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Mechanismmoderate confidence 3 of 5: One strong study, or several smaller consistent ones.Q54-immune

The class I and class II arms of menin inhibition are different mechanisms - the class I effect was tested against viral mimicry and NF-kB and excluded both

also called STAT1-independent MHC-I derepression · class I versus class II route under menin inhibition · viral mimicry excluded for MHC class I

Two teams found that a menin inhibitor makes leukaemia cells more visible to the immune system, and it is tempting to assume they found the same thing. They did not: one route works by waking up ancient viral DNA and triggering an alarm, and the other explicitly does not need that alarm at all.

The same thing, for a scientist

This graph holds two menin-inhibitor immunology stories and they are routinely collapsed into one. `cell-state-interferon-herv-mhc2` records Fetsch et al.: menin inhibition induces **human endogenous retroviruses**, which drives **interferon-stimulated genes**, which raises **CIITA and MHC class II** [E3]. Sparbier et al. tested that same chain for **class I** and excluded it point by point: **STAT1 depletion**, which abolishes interferon-induced MHC-I upregulation, **did not impair** MHC-I activation after menin targeting; **MDA-5/IFIH1 depletion**, the cytoplasmic dsRNA sensor on which viral mimicry depends, did not impair it; and **RELA was not required**, with no rise in TNF-alpha-induced p65 phosphorylation in menin- or LEDGF-deficient cells [E1]. The positive mechanism is chromatin: menin is **not bound** at bivalent MHC-I promoters, and the effect is redistribution of KMT2A from active genes onto a small set of bivalent loci where H3K4me3 rises and H3K27me3 and SUZ12 fall [E1]. **Two arms, two mechanisms, two different sets of things that would break them** - and only one of them is an interferon story.

Go deeper
Why the conflation matters practically

If the two arms were one mechanism, then one biomarker, one combination partner and one resistance mechanism would serve both. They are not, and they do not:

class II armclass I arm
proposed routeHERV -> dsRNA -> ISG -> CIITA 3; and menin occupancy at the CIITA locus 2 - contested, see belowKMT2A redistribution altering the polycomb balance at bivalent promoters 1
requires STAT1presumably yes (ISG-driven)no, tested 1
requires MDA-5presumably yes (dsRNA sensing)no, tested 1
requires NF-kB/RELAnot testedno, tested 1
requires KMT2A/KMT2Bnot testedyes, tested 1
genetic proof of the transactivatoryes - CIITA-null MV4-11 loses the induction 2no - NLRC5 never tested under a menin inhibitor, though Zhou's locus set places NLRC5 in the menin-bound group
blocked by a JAK inhibitorexpected yesexpected no

The last row is the one that has a patient in it. An MLLT10-rearranged leukaemia has a published rationale for a JAK inhibitor (gene-jak1), and a JAK inhibitor would be expected to remove the interferon-dependent arm and leave the chromatin arm running.

What 'excluded' does and does not mean

The exclusions are single-cell-line CRISPR depletions in K-562, reported with immunoblot confirmation 1. Three limits, stated plainly:

  • They test necessity, not presence. In a cell receiving interferon, STAT1 signalling still raises class I; the finding is that the menin-inhibitor effect does not require it. Both routes operate together in any real setting.
  • They are one cell line. No KMT2A-rearranged cell was tested, and neither was any cell in which HERVs had been shown to be induced. Fetsch's HERV induction was measured in AML; Sparbier's exclusion was measured in CML. The two papers may simply be describing two different cells.
  • They do not exclude a HERV contribution to class I in a cell where HERVs are strongly induced. A cell can have an MDA-5-independent chromatin route and an MDA-5-dependent interferon route at once, and the relative sizes would be cell-dependent.

What survives is narrow and useful: nobody may write, in this graph, that menin inhibition raises MHC class I by viral mimicry. The one experiment that tested it says otherwise.

The open contradiction, stated as a contradiction

Fetsch reports interferon-stimulated gene upregulation as the mechanism of enhanced antigen presentation in KMT2A-rearranged AML 3. Sparbier reports that antigen presentation rises after menin targeting without STAT1 or MDA-5, in K-562 1. Hogeling reports the class II arm as CIITA-dependent and MEIS1-independent, with menin bound at the CIITA locus, and says nothing about interferon signalling as the route 2.

