bims-scepro Biomed News
on Stem cell proteostasis
Issue of 2026–05–17
eighteen papers selected by
William Grey, University of York



  1. Cell Stem Cell. 2026 May 11. pii: S1934-5909(26)00153-0. [Epub ahead of print]
      Hepatic ketogenesis generates ketone bodies as an alternative energy source during carbohydrate restriction or ketogenic diets, yet its role in non-hepatic cell types remains poorly defined. Here, we show that leukemic stem cells (LSCs) in acute myeloid leukemia (AML) exhibit elevated ketogenesis, driven by fatty acid oxidation (FAO), to produce β-hydroxybutyrate (BHB). LSCs express high levels of 3-hydroxy-3-methylglutaryl-coenzyme A (CoA) synthase 2 (HMGCS2), the rate-limiting enzyme in ketogenesis, compared with blast cells and normal hematopoietic stem cells (HSCs). Deletion of Hmgcs2 in AML cells markedly decreases BHB levels, disrupts LSC function, and impairs leukemia progression in both mouse and human AML models while largely sparing normal hematopoiesis. Mechanistically, BHB suppresses ferroptosis by limiting pro-ferroptotic phospholipid remodeling through epigenetic regulation of fatty acid desaturase 2 (FADS2). Together, these findings identify autonomous ketogenesis as a critical metabolic program that protects LSCs from ferroptotic cell death and sustains leukemia progression.
    Keywords:  AML; BHB; FAO; HMGCS2; LSCs; acute myeloid leukemia; fatty acid oxidation; ferroptosis; ketogenesis; leukemic stem cells; lipid peroxidation; phospholipid remodeling; β-hydroxybutyrate
    DOI:  https://doi.org/10.1016/j.stem.2026.04.013
  2. Mol Ther Adv. 2026 Jun 11. 34(2): 201711
      An optimized and standardized method for ex vivo expansion of cord blood (CB) hematopoietic stem cells (HSCs) in a chemically defined medium has yet to be established. In this study, we aimed to improve ex vivo expansion of HSCs in a recently developed cytokine-free, chemically defined 3a medium. We found that the co-supplementation of the ferroptosis inhibitor ferrostatin-1 and iron chelator hinokitiol (FHK) in 3a medium significantly improves CB HSC expansion by suppressing lipid peroxidation and mitigating oxidative stress. FHK supplementation improves overall cell proliferation and promotes preferential expansion of HSCs without adversely affecting the clonogenic, engraftment, and differentiation potential of HSCs. The delayed engraftment kinetics with gradual increase in hematopoietic output further suggests that FHK treatment may preserve or expand long-term HSCs. Our findings are in alignment with recent published studies highlighting the susceptibility of HSCs to ferroptosis and corroborate the use of antioxidants to improve ex vivo expansion of HSC.
    Keywords:  HSCT; HSPC; antioxidants; cells; cord blood CD34+; ferroptosis; ferrostatin-1; hematopoietic stem cells; hinokitiol; lipid peroxidation; oxidative stress
    DOI:  https://doi.org/10.1016/j.omta.2026.201711
  3. Blood. 2026 May 12. pii: blood.2025032969. [Epub ahead of print]
      Hematopoietic stem cells (HSCs) and multipotent progenitors (MPPs) are sustained by networks of transcription factors and epigenetic regulators that prime lineage-specific programs yet maintain multipotency. Two epigenetic regulators, MLL3 and MLL4, play important but distinct roles in maintaining this balance. MLL3 promotes HSC differentiation, whereas MLL4 opposes differentiation. These activities are essential for both normal homeostasis and leukemia suppression, yet it is not clear how MLL3 and MLL4 regulate HSC and MPP gene expression to control HSC/MPP fate decisions. To resolve these mechanisms, we performed an extensive series of single cell genomic studies after conditionally deleting Mll3, Mll4 or both genes together. Mll3 deletion had only limited effects on HSC/MPP enhancer networks at steady state, whereas Mll4 deletion led to precocious activation of myeloid enhancers. Surprisingly, compound Mll3/4 deletion eliminated all myeloid, erythroid and megakaryocytic potential within the hematopoietic hierarchy and caused all progenitors to rapidly default to a B-cell-like identity. These changes were accompanied by widespread inactivation of HSC/MPP enhancers and superenhancers, and ectopic activation of B-cell superenhancers. Disabling MLL3/4 histone methyltransferase activity did not recapitulate the pervasive changes in cell identity that were observed when MLL3 and MLL4 were fully inactivated, indicating that MLL3 and MLL4 activate HSC/MPP enhancers independently from their enzymatic activities. Our findings show that HSC/MPP multipotency requires sustained tension between MLL3/4-dependent enhancers that maintain myeloid, erythroid and megakaryocyte potential, and MLL3/4-independent enhancers that prime B-cell identity. MLL3 and MLL4 therefore serve as critical linchpins of multilineage hematopoiesis.
