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The selective prolyl hydroxylase inhibitor IOX5 stabilizes HIF-1α and compromises development and progression of acute myeloid leukemia

Abstract:

Acute myeloid leukemia (AML) is a largely incurable disease, for which new treatments are urgently needed. While leukemogenesis occurs in the hypoxic bone marrow, the therapeutic tractability of the hypoxia-inducible factor (HIF) system remains undefined. Given that inactivation of HIF-1α/HIF-2α promotes AML, a possible clinical strategy is to target the HIF-prolyl hydroxylases (PHDs), which promote HIF-1α/HIF-2α degradation. Here, we reveal that genetic inactivation of Phd1/Phd2 hinders AML initiation and progression, without impacting normal hematopoiesis. We investigated clinically used PHD inhibitors and a new selective PHD inhibitor (IOX5), to stabilize HIF-α in AML cells. PHD inhibition compromises AML in a HIF-1α-dependent manner to disable pro-leukemogenic pathways, re-program metabolism and induce apoptosis, in part via upregulation of BNIP3. Notably, concurrent inhibition of BCL-2 by venetoclax potentiates the anti-leukemic effect of PHD inhibition. Thus, PHD inhibition, with consequent HIF-1α stabilization, is a promising nontoxic strategy for AML, including in combination with venetoclax.

Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1038/s43018-024-00761-w

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Role:
Author
ORCID:
0000-0002-9463-253X


Publisher:
Springer Nature
Journal:
Nature Cancer More from this journal
Publication date:
2024-04-18
Acceptance date:
2024-03-15
DOI:
EISSN:
2662-1347
Pmid:
38637657


Language:
English
Keywords:
Pubs id:
1991403
Local pid:
pubs:1991403
Deposit date:
2024-05-01
ARK identifier:

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