bims-mibica Biomed News
on Mitochondrial bioenergetics in cancer
Issue of 2026–05–17
fifteen papers selected by
Kelsey Fisher-Wellman, Wake Forest University



  1. 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
  2. Elife. 2026 May 15. pii: RP102680. [Epub ahead of print]13
      Here, we identify the subunit e of F₁F₀-ATP synthase (ATP5I) as a target of metformin, a first-in-class antidiabetic biguanide. ATP5I maintains the stability of F₁F₀-ATP synthase dimers, which is crucial for shaping cristae morphology. We demonstrate that ATP5I interacts with a biguanide analogue in vitro, and disabling its expression by CRISPR-Cas9 in pancreatic cancer cells leads to the same phenotype as biguanide-treated cells, including mitochondrial morphology alterations, reduction of the NAD+/NADH ratio, inhibition of oxidative phosphorylation (OXPHOS), rescue of respiration by uncouplers, and a compensatory increase in glycolysis. Notably, metformin disrupts F₁F₀-ATP synthase oligomerization, leading to the accumulation of vestigial assembly intermediates in pancreatic and osteosarcoma cancer cells, a phenotype also observed upon ATP5I inactivation in pancreatic cancer cells. Moreover, ATP5I knockout (KO) cells exhibit resistance to the antiproliferative effects of biguanides, but reintroduction of ATP5I rescues the metabolic and antiproliferative effects of metformin and phenformin. Finally, a genome-wide CRISPR screening in NALM-6 lymphoma cells revealed that metformin-treated cells exhibit genetic interaction profiles similar to those observed with the F₁F₀-ATP synthase inhibitor oligomycin, but not with the complex I inhibitor rotenone. This provides unbiased support for the relevance of the newly proposed target.
    Keywords:  ATP5I; F1ATPase; NAD metabolism; biguanides; biochemistry; chemical biology; human; mitochondria; pancreatic cancer
    DOI:  https://doi.org/10.7554/eLife.102680
  3. Nat Commun. 2026 May 13.
      Why some tumors respond to immunotherapy ("hot" tumors) while others remain resistant ("cold" tumors) is a central challenge in oncology. Elevated RAB5A-dependent endocytosis drives tissue fluidization during the transition to invasive breast carcinoma, but its immunological consequences are unclear. Here we show that RAB5A-driven fluidization induces a mechano-metabolic stress response that disrupts the AMPK-AKAP1-DRP1 mitochondrial fission pathway, causing mitochondrial elongation. RAB5A vesicles interact with hyperfused mitochondria and promote BAX/BAK-dependent pore formation, leading to limited mitochondrial outer membrane permeabilization. This sub-lethal event is amplified by palmitoylated GASDERMIN A oligomerization on mitochondria, establishing a positive feedback loop. The resulting release of mitochondrial DNA activates the cGAS-STING innate immune pathway and drives a hyperinflammatory state. Consequently, RAB5A-expressing tumors in immunocompetent mice grow more slowly, show increased immune infiltration, and display enhanced sensitivity to immune-checkpoint blockade in a BAX/BAK-, cGAS/STING-, and mtDNA-dependent manner. These findings connect mechanical stress, mitochondrial dynamics, and innate immunity, revealing strategies to potentiate antitumor immunotherapy.
    DOI:  https://doi.org/10.1038/s41467-026-71795-0
  4. Mol Cells. 2026 May 13. pii: S1016-8478(26)00060-9. [Epub ahead of print] 100369
      Voltage dependent anion channels (VDACs 1, 2 and 3) in the outer mitochondrial membrane control the flux of anions and oxidizable substrates that sustain mitochondrial metabolism. NADH closes VDAC by binding to a pocket, conserved in all isoforms, located in the inner wall of the channel. Previously, we identified the small molecule SC18 that targets the NADH-binding pocket of VDAC1 employing computational analysis. Here, we explored the interaction between SC18 and VDAC1 using High-resolution Nuclear Magnetic Resonance spectroscopy and Molecular Dynamics simulations. Atomically resolved data precisely confirmed the computational results, showing that SC18 binds to a site on VDAC1 that partially overlaps with the NADH binding pocket. SC18, in the presence of NADH blocked the conductance of VDAC1 reconstituted in lipid bilayers. To determine the metabolic effect of SC18, we combined readouts of mitochondrial metabolism and glycolysis with functional metabolomics and proteomics. Short-term treatment with SC18 inhibited mitochondrial metabolism and ATP production. Treatment over 24 h and 48 h further reduced mitochondrial uptake of pyruvate and glutamine, utilization of tricarboxylic acid cycle intermediates, as well as lipid, DNA and amino acid synthesis. Concomitant with the inhibition of mitochondrial metabolism, cellular uptake of glucose and glutamine increased in parallel with augmented lactate release. These results indicate that compensatory enhanced glycolysis sustains ATP production after impaired mitochondrial function induced by SC18 blockage of VDAC1. Our work set a mechanistic foundation for VDAC1 inhibition as a novel strategy to target and reprogram cancer metabolism through modulation of the biosynthetic ability of mitochondria.
