ArticleThe Journal of clinical investigation2019
Mutated nucleophosmin 1 as immunotherapy target in acute myeloid leukemia.
Article in The Journal of clinical investigation, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06499025 (A Clinical Study to Evaluate the Safety and Initial Efficacy of Targeted AML1-ETO Neoantigen Cytotoxic T Cells), which is not on this map. Cited by 124 papers, 2 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Clinical Study to Evaluate the Safety and Initial Efficacy of Targeted AML1-ETO Neoantigen Cytotoxic T Cells (CTL) in the Treatment of Relapsed or Refractory t(8; 21) AML
Who cites it
124 citing papers in PubMed, 2 syntheses or guidelines pooled it, 228 citations in OpenAlex.
- Genome-wide association study identifies susceptibility loci for acute myeloid leukemia.Nature communications · 2021Pooled it
- Acute Myeloid Leukemia: From Biology to Clinical Practices Through Development and Pre-Clinical Therapeutics.Frontiers in oncology · 2020Pooled it
- Minor histocompatibility antigen TCR-T.Blood advances · 2026Review
- Sequence-Anchored Shared Tumor-Specific Epitopes for Pre-Manufactured HLA-Matched mRNA Cancer Vaccine Libraries: A Pan-Cancer Framework.Biomolecules · 2026Article
- mRNA vaccination targeting AML1::ETO fusion gene eliminates leukemia cells via activating T cells.Leukemia · 2026Article
- Sensitive detection of cancer antigens enabled by user-defined peptide libraries.Nature biotechnology · 2026Article
- Article
- Cellular immunotherapy for acute myeloid leukemia: from bench to bedside.Immunotherapy advances · 2026Review
- T-Cell Responses and T-Cell-Based Therapy in Acute Myeloid Leukemia.Current medical science · 2025Review
- Targeting the roots of myeloid malignancies with T cell receptors.Nature reviews. Cancer · 2025Review
- Identification of specific T-cell response and T-cell receptor targeting shared neoantigen for acute myeloid leukemia.Blood cancer journal · 2025Article
- SF3B1K700E Neoantigen Is a CD8+ T-cell Target Shared across Human Myeloid Neoplasms.Cancer immunology research · 2025Article
- Cell surface surprise: NPM1 is an immune therapy target for acute myeloid leukemia.HemaSphere · 2025Article
- Current developments in T-cell receptor therapy for acute myeloid leukemia.Blood advances · 2025Review
- Review
- Bleximenib, the novel menin-KMT2A inhibitor JNJ-75276617, impairs long-term proliferation and immune evasion in acute myeloid leukemia.Haematologica · 2025Article
- The interaction between common genetic mutations in AML and the immune landscape: mechanisms and implications for immune response.Frontiers in immunology · 2025Review
- Identification of shared neoantigens derived from frameshift mutations in theFrontiers in immunology · 2025Article
- MutatedFrontiers in immunology · 2025Article
- Protective effects for HLA-B*40:01 and C*03:04 in NPM1-mutated AML: result of a large HLA association study.Frontiers in immunology · 2025Article
64 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
16 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The most frequent subtype of acute myeloid leukemia (AML) is defined by mutations in the nucleophosmin 1 (NPM1) gene. Mutated NPM1 (ΔNPM1) is an attractive target for immunotherapy, since it is an essential driver gene and 4 bp frameshift insertions occur in the same hotspot in 30%-35% of AMLs, resulting in a C-terminal alternative reading frame of 11 aa. By searching the HLA class I ligandome of primary AMLs, we identified multiple ΔNPM1-derived peptides. For one of these peptides, HLA-A*02:01-binding CLAVEEVSL, we searched for specific T cells in healthy individuals using peptide-HLA tetramers. Tetramer-positive CD8+ T cells were isolated and analyzed for reactivity against primary AMLs. From one clone with superior antitumor reactivity, we isolated the T cell receptor (TCR) and demonstrated specific recognition and lysis of HLA-A*02:01-positive ΔNPM1 AML after retroviral transfer to CD8+ and CD4+ T cells. Antitumor efficacy of TCR-transduced T cells was confirmed in immunodeficient mice engrafted with a human AML cell line expressing ΔNPM1. In conclusion, the data show that ΔNPM1-derived peptides are presented on AML and that CLAVEEVSL is a neoantigen that can be efficiently targeted on AML by ΔNPM1 TCR gene transfer. Immunotherapy targeting ΔNPM1 may therefore contribute to treatment of AML.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.