Acute myeloid leukemia

Subtype of Leukemias

Clinical / Scientific

AML is genetically diverse (FLT3, NPM1, IDH, splicing factors, TP53-complex karyotype). Differentiation block plus proliferative signalling and apoptotic resistance (BCL-2) dominate. IDH mutations generate 2-HG. Metabolic and marrow-niche ideas are adjunctive to, not replacements for, hematologic oncology.

Core Biological Drivers

FLT3 / signalling kinases

Proliferative mutations in a subset.

NPM1 / transcription-splicing

Common founder mutations.

IDH1/2

Oncometabolite 2-HG.

TP53 / complex karyotype

Adverse-risk biology.

Key Pathways

JAK/STAT

Scientific explanation

receptors signal through JAKs to STATs. in particular supports survival, invasion and inflammatory gene programmes in many solid and hematologic tumours.

PI3K/AKT

Scientific explanation

phosphorylates PIP2 to PIP3, recruiting . supports growth, survival, glucose uptake and mTORC1 input. Pathway activation is common via PIK3CA mutation, PTEN loss or -tyrosine- signalling.

MAPK/ERK

Scientific explanation

The RAS–RAF–MEK–ERK cascade transmits mitogenic RTK signals to programmes for proliferation and differentiation.

BCL-2 family

Scientific explanation

BCL-2, BCL-XL, MCL-1 and BAX/BAK control outer-membrane permeabilization, a core checkpoint frequently skewed toward survival in lymphoid and solid tumours.

Apoptosis

Scientific explanation

Intrinsic and extrinsic apoptotic programmes remove damaged cells. Evasion of is a hallmark, via BCL-2 family imbalance, death- decoys, or p53 loss.

MYC

Scientific explanation

MYC factors coordinate biomass accumulation, ribosome biogenesis, and glutamine use. Amplification or pathway activation is common.

Glycolysis

Scientific explanation

Aerobic (Warburg metabolism) supports ATP, biomass and redox buffering even when oxygen is available. Hexokinase, PKM2 and lactate export are frequent nodes.

Glutaminolysis

Scientific explanation

Glutamine supplies nitrogen and anaplerotic carbon via glutaminase and glutamate dehydrogenase, supporting nucleotide synthesis and TCA replenishment in MYC-driven and other tumours.

One-carbon / methionine metabolism

Scientific explanation

Folate-methionine cycles supply nucleotides and methylation. Some tumours show methionine dependence and PHGDH or SHMT rewiring.

Pathway Convergence

Target → pathway → downstream effect → biological consequence. Shared intersections are mechanistic maps, not protocols.

Growth-factor signalling

Ligand or mutation-driven RTK input feeds PI3K/AKT and mTORC1, supporting anabolic growth. This is a map of signalling, not a treatment protocol.

Receptor tyrosine kinase
↓
PI3K/AKT
↓
mTOR
↓
Protein synthesis / growth

Energy stress

Energetic stress activates AMPK, which can restrain mTORC1. Biguanides and related tools map onto this axis in models.

Complex I / ATP stress
↓
AMPK
↓
mTOR restraint
↓
Reduced anabolism

Mitochondrial stress

Electron-transport stress raises ROS; NRF2-driven transcription can buffer that stress and support survival. Antioxidant interventions are dual-edged.

Mitochondrial ROS
↓
NRF2 antioxidant programme
↓
Redox-buffered survival

Metabolic Vulnerabilities

Aerobic supports ATP, biomass and acidification even when oxygen is available. Extent varies by tumour and remains a vulnerability hypothesis rather than a uniform target.

Glutamine anaplerosis and nucleotide nitrogen demand are prominent in MYC-high and rapidly proliferating tumours. Dependence is heterogeneous.

IDH-mutant AML depends on 2-HG epigenetic rewiring.

Tumor Microenvironment

Endosteal and vascular marrow niches protect LSCs.

Metastasis Module

Leukemias and related neoplasms disseminate by trafficking rather than classical -driven carcinoma . Marrow, blood and lymphoid niches dominate.

Resistance Biology

FLT3 on-target mutations, BCL-2 upregulation, and LSC quiescence.