    Keywords:  SC18; VDAC1; cancer metabolism; glycolysis; mitochondria
    DOI:  https://doi.org/10.1016/j.mocell.2026.100369
  5. Stem Cells. 2026 May 07. pii: sxag026. [Epub ahead of print]
      Mitochondria are essential cell signaling, survival, and bioenergetic organelles that uniquely harbor a maternally inherited, multicopy genome called mitochondrial DNA (mtDNA). The occurrence or accumulation of mtDNA mutations underlies a spectrum of inherited and acquired mitochondrial syndromes and diseases and is increasingly recognized as a source of metabolic plasticity, clonal fitness, and therapy tolerance in cancer. Recent studies have revealed mitochondrial transfer as a potential mode of intercellular communication that could compensate for mtDNA mutation-associated mitochondrial dysfunction. Transfer of mitochondria can restore homeostasis in stressed recipient cells by rebuilding respiratory capacity, rebalancing redox state, and reshaping cell fate. Reported mechanisms of transfer include tunneling nanotubes, extracellular vesicles, cell fusion, and others, such as macropinocytosis. Here, we review and evaluate emerging technologies developed for mitochondrial transfer studies and define the impact of transfer on cell physiology and pathology. We discuss translational opportunities for mitochondrial transfer-based interventions, as well as how mitochondrial exchange may represent a new framework for understanding tumor heterogeneity, adaptation, and aggressiveness.
    Keywords:  Mitochondria; Mitochondrial transfer; mtDNA; techniques; transplantation
    DOI:  https://doi.org/10.1093/stmcls/sxag026
  6. 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
  7. 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
  8. Carcinogenesis. 2026 May 16. pii: bgag030. [Epub ahead of print]
      Pleural mesothelioma (PM) usually occurs many years after asbestos fiber exposure; yet the mechanisms that convert chronic damage into malignancy remain unclear. Asbestos fibers induce persistent oxidative and genomic stress that should activate apoptosis via mitochondrial outer membrane permeabilization (MOMP). MOMP normally triggers cytochrome c (cyt c) release as well as mitochondrially derived damaged-associated molecular patterns (DAMPs) resulting in downstream caspase activation which leads to DNA damage and cell death. With sublethal activation, a phenomenon known as a 'Incomplete or Minority MOMP (mMOMP)' occurs in which the cell survives the damage enabling retention and propagation of somatic mutations. We tested whether prolonged asbestos fiber exposure drives mMOMP in the mesothelial cells, MeT-5A. After culturing the cells for six months with chrysotile asbestos fibers (Chry-Asb) or crocidolite asbestos fibers (Croc-Asb), we observed an increase in clonogenicity, migration, and invasion, suggesting that transformation to a malignant-like phenotype was occurring. Next, we found increases in ROS production, cyt c release, and γH2AX phosphorylation without activation of caspase-3 and apoptotic induction. These cellular features are consistent with mMOMP. Additionally, an anti-apoptotic, mitochondrial protein, Myeloid Cell Leukemia (MCL)-1 was upregulated by asbestos and enabled mMOMP by preventing MOMP. Furthermore, we observed that asbestos-induced mMOMP was associated with features of drug-tolerant persister cells (DTPs). mMOMP facilitates avoidance of apoptosis by mesothelial cells while acquiring malignant traits. Our study indicates that mMOMP is a mechanism that promotes carcinogenesis, mitochondrial changes, and metabolic reprogramming. These findings offer a foundation for future therapeutic investigations.