Three papers, three proposed mechanisms, one drug class, overlapping phenotypes. That is not a defect in any of them; it is an unresolved question that has never been posed as one. The discriminating experiment is small: menin inhibitor in a KMT2A-rearranged line, with and without STAT1 knockout, reading class I and class II separately and HERV transcripts alongside. It is written up as exp-mhc-class-i-and-nk-ligand-timecourse-under-menin-inhibition-kmt2a-r.

Where this stands against the transplant beat's MHC-II headroom hypothesis: concord on the conclusion, contest on the argument, and it does not transfer to class I

hyp-menin-inhibition-restores-gvl-only-where-mhc-ii-is-limiting (Q53-transplant, same cycle) argues that because MHC class II is constitutively expressed in the B lineage, the antigen-presentation arm of the Fetsch mechanism has little headroom in B-precursor KMT2A::AFF1 infant ALL, so any post-transplant benefit there must come from the T-cell-intrinsic arm alone. This beat's class II material bears on it in three ways, and they do not all point the same way.

1. Concord on the conclusion. Nothing here contradicts the prediction that MHC-II headroom is small in B-precursor disease. This beat independently flagged the mirror-image concern: the bleximenib class II result was obtained in myelomonocytic cells (MV4-11, MOLM13, primary AML) which express class II constitutively, and no source in this beat reports a lineage-matched control for it 2. Both beats are pointing at the same missing control from opposite ends.

2. Contest on the argument, with a named discriminator. 'Constitutively expressed' is a statement about a flow gate, not about a ceiling - and the bleximenib data show the distinction matters. MV4-11 is constitutively class II-positive and the drug still raised HLA-DR dose-dependently in it, while CIITA-null MV4-11 had significantly lower baseline HLA-DR and did not respond 2. So a constitutively class II-positive cell demonstrably retained headroom. The premise 'B-lineage cells are HLA-DR-positive, therefore there is nothing to gain' does not follow on its own; what would settle it is a quantitative baseline against the achievable maximum - an isotype-referenced MFI and a CIITA-saturating positive control such as IFN-gamma - not a positive/negative call. That measurement is in exp-mhc-ii-headroom-under-menin-inhibition-in-b-precursor-infant-all, and this note is a request that it be read quantitatively rather than as a gate.

3. It does not transfer to class I, and for class I the sign may be opposite. The headroom argument is scoped to class II and rests on constitutive B-lineage expression. Class I is expressed by essentially every nucleated cell, so a naive headroom argument would predict no class I benefit anywhere - yet the class I effect is exactly what PMID 36635503 and PMID 39704147 measure, because the relevant variable is not whether the gene is on but whether its promoter is polycomb-restrained 1. Baseline class I on a KMT2A-rearranged blast has never been measured, so headroom is unknown in both directions.

And the asymmetry matters therapeutically. If class II headroom is absent, the conclusion is nothing to gain. If class I headroom is absent because class I is already high - which is what the MLL-AF9 NK-dependency data suggest (mechanism-mhc-class-i-is-an-nk-brake-in-kmt2a-fusion-leukaemia) - the conclusion is something to lose. Two hypotheses with the same shape and opposite consequences, and only one measurement separates them.

Update: a genome-wide locus set splits the two transactivators the opposite way round

Added after the King's join of this graph onto Zhou's published 9,292-locus menin/H2AK119ub target set in KMT2A-rearranged AML (PMID 39158067).

transactivatorZhou groupreading
CIITA (class II)H2AK119ub solo, chr16:10877200polycomb-marked, menin-unbound
NLRC5 (class I)Menin solo, chr16:56989555menin-bound, no H2AK119ub

This cuts two ways at once, and both are useful.

It supports this entry's central claim that the class I and class II arms are different mechanisms - and it supplies independent, orthogonal evidence for it, from a chromatin target list rather than from a depletion experiment.

But it inverts the directionality this entry assumed. The table above was built expecting class II to be the direct arm (menin at CIITA by ChIP-qPCR) and class I to be the indirect one (no menin at MHC-I promoters). Zhou's set says the reverse at the transactivator level: class II's transactivator is menin-free and polycomb-marked, class I's is menin-bound. If that holds, the honest formulation is that both arms are indirect with respect to the presented genes, and the class I arm may have a second, direct route through NLRC5 that no published model includes.

