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Front-line treatment for acute myeloid leukemia (AML) has long relied on intensive cytotoxic chemotherapy, which continues to serve as the backbone of curative therapy. Despite substantial improvements in supportive care and the introduction of targeted agents for select molecular subtypes, disease-free survival remains poor for most patients, underscoring a persistent unmet clinical need. With recent advances in next-generation therapeutic modalities, particularly nucleic acid-based technologies, the question arises whether RNA-based therapies may be on the horizon for AML. In this perspective, we examined the emerging landscape of messenger RNA (mRNA)-based therapies for AML. Specifically, we provided an overview of key developments in mRNA delivery to hematopoietic cells, summarized proof-of-concept preclinical and translational studies, and discussed ongoing and completed clinical trials exploring mRNA-based cell therapies. Additionally, we highlighted promising opportunities within the field, as well as critical limitations that must be addressed to enable clinical success. By offering a broad perspective on mRNA therapeutics for AML, we aimed to clarify the therapeutic roles, constraints, and opportunities that will shape their clinical development.
Hamed et al. (2026) studied this question.