    Keywords:  Asbestos; Mesothelioma; Mitochondria; incomplete MOMP
    DOI:  https://doi.org/10.1093/carcin/bgag030
  9. Health Sci Rep. 2026 May;9 e72501
       Background: The apoptotic pathway mediated by mitochondria depends on the activation of pro-apoptotic Bcl-2 proteins Bax and Bak. When they permeabilize the outer mitochondrial membrane (OMM), mitochondrial dysfunction occurs, leading to caspase activation. A recent study proposed that Bax accumulation on mitochondria increases apoptotic susceptibility, with adhesion-initiated signals regulating Bax. When adhesion signaling is inhibited, Bax translocates to the OMM, undergoes conformational change, and forms complexes that create pores. Although MOMP is well studied, mechanisms regulating Bax shuttling, non-canonical partners, and mitochondrial dynamics remain poorly understood.
    Aims: This review aims to provide insights by discussing the mechanisms through which Bax regulates its mitochondrial targeting, the mitochondrial dynamics associated with Bax translocation, and their influence to apoptosis. Understanding these processes could reveal new insights into the decision-making checkpoints that determine cell death.
    Methods: A comprehensive literature search was conducted across scientific databases for peer-reviewed publications up to 2025. Search terms included Bax retrotranslocation, mitochondrial dynamics, Bcl-2 family interactome, and apoptotic priming.
    Results: Recent evidence shows that Bax continuously retrotranslocates from mitochondria to the cytosol under survival conditions, a process driven by anti-apoptotic Bcl-2 proteins. When adhesion signaling or other survival cues are inhibited, this cycle is disrupted, leading to Bax accumulation on mitochondria, its oligomerization, and the onset of MOMP. Several mitochondrial proteins, including Drp1, MAVS, OCIAD1, PTPN1, and AKAP1, have also been linked to a mitochondrial interaction network that influences Bax localization and retrotranslocation. Proximity-labeling approaches such as BioID are further identifying new proteins that may regulate Bax's mitochondrial targeting and apoptotic priming.
    Conclusion: The mechanisms driving Bax accumulation on mitochondria and its retrotranslocation remain unclear, especially the role of mitochondrial proteins. Evidence suggests mitochondrial interaction networks influence Bax and Bak regulation. Further research is needed to define how these networks integrate with the Bcl-2 interactome to shape apoptotic decisions.
    Keywords:  Bax retrotranslocation; Bcl‐2 family proteins; apoptotic priming; cell death regulation; mitochondrial dynamics; mitochondrial outer membrane permeabilization (MOMP); protein–protein interaction networks; pro‐apoptotic signaling
    DOI:  https://doi.org/10.1002/hsr2.72501
  10. J Biol Chem. 2026 May 14. pii: S0021-9258(26)02022-3. [Epub ahead of print] 113150
      Intracellular lactic acidosis, a metabolic state newly defined in this study, is characterized by a coupled increase in intracellular lactate and proton concentrations, resulting in higher levels inside cancer cells than outside. This finding expands the Warburg paradigm: lactic acidosis is not merely extracellular but intracellular, reshaping metabolism through direct biochemical mechanisms. Acidic pH and elevated lactate jointly suppress glycolysis by inhibiting HK, PFK1 and GAPDH, enforcing a low-flux, energy-efficient state. Meanwhile, pyruvate enters the TCA cycle through a pyruvate - lactate -export - reimport - lactate - pyruvate cycle that both fuels mitochondrial metabolism and maintains lactic acidosis intracellularly and extracellularly. Lactic acidosis also reprograms anaplerosis by promoting lactate-derived oxaloacetate formation and reducing glutamine dependence. Together, these findings establish lactic acidosis as an active regulator of cancer metabolism, revealing a distinct metabolic state. This coupled lactate-proton state drives coordinated metabolic reprogramming across glycolysis and mitochondrial metabolism. representing a fundamental tumor adaptation that may be exploited to disrupt cancer metabolic resilience.
