causing increased TNF-α, IFN-ү,
perforin and granzyme A/B production and cytolytic activity.
T-cell exhaustion and menin-KMT2A binding to genes encoding
for negative regulators of T-cell activation were reduced by
menin-inhibition. These findings indicate that
menin-inhibition enhances the GVL-effect via the HERV/MHC-II
axis in AML cells and promotes cytotoxicity of donor
T-cells, which provides a rationale for a clinical trial
using menin-inhibition as maintenance after allo-HCT.},
cin = {D270 / FR01},
ddc = {610},
cid = {I:(DE-He78)D270-20160331 / I:(DE-He78)FR01-20160331},
pnm = {314 - Immunologie und Krebs (POF4-314)},
pid = {G:(DE-HGF)POF4-314},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:41144759},
doi = {10.1182/blood.2025029712},
url = {https://inrepo02.dkfz.de/record/305544},
}