Caveats that keep this from being a conclusion. Occupancy is not function - Sparbier found VTP50469 produced remarkably few significant expression changes despite globally reducing menin occupancy, so a menin-solo NLRC5 need not move under the drug. The system is adult myeloid KMT2A-rearranged AML. And absence from Zhou's called set is absence from a thresholded list, not demonstrated absence of binding. The test is one qPCR: NLRC5 transcript under a menin inhibitor. It has never been run.

Facts and figures
discriminating experimentmenin inhibitor in a KMT2A-rearranged line, +/- STAT1 knockout, reading class I and class II separately with HERV transcripts alongside
prohibited claimmenin inhibition raises MHC class I by viral mimicry - excluded by the only experiment that tested it
arms
class_I2,023class_I: 2023class_II_route_b2,025class_II_route_b: 2025class_II_route_a2,026class_II_route_a: 2026
arms — the numbers behind the chart
{
  "class_I": {
    "route": "KMT2A/KMT2B redistribution altering polycomb balance at bivalent promoters",
    "requires_STAT1": false,
    "requires_IFIH1_MDA5": false,
    "requires_RELA": false,
    "requires_KMT2A_KMT2B": true,
    "menin_bound_at_promoters": false,
    "transactivator_knockout_proof": "none - NLRC5 never tested under a menin inhibitor",
    "pmid": "36635503",
    "title": "Targeting Menin disrupts the KMT2A/B and polycomb balance to paradoxically activate bivalent genes.",
    "authors": "Sparbier CE, Gillespie A, Gomez J, et al.",
    "venue": "Nat Cell Biol",
    "year": 2023,
    "title_provenance": "Title/authors/venue backfilled from PubMed esummary on 2026-09-20 because this citation stored a bare PMID with no title, which made it unscoreable by verify-citations.py. BACKFILLING IS NOT VERIFICATION: it confirms the PMID resolves to a real paper, not that this is the right paper for the claim. That still requires reading."
  },
  "class_II_route_a": {
    "route": "HERV derepression -> dsRNA -> interferon-stimulated genes -> CIITA -> MHC-II",
    "pmid": "41144759",
    "graph_entry": "cell-state-interferon-herv-mhc2",
    "title": "Menin inhibition enhances graft-versus-leukemia effects by T-cell activation and endogenous retrovirus induction in AML.",
    "authors": "Fetsch V, Schwöbel L, Ozyerli-Goknar E, et al.",
    "venue": "Blood",
    "year": 2026,
    "title_provenance": "Title/authors/venue backfilled from PubMed esummary on 2026-09-20 because this citation stored a bare PMID with no title, which made it unscoreable by verify-citations.py. BACKFILLING IS NOT VERIFICATION: it confirms the PMID resolves to a real paper, not that this is the right paper for the claim. That still requires reading."
  },
  "class_II_route_b": {
    "route": "direct menin occupancy at the CIITA locus -> CIITA -> HLA-DR",
    "transactivator_knockout_proof": "CIITA-null MV4-11 loses the induction",
    "pmid": "39704147",
    "title": "Bleximenib, the novel menin-KMT2A inhibitor JNJ-75276617, impairs long-term proliferation and immune evasion in acute myeloid leukemia.",
    "authors": "Hogeling SM, Lê DM, La Rose N, et al.",
    "venue": "Haematologica",
    "year": 2025,
    "title_provenance": "Title/authors/venue backfilled from PubMed esummary on 2026-09-20 because this citation stored a bare PMID with no title, which made it unscoreable by verify-citations.py. BACKFILLING IS NOT VERIFICATION: it confirms the PMID resolves to a real paper, not that this is the right paper for the claim. That still requires reading."
  }
}
relationship to the mhc ii headroom hypothesis
{
  "entry": "hyp-menin-inhibition-restores-gvl-only-where-mhc-ii-is-limiting",
  "beat": "Q53-transplant, same cycle",
  "concord": "both beats independently identify the same missing lineage-matched control; nothing here contradicts the prediction that class II headroom is small in B-precursor disease",
  "contest": "'constitutively expressed' is a gating statement, not a ceiling statement - MV4-11 is constitutively class II-positive and the drug still raised HLA-DR dose-dependently in it (PMID 39704147), so the premise does not establish the conclusion on its own; the discriminating measurement is a quantitative baseline against a saturating control",
  "does_not_transfer": "the argument is scoped to class II. Class I is expressed by essentially every nucleated cell, and the variable that matters there is polycomb restraint rather than on/off status. If class I headroom is absent BECAUSE class I is already high, the therapeutic consequence is the opposite of the class II case - something to lose rather than nothing to gain."
}
zhou locus set split
{
  "CIITA": "H2AK119ub solo (chr16:10877200) - polycomb-marked, menin-unbound",
  "NLRC5": "Menin solo (chr16:56989555) - menin-bound",
  "source_pmid": "39158067",
  "join": "analyses/zhou-locus-set-join/results.json",
  "supports": "the central claim that the class I and class II arms are different mechanisms, from an orthogonal data type",
  "inverts": "the assumed directionality - class II's transactivator is menin-free, class I's is menin-bound",
  "untested": "NLRC5 transcript under a menin inhibitor, which would be a direct route to class I absent from every published model",
  "system_caveat": "adult myeloid KMT2A-r AML; occupancy is not function; absence from the called set != unbound"
}
How this could bite us back
  • If the class I arm does not need interferon signalling and the class II arm does, then a single 'MHC-high' pharmacodynamic endpoint would look reassuring in a patient whose class I response has failed, and vice versa.
  • Combination partners chosen on the wrong mechanism will look inert for no visible reason: a JAK inhibitor, an anti-interferon agent, or an immunosuppressant would each remove one arm and leave the other, and a trial reading only total MHC would attribute the loss to the wrong thing.
  • A viral-mimicry framing invites combination with hypomethylating agents, which is a reasonable idea for the class II arm and has no evidential support for the class I arm.
  • All three mechanisms are reported by groups with an interest in the drug class; none has been replicated by an independent group in the same system, and the exclusions in particular are single-laboratory negatives.
Still open
  1. Does NLRC5 transcript move under a menin inhibitor? Zhou's locus set places the class I transactivator in the menin-bound group and the class II transactivator in the polycomb-marked menin-free group - the reverse of what this entry assumed - and the discriminating experiment is a single qPCR nobody has run.
  2. Are the three proposed routes three mechanisms, or one mechanism seen through three assays in three cell types? Nobody has run them against each other in one system.
  3. Does the class I arm survive a JAK inhibitor while the class II arm does not? That is the clinically consequential prediction of the STAT1-independence result and it is one flow experiment.
  4. Is HERV induction by a menin inhibitor accompanied by any class I change? Fetsch reports class II; whether class I moved in the same cells is not stated in the abstract and would decide whether the two arms are genuinely separate in AML.
  5. Is MHC class II headroom in B-precursor KMT2A::AFF1 a real ceiling or a gating artefact? The transplant beat's hypothesis turns on it, and the bleximenib data show a constitutively class II-positive myeloid cell can still be induced - so the measurement has to be quantitative against a saturating control, not a positive/negative call.
Every source behind this entry (3)
  1. E1strong confidence 4 of 5: Replicated in peer-reviewed work by more than one group.paper