    DOI:  https://doi.org/10.1182/blood.2025032969
  4. Cells. 2026 May 01. pii: 831. [Epub ahead of print]15(9):
      Proteasome inhibitors (PIs) are central to multiple myeloma (MM) therapy; however, resistance remains a major clinical challenge, particularly in relapsed/refractory disease. To identify functional mediators of carfilzomib (CFZ) resistance, we performed complementary gain-of-function CRISPR activation and pharmacological screening approaches. These unbiased strategies converged on the E3 ubiquitin ligase MDM2 as a modulator of PI response. MDM2 transactivation enhanced MM cell survival and accelerated recovery following CFZ exposure, supporting a causal role in proteotoxic stress tolerance. Pharmacologic inhibition of MDM2 with NVP-CGM097 synergized with CFZ across multiple PI-sensitive and PI-resistant MM cell lines, irrespective of TP53 status. Mechanistically, MDM2 inhibition induced p21 upregulation, cell-cycle arrest, and reduced c-MYC expression, accompanied by impaired activation of DNA damage response mediators. Genetic silencing of MDM2 phenocopied these effects and increased CFZ sensitivity. Importantly, the combination retained efficacy in MM-stromal co-culture models and in primary patient samples, including cases harboring del(17p), while sparing normal peripheral blood mononuclear cells. Collectively, these findings identify MDM2 as a functional driver of PI resistance and support combined MDM2 and proteasome inhibition as a rational therapeutic strategy in MM, including TP53-deficient contexts.
    Keywords:  MDM2; TP53; drug resistance; multiple myeloma; proteasome inhibitors; synthetic lethality
    DOI:  https://doi.org/10.3390/cells15090831
  5. Cell Death Dis. 2026 May 13.
      Outcomes for pediatric acute myeloid leukemia (AML) have improved significantly in recent years. However, relapsed and refractory disease remains a significant problem. The chemotherapy burden experienced by these patients makes the translational development of non-genotoxic experimental therapies attractive. We previously reported that the anti-helminth drug mebendazole induces degradation of the transcription factor MYB and has potent anti-AML activity. In the present study, we use CRISPR drop-out screening to identify genes encoding the proapoptotic regulators BAK and NOXA as hits conferring resistance to mebendazole activity in AML cells. Conversely, targeting MCL1 with a BH3-mimetic significantly enhanced the anti-AML activity of mebendazole in both AML cell lines in vitro and pediatric patient-derived xenograft (PDX) AML cells ex vivo. Treatment of mice transplanted with THP-1 AML cells or aggressive infant PDX AML cells with this drug combination significantly impaired disease progression in vivo. Our data indicate that mebendazole-induced MYB degradation in combination with MCL1 targeting is a novel non-genotoxic therapeutic strategy for pediatric AML.
    DOI:  https://doi.org/10.1038/s41419-026-08847-2
  6. Leukemia. 2026 May 15.
      Hematopoietic stem and progenitor cells support a lifetime supply of blood and immune cells and constitute a powerful cell therapy platform for hematologic diseases. In this study, we define oxygen-dependent activities of hematopoietic stem and progenitor cells. We found that lineage-defined progenitor cells from umbilical cord blood, bone marrow, and mobilized peripheral blood showed increased expansion in high oxygen, while primitive cells- including those from cord blood with in vivo potency- were maintained at higher frequencies in low physiologic O2. Single cell transcriptomic profiling of hematopoiesis under varying oxygen revealed the expected modulation of molecular hypoxia programs, including HIF and MTORc signaling. Transcriptomics also identified genes that are understudied in the context of oxygen dependency including MDM4 pathway and PRSS2, which we found is an mRNA biomarker for hematopoietic cell potency. Transcriptional changes together with biochemical validation revealed that low oxygen preserves cells with lower metabolic activity in a less proliferative state that exhibit decreased accumulation of stress markers. This likely occurs via dynamic interplay of multiple molecular programs and may drive differences in cell potency. Collectively, these data identify oxygen-sensing pathways as targets to improve cell therapies and suggest that local oxygenation dictates hematopoietic potential in anatomic niches.