    Keywords:  TCA cycle; Warburg effect; glycolysis; lactic acidosis; tumor microenvironment
    DOI:  https://doi.org/10.1016/j.jbc.2026.113150
  11. Cells. 2026 Apr 29. pii: 811. [Epub ahead of print]15(9):
      The ATP-dependent inhibition of cytochrome c oxidase (CytOx, complex IV of the electron transport chain) is the second mechanism of respiratory control adjusting mitochondrial respiration in order to prevent excessive electron flow and reactive oxygen species (ROS) production. Here, we investigate how tricarboxylic acid (TCA) cycle metabolites and the subsequent complex I or complex II activities influence this regulatory mechanism. Therefore, CytOx activity was assessed by the oxygen consumption rate after cytochrome c (Cyt c) titration to stimulate complex IV activity in isolated rat heart mitochondria (RHM) and permeabilized AC16 cells. Mitochondrial membrane potential (Δψm) and ROS formation were analysed by flow cytometry. Our results show that TCA cycle intermediates differed in their impact on CytOx activity and subsequent ROS formation. NADH-linked substrates such as α-ketoglutarate, glutamate and malate increased respiratory capacity, but preserved ATP-dependent control of CytOx, indicating that elevated electron supply alone does not necessarily abolish ATP sensitivity. In contrast, succinate, which feeds electrons directly into complex II, strongly increased respiration causing the loss of ATP-dependent respiratory control in both model systems. Despite this strong respiratory effect, succinate induced only modest changes in mitochondrial membrane potential in isolated mitochondria, whereas permeabilized cardiomyocytes exhibited reduced polarization accompanied by increased superoxide formation. Together, these findings demonstrate that the effectiveness of ATP-dependent CytOx inhibition is influenced by TCA cycle activity and depends on the site of electron entry into the respiratory chain. Thus, substrate-dependent modulation of respiratory control links metabolite availability to mitochondrial redox regulation in cardiac cells.
    Keywords:  ATP-dependent inhibition; TCA cycle metabolites; cardiac mitochondria; complex I and II; cytochrome c oxidase; mitochondrial ROS; respiratory control
    DOI:  https://doi.org/10.3390/cells15090811
  12. 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
  13. J Transl Med. 2026 May 14.
       BACKGROUND: Relapsed/refractory (R/R) acute myeloid leukemia (AML) remains difficult to treat due to limited actionable targets and frequent drug resistance. Integrated analyses of multiple AML cohorts identified S100A9 as a candidate factor associated with disease aggressiveness and suboptimal therapeutic response in R/R AML.
    METHODS: We combined genetic perturbation of S100A9 with mitochondrial Ca2+ measurements to define its functional role in AML cells. A structure-guided virtual screening strategy was then used to identify small molecules with direct affinity for S100A9, followed by biochemical validation and anti-leukemic profiling in AML cell lines, primary patient samples, normal hematopoietic cells, and xenograft models. Transcriptomic (RNA-seq) and protein assays were performed to characterize pathway changes induced by the lead compound.
    RESULTS: S100A9 modulation altered mitochondrial Ca2+ homeostasis and AML cell fitness. We identified B2, a novel S100A9-binding small molecule that reduces S100A9 abundance and is associated with increased mitochondrial Ca2+ accumulation. RNA-seq and immunoblotting demonstrated concomitant attenuation of survival signaling, including reduced STAT5 and AKT activation. B2 preferentially impaired S100A9-high AML cell lines and primary samples with minimal toxicity to normal hematopoietic cells, and significantly reduced leukemia burden in xenograft models.
    CONCLUSIONS: These findings establish S100A9 as a regulator of mitochondrial Ca2+ homeostasis in AML and support B2 as a translational candidate that targets mitochondrial vulnerabilities and downstream survival pathways in R/R AML.
    Keywords:  Acute myeloid leukemia (AML); Calcium homeostasis; Inhibitor; Mitochondria; S100A9
    DOI:  https://doi.org/10.1186/s12967-026-08256-1
  14. EMBO Rep. 2026 May 14.
      Mitochondrial biogenesis and inheritance must be tightly coordinated with cell division to maintain mitochondrial function and cell survival. The dynamics of the mitochondrial network, including fusion and fission, are essential for mitochondrial inheritance and quality control. In budding yeast, simultaneous inhibition of both processes compromises mitochondrial DNA (mtDNA) integrity, increasing the frequency of petite cells. Loss of fusion alone completely eliminates mtDNA. Although this has been known for decades, why mtDNA is lost remained unclear. Here, we examine the effects of impaired mitochondrial fusion by depleting the mitofusin Fzo1. By analyzing over thirty thousand single cells across their cell cycles, we show that Fzo1-depletion induces rapid mitochondrial fragmentation and loss of membrane potential, followed by progressive declines in mtDNA content and growth rate. During division, Fzo1-depleted daughters inherit disproportionately large mitochondrial amounts, leaving mothers with too little. This imbalance, combined with an inability to upregulate compensatory mtDNA synthesis, drives rapid mtDNA loss. Our results reveal how fusion defects cause mtDNA loss and mitochondrial dysfunction, which might have implications for diseases linked to impaired fusion.
    DOI:  https://doi.org/10.1038/s44319-026-00794-5
  15. 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