Cancer Stemness

Wnt, Notch, Hedgehog, ALDH and CD44-associated programmes can mark stem-like fractions with quiescence and therapy tolerance. These markers are not interchangeable across tumour types.

Mechanism-Based Adjunctive Strategies

Compounds appear only where a mechanistic overlap exists for this cancer. Evidence tiers are not equivalent. Nothing here is a treatment recommendation.

Metformin

Clinical / Human EvidenceIn VivoIn VitroMechanistically Plausible

Target / Mechanism

Modest complex I inhibition raises AMP:ATP, activating and restraining hepatic and -linked anabolism. Direct antineoplastic efficacy is not established from that pharmacology alone.

Cancer relevance

activation and restraint provide a metabolic rationale in - and -linked tumours. Human data are mixed and do not establish metformin as cancer therapy.

Metabolic adjunctive research context. Convergence: AMPK, mTOR, Glycolysis.

Disulfiram

In VitroIn VivoEarly Clinical

Target / Mechanism

ALDH ; copper-complexed forms can inhibit proteasome and NF-κB-related survival programmes in models. Clinical oncology evidence remains limited.

Cancer relevance

ALDH and copper-dependent proteasome/NF-κB stress in models; clinical oncology remains limited.

ALDH / redox experimental context. Convergence: Cancer stemness, NF-κB, Oxidative stress.

Mebendazole

In VitroIn VivoMechanistically Plausible

Target / Mechanism

Benzimidazole that binds β-. Mammalian disruption, mitotic arrest and related signalling in cancer models are preclinical and are not an approved anticancer use.

Cancer relevance

disruption can trigger mitotic stress and in cell and animal models. This is not an established oncology use.

Experimental antimitotic / microtubule stress. Convergence: Apoptosis, p53.

Curcumin

In VitroMechanistically Plausible

Target / Mechanism

Polyphenol with promiscuous in-vitro NF-κB, and ROS effects. Bioavailability is poor; dish activity does not establish clinical anticancer efficacy.

Cancer relevance

In-vitro NF-κB/ effects are frequent. Poor bioavailability and absence of robust clinical anticancer efficacy keep this pathway-level.

Inflammatory-signalling dish models. Convergence: NF-κB, JAK/STAT.

Berberine

In VitroIn VivoMechanistically Plausible

Target / Mechanism

Isoquinoline alkaloid that can inhibit complex I and activate in metabolic models, with additional -independent reports. Not an approved antineoplastic.

Cancer relevance

Complex I / pharmacology overlaps metformin-like energy stress in models. Bioavailability and lack of oncology indication keep this mechanistic.

Metabolic energy-stress hypothesis. Convergence: AMPK, mTOR, Mitochondrial oxidative phosphorylation.

Hydroxychloroquine

Early ClinicalIn VivoIn Vitro

Target / Mechanism

Lysosomotropic agent that raises endosomal/autophagosomal pH, impairing flux. Combination trials in oncology have been mixed; blockade is not equivalent to proven benefit.

Cancer relevance

Lysosomal pH elevation impairs flux. Early combination trials exist; benefit is not established and toxicity/retinal risk remain labelled concerns.

Autophagy-modulation research combinations. Convergence: Autophagy.

Research Context

  1. AML. Döhner H, Weisdorf DJ, Bloomfield CD. Acute Myeloid Leukemia. N Engl J Med. 2015;373(12):1136-1152. https://doi.org/10.1056/NEJMra1406184
  2. Hallmarks. Hanahan D, Weinberg RA. Hallmarks of cancer: the next generation. Cell. 2011;144(5):646-674. https://doi.org/10.1016/j.cell.2011.02.013
  3. Warburg. Vander Heiden MG, Cantley LC, Thompson CB. Understanding the Warburg effect: the metabolic requirements of cell proliferation. Science. 2009;324(5930):1029-1033. https://doi.org/10.1126/science.1160809

This oncology atlas is educational. Pathway maps, adjunctive strategies, and compound listings describe mechanistic relevance. They do not establish clinical efficacy, do not recommend treatment, and are not a substitute for oncology care. Evidence tiers are not equivalent.