    DOI:  https://doi.org/10.1038/s41375-026-02971-w
  7. Blood Immunol Cell Ther. 2025 Jun;pii: 100004. [Epub ahead of print]1(1):
      Acute myeloid leukemia (AML) is a heterogeneous hematologic malignancy characterized by clonal expansion of myeloid precursors, yet the interplay between leukemic and immune cells across disease stages remains poorly understood. Here, we used spectral flow cytometry and high-dimensional computational analyses to profile peripheral blood mononuclear cells from 72 patients with AML across 3 disease stages: newly diagnosed, remission, and relapsed/refractory. Clustering analyses identified stage-specific enrichment patterns in myeloid and lymphoid populations, with leukemic CD34+ and CD123+ cells dominating in relapsed/refractory patients and monocytic and CD45low clusters enriched in remission. T-cell profiling revealed terminal effector and senescent subsets in relapsed/refractory patients, suggesting immune exhaustion as a contributor to disease progression. Mutation-specific analyses linked TP53, DNMT3A, and NPM1 mutations to distinct enrichment patterns in both leukemic and immune populations, including increased CD71+ AML cells and altered T-cell distributions. These findings provide insights into the dynamic cellular ecosystem of AML, highlighting mutation-driven immune dysregulation and potential therapeutic targets to improve outcomes. This comprehensive profiling underscores the critical role of immune modulation in AML progression and relapse, paving the way for novel immune-targeted therapies.
    DOI:  https://doi.org/10.1016/j.bict.2025.100004
  8. bioRxiv. 2026 Feb 23. pii: 2026.02.21.707190. [Epub ahead of print]
      Aggressive subtypes of acute myeloid leukemia (AML) are characterized by increased migratory behavior and poor prognosis prioritizing the need for uncovering relevant mechanisms. While attributed to transcriptional changes, these AMLs manifest dysregulated eIF4E implicating disrupted mRNA metabolism. Here, we observed in AML mouse models, patient specimens, and cell lines that eIF4E drives motility, colonization, engraftment and AML progression. AML cells migrate utilizing Ezrin-positive pseudopods. Unexpectedly, we discovered that eIF4E interacts with Ezrin, that these physically associated factors are required and cooperated to drive an on-demand translation program in pseudopods for motility. Indeed, pseudopods were sites of eIF4E- and Ezrin-dependent translation by implementing the first method to directly mark active ribosomes in situ ( Vis ualizing T ranslation A ctivity using R iboLace, VISTA-R). Biochemically, Ezrin bound eIF4E, ribosomal components, and mRNAs consistent with our observed Ezrin-dependent modulation of protein production. This unprecedented physical coupling of motility and translation provisions migratory sites to sustain AML progression.
    Highlights: eIF4E reduction impairs AML cell motility and disease progressioneIF4E-dependent motility requires EzrinEzrin binds eIF4E, transcripts encoding motility factors and active ribosomesVISTA-R enabled visualization of active ribosomes and translationally active pseudopods (T-PODs)T-PODs provide novel on-demand localized translation to sustain mobility at migratory sites.
    DOI:  https://doi.org/10.64898/2026.02.21.707190
  9. Blood. 2026 May 12. pii: blood.2026034144. [Epub ahead of print]
      Arginine methylation catalyzed by protein arginine methyltransferases (PRMTs) is required for cancer cell proliferation, but whether PRMTs mediate resistance to therapy remains elusive. Here, we have performed loss-of-function screens in venetoclax-resistant (VEN-R) AML patient-derived xenograft (PDX) cells and found that PRMT9 plays a critical role in promoting VEN resistance. Specifically, VEN-R AML samples exhibited high levels of PRMT9, and PRMT9 inhibition re-sensitized the AML cells to VEN treatment. In preclinical resistant models, genetic ablation of PRMT9 synergized with VEN to eradicate AML cells. Consistently, pharmacologic inhibition of PRMT9 combined with VEN yielded similar effects in VEN-R AML mouse models. Mechanistically, PRMT9 ablation disrupted RNA splicing by inducing exon-skipping of mRNA encoding ALG13, an UDP-N-Acetylglucosaminyltransferase subunit, downregulating expression of a VEN-efflux transporter encoded by the adenosine triphosphate binding cassette subfamily C member 1 (ABCC1) gene. PRMT9 inhibition also suppressed protein synthesis, downregulating short-lived oncoproteins, such as MCL1. These findings establish a connection between PRMT9-mediated arginine methylation and poor VEN responsiveness, also demonstrate that targeting PRMT9 may represent a viable strategy to overcome VEN resistance.