    Neither depletion of STAT1, which is essential for interferon-induced MHC class I upregulation, nor depletion of MDA-5 (IFIH1), the key cytoplasmic double-stranded RNA receptor implicated in viral mimicry, impaired MHC class I gene activation following Menin targeting; RELA was not required for MHC-I gene activation following MEN1 targeting and there was no increase in TNF-alpha-induced p65 phosphorylation in Menin- or LEDGF-deficient cells; and Menin ChIP-seq detected ubiquitous Menin binding at active gene promoters but no significant Menin binding at bivalent MHC class I promoters, arguing against a direct repressive role for Menin at those genes.

    Targeting Menin disrupts the KMT2A/B and polycomb balance to paradoxically activate bivalent genes. · Sparbier CE, Gillespie A, Gomez J, Kumari N, Motazedian A, Chan KL, et al., Dawson MA · Nat Cell Biol 2023
    What this evidence cannot show

    All in K-562, a BCR::ABL1 CML line, by CRISPR depletion; these are necessity tests in one cellular context and do not show that interferon or NF-kB signalling is absent or unimportant, only that the menin-inhibitor effect does not depend on them there. No KMT2A-rearranged cell was tested and HERV transcripts were not measured in this paper, so the exclusion is of the SENSING arm of viral mimicry rather than of HERV induction itself. Read: full text via Europe PMC fullTextXML (PMC7614190), 2026-09-20.

