Ectopic activation of the EVI1 gene, located in the MDS1 And EVI1 Complex Locus (MECOM) on chromosomal band 3q26.2, is associated with a dismal outcome in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome. In studying transcriptomes from AML patients with chromosome 3q rearrangements, Grasedieck et al. identified significant upregulation of the Nuclear Receptor Interacting Protein 1 (NRIP1) as well as its adjacent non-coding RNA LOC101927745. They showed that NRIP1 knockdown negatively affects the proliferation and survival of 3q-rearranged AML cells and increases their sensitivity to all-trans retinoic acid, suggesting that NRIP1 is relevant for the pathogenesis of inv(3)/t(3;3) AML and could serve as a novel therapeutic target in myeloid malignancies with 3q abnormalities.

Chimeric antigen receptor (CAR) T cells targeting B-cell maturation antigen (BCMA) demonstrate appealing antitumor activity in patients with relapsed/refractory multiple myeloma (RRMM), but toxicity and short-term efficacy limit their clinical usage. In order to improve safety, overcome limited efficacy, and reduce immunogenicity from non-human-derived components, Yang et al. developed autologous CAR-BCMA T cells (CT053) expressing the fully-human BCMA-specific scFv (25C2). Their study demonstrated that CT053 had strong efficacy and a good safety profile when administered to RRMM patients.