    DOI:  https://doi.org/10.1182/blood.2026034144
  10. Am J Physiol Cell Physiol. 2026 May 13.
      Protein homeostasis is critical for mitochondrial function and is maintained by proteases and chaperones that respond to stress and mediate adaptive changes such as the mitochondrial unfolded protein response (UPRmt), the integrated stress response (ISR) and antioxidant signaling. However, the mechanisms by which stressors regulate these retrograde responses remains uncharacterized in muscle. Thus, we examined the effect of mitochondrial stressors on the activation of these pathways in myoblasts and differentiated myotubes. Cells were exposed to either 1) CDDO, a LonP1 protease inhibitor, 2) GTPP, an HSP90 chaperone inhibitor, 3) CCCP, an energetic uncoupler, or 4) MB-10, an inhibitor of protein import, and responses were compared to those induced by acute contractile activity (ACA). LonP1 inhibition activated ATF4 and Nrf2 signaling, increased mitochondrial chaperones, and resulted in protein aggregation without elevating reactive oxygen species (ROS). In contrast, blocking HSP90 led to increases in mitochondrial ROS and activation of CHOP, indicating protein homeostasis-related stress with limited antioxidant signaling. ACA elicited responses similar to the inhibition of LonP1, including the activation of ATF4 and Nrf2, increased UPRmt markers, and a redox balance. Although CCCP and MB-10 both impaired protein import, they activated distinct downstream responses. CCCP resulted in ISR activation, while MB-10 induced Nrf2-mediated antioxidant responses. Together, these findings show that the type of mitochondrial stress determines the direction of the retrograde signaling pathways between protein homeostasis and redox signaling in muscle cells, and they provide insights on how muscle coordinates signaling pathways as part of mitochondrial adaptations to contractile activity.
    Keywords:  integrated stress response; mitochondrial biogenesis; mitochondrial proteostasis; mitochondrial unfolded protein response; muscle contractile activity
    DOI:  https://doi.org/10.1152/ajpcell.00167.2026
  11. Sci Adv. 2026 May 15. 12(20): eaed7122
      TP53-mutated acute myeloid leukemia (AML) represents a particularly aggressive and therapeutically refractory subtype of the disease. While recurrent chromosomal abnormalities such as -5/del(5q), -7/del(7q), and del(17p) are well studied in this context, additional co-occurring events remain less well defined. Using the multidimensional Leucegene dataset (~700 primary AML specimens), we identified and comprehensively characterized a distinct subset of TP53-altered AML marked by recurrent deletions on the short arm of chromosome 3 [del(3p), >20% TP53-mutated cases]. These deletions frequently co-occur with del(5q) and encompass several ribosomal protein genes (RPGs), leading to a global down-regulation of the ribosomal network and reduced protein synthesis. We show that this ribosomopathy-like phenotype is most pronounced in TP53-mutated cases with combined RPG deletions on chromosomes 3p and 5q, suggesting a cooperative oncogenic mechanism. Chemical screening identified HSP90 inhibition as a selective vulnerability in AML with low RPG expression. These findings highlight a previously unappreciated TP53-altered AML subset characterized by converging genomic and translational defects and suggest that ribosomal stress may serve as a therapeutic entry point for targeted intervention of this patient subgroup.
    DOI:  https://doi.org/10.1126/sciadv.aed7122
  12. Biochem J. 2026 May 27. 483(6): 907-925
      Targeting mitochondrial oxidative phosphorylation (OxPhos) has become a recurring strategy in the treatment of cancer, particularly in acute myeloid leukemia (AML). Early reports suggested that leukemic blasts, and especially leukemia stem cells, depend disproportionately on mitochondrial respiration, implying a therapeutic window for systemic inhibition of the electron transport system (ETS) and OxPhos. Yet, the clinical record of broad OxPhos inhibition has been disappointing. In the present review, we argue that the pivotal question is not whether mitochondria matter for cancer, but whether specific mitochondrial processes are disproportionately essential to malignant cells compared with the organism's most OxPhos-dependent organs. We clarify what OxPhos is (and is not), emphasizing why oxygen consumption rate (OCR) is an incomplete surrogate for ATP-producing OxPhos flux and why transcriptomic 'OxPhos signatures' often confound energetic demand with compensatory responses to mitochondrial damage. We then benchmark OxPhos capacity and flux across normal tissues versus tumors, highlighting that highly oxidative organs typically operate at far higher respiratory flux than most cancers. Using the Complex I inhibitor IACS-010759 as a case study, we discuss why systemic ETS inhibition predictably collided with dose-limiting toxicity. Finally, focusing on AML, we dissect how OxPhos 'dependency' was inferred from indirect assays, how the failure to normalize for mitochondrial content may invert conclusions, and how ATP synthase reversal can masquerade as 'ATP-linked respiration.' We conclude with practical criteria for identifying mitochondrial liabilities that are targetable rather than merely essential, and we outline alternative strategies, which may better align mitochondrial biology with a realistic therapeutic index.