    Evidence that argues against this (1)
    • A necessity test in a cell that may not induce HERVs strongly cannot exclude a viral-mimicry contribution in a cell that does. Fetsch measured HERV induction in KMT2A-rearranged and NPM1-mutated AML and Sparbier measured MDA-5 dependence in CML; the two are not the same experiment in the same cell, and a reader should not treat the exclusion as settled for AML. Fetsch V et al. 2026
  2. E2strong confidence 4 of 5: Replicated in peer-reviewed work by more than one group.paper

    The class II arm of menin-KMT2A inhibition is CIITA-dependent and MEIS1-independent: CIITA mRNA rose with a strong positive correlation to HLA-DR fold change, menin binding at the CIITA and HLA-DR loci was confirmed by ChIP-qPCR, HLA-DR induction was dose-dependent in scrambled MV4-11 and absent in CIITA-null MV4-11, and HLA-DR remained upregulated in MEIS1-overexpressing cells with no correlation between MEIS1 and HLA-DR fold changes.

    Bleximenib, the novel menin-KMT2A inhibitor JNJ-75276617, impairs long-term proliferation and immune evasion in acute myeloid leukemia. · Hogeling SM, Le DM, La Rose N, Kwon MC, Wierenga ATJ, Van den Heuvel FAJ, et al., Schuringa JJ · Haematologica 2025
    What this evidence cannot show

    A third proposed route, distinct from both the HERV-interferon route and the bivalency route, and the only one with a knockout proof. It is class II only and the knockout is in one cell line, MV4-11. The paper does not test whether the CIITA effect and the HERV effect are the same effect, nor whether its class I arm shares the CIITA mechanism - which it cannot, since CIITA does not drive class I. Read: full text (PMC12130779), 2026-09-20. CONTESTED SINCE WRITING: the menin-occupancy half of this claim is disputed. Zhou's genome-wide 9,292-locus menin/H2AK119ub target set places CIITA in the H2AK119ub-SOLO group - polycomb-marked and menin-unbound (PMID 39158067, join at analyses/zhou-locus-set-join). The CIITA-DEPENDENCE of the drug effect is not disputed; the DIRECTNESS is. See gene-ciita, which now carries status 'disputed' for this reason.

    Evidence that argues against this (2)
    • Menin occupancy at the CIITA locus and absence of menin occupancy at bivalent class I promoters are reported by different groups in different cells with different antibodies, so the contrast that makes the class I and class II arms look mechanistically distinct rests on a cross-study comparison, which is weaker than it reads. Sparbier CE et al. 2023
    • A genome-wide called set disagrees with this targeted ChIP-qPCR. In Zhou's 9,292-locus menin and H2AK119ub target set from KMT2A-rearranged AML, CIITA is H2AK119ub-solo at chr16:10877200 - polycomb-marked, menin-unbound - which is what an INDIRECT route to class II predicts and which agrees with the class I side of this entry, where menin is likewise not bound at the promoters it regulates. Neither result is obviously wrong: a thresholded caller can miss a modest peak and a targeted amplicon can report occupancy a caller would not credit, and the systems differ (OCI-AML2/KMT2A::AFDN-dominated versus MV4-11/KMT2A::AFF1). Bounded to adult myeloid AML; absence from Zhou's table is absence from the called set, not demonstrated absence of binding. Zhou X et al. 2024
  3. E3strong confidence 4 of 5: Replicated in peer-reviewed work by more than one group.paper

    Menin inhibition increased expression of multiple human endogenous retroviruses, leading to consecutive interferon-stimulated gene upregulation and enhanced MHC class II expression, in KMT2A-rearranged and NPM1-mutated AML in vitro and in vivo, with sensitisation to T-cell-mediated elimination after allogeneic transplant.

    Menin inhibition enhances graft-versus-leukemia effects by T-cell activation and endogenous retrovirus induction in AML. · Fetsch V, Schwobel L, Ozyerli-Goknar E, Stell AV, Punta M, Plenge T, et al., Zeiser R · Blood 2026
    What this evidence cannot show

    Class II only. This graph's entry for this paper is `cell-state-interferon-herv-mhc2` and is not duplicated here; it is cited to establish the route being contrasted. Murine and xenograft transplant models; read: abstract only.

    Evidence that argues against this (1)
    • Interferon-stimulated gene upregulation is also the regime in which low-dose interferon-gamma supports leukaemia-stem-cell self-renewal in MLL-AF9 models (PMID 37872214), so a chronic ISG-high state induced by a menin inhibitor is not unambiguously antileukaemic even where it raises antigen presentation. Xie X et al. 2023
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