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Harnessing ALDH1A2 vulnerability in T-cell acute lymphoblastic leukemia

Andrieu, G. P.; Courtois, L.; Bonnet, M.; Gillet, C.; Queri, M.; Feroul, M.; Lhermitte, L.; Villarese, P.; Cabannes-Hamy, A.; Rousselot, P.; Perez, M.; Ceylan, I.; Spicuglia, S.; Asnafi, V.

2025-03-14 cancer biology
10.1101/2025.03.11.642712 bioRxiv
Show abstract

T-cell acute lymphoblastic leukemia (T-ALL) is an aggressive hematologic malignancy with limited therapeutic options, particularly in the relapsed/refractory (R/R) setting. Unlike other hematological malignancies, which benefit from targeted immunotherapies, T-ALL remains reliant on chemotherapy, leading to poor outcomes in R/R cases. Identifying novel therapeutic vulnerabilities is crucial to improving the outcomes of patients. Herein, we identify aldehyde dehydrogenase 1A2 (ALDH1A2) as a T-ALL-specific enzyme essential for leukemic cell survival. Transcriptomic and epigenetic analyses reveal its selective expression, regulated by the TAL1 oncogene. Pharmacological inhibition of ALDH1A2 using Dimate demonstrates potent anti-leukemic activity across diverse T-ALL subtypes, including primary samples of relapsed disease. These findings establish ALDH1A2 as a therapeutic target in T-ALL and support ALDH inhibition as a promising strategy to overcome drug resistance and improve treatment outcomes in R/R T-ALL.

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