    Keywords:  acute myeloid leukaemia; complex I; mitochondria; oxidative phosphorylation
    DOI:  https://doi.org/10.1042/BCJ20260185
  13. Sci Adv. 2026 May 15. 12(20): eaec9305
      Acute myeloid leukemia (AML) is a fatal blood cancer with cytotoxic chemotherapy offering at best 25% 5-year survival. While targeted BCL2 and FLT3 inhibitors venetoclax and gilteritinib are used upfront in the treatment of a subset of adult patients with AML and help to extend the survival of some patients, a curative treatment combination with minimal side effects has yet to be discovered. We find that use of the dual histone acetyltransferase p300/CBP bromodomain inhibitor CCS1477 (inobrodib), together with venetoclax and gilteritinib, virtually eliminates leukemia stem cells in an aggressive preclinical model of DNMT3A/FLT3-mutant AML by impairing pro-oncogenic survival and proliferation factors to effectively block leukemogenesis. This work identifies potential clinical utility of a targeted, triplet combination therapy for treatment of AML.
    DOI:  https://doi.org/10.1126/sciadv.aec9305
  14. Trends Cell Biol. 2026 May 13. pii: S0962-8924(26)00065-6. [Epub ahead of print]
      Mitochondria divide and fuse, and the balance between these processes maintains mitochondrial morphology and function. Although the core fusion and division machinery is well established, how cells sense mitochondrial morphology and actively adjust it remains unclear. In this Opinion article, we propose a new conceptual framework, termed 'Mitochondrial Safeguard (MitoSafe)', in which cells monitor mitochondrial size and rebalance division and fusion through four branches: activation of fusion or inhibition of division in small mitochondria and activation of division or inhibition of fusion in enlarged mitochondria. Recent findings show that fusion is suppressed once mitochondria exceed a healthy size threshold. Dysregulation of this branch of MitoSafe, involving Parkin, PINK1, SLC25A3, SOD1, and cytochrome-c oxidase, causes mitochondrial enlargement, mitochondrial DNA release, and stimulator of interferon genes (STING)-mediated inflammation.
    Keywords:  OMA1; PINK1; Parkin; dynamin-related GTPase; inflammation; mitochondria
    DOI:  https://doi.org/10.1016/j.tcb.2026.04.007
  15. Open Life Sci. 2026 Jan;21(1): 20251322
      Previous research has established the pivotal role of bone marrow stromal cells (BMSCs) in supporting multiple myeloma (MM) pathogenesis. However, the precise molecular mechanisms underlying this role remain elusive. To investigate this, we established a co-culture system of MM cells (U266) with BMSCs (HS-5), demonstrating that HS-5 significantly promoted U266 proliferation. Subsequent RNA sequencing identified HS-5-induced differentially expressed long non-coding RNAs (lncRNAs) and mRNAs. Pathway enrichment analysis, clinical sample validation, and in vitro functional assays were then employed to elucidate the mechanistic basis of HS-5-mediated MM cell proliferation. A total of 79 downregulated RNAs and 619 upregulated RNAs induced by HS-5 were identified. Pathway enrichment analysis of these mRNAs indicated that the pantothenate and coenzyme A biosynthesis pathway functions as a convergence hub enriched by both up-regulated and down-regulated mRNAs, with ENPP1 being enriched in this pathway. Further lncRNA data analysis and validation experiments established lncRNAs OVAAL's targeting of ENPP1 and HS-5-mediated regulation of OVAAL. Additional in vitro experiments showed that OVAAL knockdown reduce the levels of LDH, GLU, and ATP and increase the levels of ROS in MM cells, which were reversed by co-culture with HS-5 cells. Knockdown of ENPP1 significantly suppresseed MM cell proliferation. This suppression could be rescued by co-culture with HS-5 stromal cells. This work indicates that BMSCs drive MM progression via OVAAL upregulation, which activates the ENPP1-mediated cells proliferation and metabolic reprogramming.
