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Mcl1 haploinsufficiency protects mice from Myc-induced acute myeloid leukemia
Journal article   Open access   Peer reviewed

Mcl1 haploinsufficiency protects mice from Myc-induced acute myeloid leukemia

Zhifu Xiang, Hui Luo, Jacqueline E Payton, Jennifer Cain, Timothy J Ley, Joseph T Opferman and Michael H Tomasson
The Journal of clinical investigation, Vol.120(6), pp.2109-2118
06/01/2010
DOI: 10.1172/JCI39964
PMCID: PMC2877934
PMID: 20484815
url
https://doi.org/10.1172/JCI39964View
Published (Version of record) Open Access

Abstract

Antiapoptotic BCL2 family members have been implicated in the pathogenesis of acute myelogenous leukemia (AML), but the functional significance and relative importance of individual proteins (e.g., BCL2, BCL-XL, and myeloid cell leukemia 1 [MCL1]) remain poorly understood. Here, we examined the expression of BCL2 , BCL-XL , and MCL1 in primary human hematopoietic subsets and leukemic blasts from AML patients and found that MCL1 transcripts were consistently expressed at high levels in all samples tested. Consistent with this, Mcl1 protein was also highly expressed in myeloid leukemic blasts in a mouse Myc -induced model of AML. We used this model to test the hypothesis that Mcl1 facilitates AML development by allowing myeloid progenitor cells to evade Myc -induced cell death. Indeed, activation of Myc for 7 days in vivo substantially increased myeloid lineage cell numbers, whereas hematopoietic stem, progenitor, and B-lineage cells were depleted. Furthermore, Mcl1 haploinsufficiency abrogated AML development. In addition, deletion of a single allele of Mcl1 from fully transformed AML cells substantially prolonged the survival of transplanted mice. Conversely, the rapid lethality of disease was restored by coexpression of Bcl2 and Myc in Mcl1 -haploinsufficient cells. Together, these data demonstrate a critical and dose-dependent role for Mcl1 in AML pathogenesis in mice and suggest that MCL1 may be a promising therapeutic target in patients with de novo AML.

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