    Keywords:  LncRNA; OVAAL; bone marrow stromal cells; multiple myeloma
    DOI:  https://doi.org/10.1515/biol-2025-1322
  16. Nature. 2026 May 13.
      Cellular dormancy enables survival during prolonged nutrient limitation by reversibly suppressing protein synthesis1-4. How inactive eukaryotic ribosomes are reactivated when nutrients return remains unclear. Here, using high-resolution in situ cryo-electron tomography in Schizosaccharomyces pombe, we identify SNOR, an SBDS domain-containing ribosome-associated factor that binds at the peptidyl transferase centre and contacts the hypusinated loop of eIF5A during glucose depletion-induced dormancy. Rather than acting as a canonical hibernation factor, SNOR licenses dormant ribosomes for rapid translational restart. Upon glucose repletion, SNOR and eIF5A act together to promote efficient recovery of polysomes and exit from dormancy. These findings define a stress-responsive ribosome restart module that couples carbon-source limitation to surveillance of the ribosomal active site and reactivation of protein synthesis.
    DOI:  https://doi.org/10.1038/s41586-026-10530-7
  17. Mol Syst Biol. 2026 May 11.
      Cell state transitions underlie the emergence of diverse cell types and are traditionally defined by changes in gene expression. Yet these transitions also involve coordinated shifts in cell morphology and behavior, which remain poorly characterized in densely packed epithelia. We developed a quantitative live-imaging and computational framework to track thousands of individual cells over time in the rapidly differentiating Xenopus mucociliary epithelium (MCE). From segmentations and trajectories, we extracted dynamic features-cell and nuclear shape, movement, and position-to create a time-resolved morphodynamic dataset spanning the full course of differentiation. While single features showed high noise and low separability of ground-truth cell types, supervised machine learning revealed that integrating time-resolved features improves the prediction of final cell fate. Gradient-boosted trees and multinomial logistic regression achieved moderate but consistent accuracy, especially for abundant epithelial lineages. Key discriminants included normalized Z position, membrane-nucleus offset, and absolute experimental time, whereas movement contributed minimally to the results. Our data show that morphodynamic signatures encode predictive information about cell identity and provide a framework linking cellular dynamics with molecular state.
    DOI:  https://doi.org/10.1038/s44320-026-00212-x
  18. Blood Neoplasia. 2026 May;3(2): 100229
      Multiple myeloma (MM) is an incurable cancer of monoclonal plasma cells. Despite its dependency on the bone marrow (BM), therapies targeting the BM microenvironment are lacking, barring immunotherapies. Obesity is associated with worse outcomes in MM, and although antiobesity treatments may benefit patients with MM, this is not yet known. Moreover, those treatments have side effects, and their specific mechanisms of action are elusive because of the interconnectedness of obesity, metabolic syndrome, diet, fiber intake, gut microbiome, inflammation, and the immune system. Fatty acid-binding proteins (FABPs) play a role in obesity and other diseases, but no studies of microenvironmentally derived FABPs' effects on cancer progression exist. Therefore, we tested the hypothesis that microenvironmentally derived FABPs support MM progression using single-cell sequencing data, in vivo models, and MM Research Foundation Relating Clinical Outcomes in MM to Personal Assessment of Genetic Profile data. We found that global Fabp4/Fabp5 double-knockout (Fabp4/5 dKO) mice have modifications in body composition, immune cells, and skeletal parameters. Murine myeloma cell (Vk12598) engraftment and growth (tumor incidence) were higher in wild-type (WT) vs Fabp4/5 dKO mice. High-fat diet-fed Fabp4/5 dKO mice were further protected from metabolic and skeletal diseases, and tumor incidence was reduced, whereas survival was increased in Fabp4/5 dKO vs WT mice. Finally, low FABP5 expression in granulocyte-monocyte progenitors, typically considered immunosuppressive, is associated with improved survival of patients with MM, implicating reduced immunosuppression and improved immune-mediated tumor eradication as one mechanism of action. Overall, the data suggest that tumor-extrinsic FABP4/5 support MM progression, which, combined with previous myeloma cell-intrinsic findings, suggests targeting FABP4/5 may cause a 2-pronged attack in MM.
    DOI:  https://doi.org/10.1016/j.bneo.2026.100229