Evidence map›Paper›PMID 40269321›Full record

ArticleNature biotechnology2026

Treatment of acute myeloid leukemia models by targeting a cell surface RNA-binding protein.

Benson M George, Maria Eleftheriou, Eliza Yankova, Jonathan Perr, Peiyuan Chai, Gianluca Nestola, Karim Almahayni, Siân Evans, Aristi Damaskou, Helena Hemberger and 20 more

Abstract read
In one paragraph

Article in Nature biotechnology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.

0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

17 citing papers in PubMed.

  1. Article
  2. The cellular landscape of druggable RNA-binding proteins.Nature reviews. Drug discovery · 2026
    Review
  3. Pediatric AML CAR T cell therapy.Molecular therapy. Oncology · 2026
    Review
  4. Article
  5. Article
  6. Review
  7. Regulation of RNA-binding proteins by small biomolecules.Nature reviews. Molecular cell biology · 2026
    Review
  8. Article
  9. Review
  10. Review
  11. Review
  12. Review
  13. Article
  14. Review
  15. Review
  16. Article
  17. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

30 authors.

Benson M George *Stem Cell Program and Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA.
Maria Eleftheriou *Cambridge Stem Cell Institute, University of Cambridge, Cambridge, UK.
Eliza YankovaCambridge Stem Cell Institute, University of Cambridge, Cambridge, UK.ORCID http://orcid.org/0000-0003-2913-0012
Jonathan PerrStem Cell Program and Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA.
Peiyuan ChaiStem Cell Program and Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA.
Gianluca NestolaDepartment of Physics, Friedrich-Alexander University of Erlangen-Nuremberg, Erlangen, Germany.
Karim AlmahayniDepartment of Physics, Friedrich-Alexander University of Erlangen-Nuremberg, Erlangen, Germany.ORCID http://orcid.org/0000-0001-6844-4327
Siân EvansCambridge Stem Cell Institute, University of Cambridge, Cambridge, UK.
Aristi DamaskouCambridge Stem Cell Institute, University of Cambridge, Cambridge, UK.
Helena HembergerStem Cell Program and Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA.
Charlotta G LebedenkoStem Cell Program and Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA.
Justyna RakCambridge Stem Cell Institute, University of Cambridge, Cambridge, UK.
Qi YuStem Cell Program and Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA.
Ece BapcumStem Cell Program and Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA.
James RussellCambridge Stem Cell Institute, University of Cambridge, Cambridge, UK.
Jaana BagriCambridge Stem Cell Institute, University of Cambridge, Cambridge, UK.
Regan F VolkDepartment of Pharmaceutical Chemistry, Cardiovascular Research Institute, University of California, San Francisco, San Francisco, CA, USA.ORCID http://orcid.org/0000-0001-6748-719X
Malte SpiekermannMax Planck Institute for the Science of Light, Erlangen, Germany.
Richard M StoneDepartment of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA, USA.
George GiotopoulosCambridge Stem Cell Institute, University of Cambridge, Cambridge, UK.
Brian J P HuntlyCambridge Stem Cell Institute, University of Cambridge, Cambridge, UK.ORCID http://orcid.org/0000-0003-0312-161X
Joanna BaxterDepartment of Haematology, University of Cambridge, Cambridge, UK.
Fernando CamargoStem Cell Program and Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA.
Jie LiuInstitute for Stem Cell Biology and Regenerative Medicine, Stanford, CA, USA.
Balyn W ZaroDepartment of Pharmaceutical Chemistry, Cardiovascular Research Institute, University of California, San Francisco, San Francisco, CA, USA.ORCID http://orcid.org/0000-0002-8938-9889
George S VassiliouCambridge Stem Cell Institute, University of Cambridge, Cambridge, UK.ORCID http://orcid.org/0000-0003-4337-8022
Leonhard MöcklMax Planck Institute for the Science of Light, Erlangen, Germany.ORCID http://orcid.org/0000-0003-1387-886X
Jorge de la RosaCambridge Institute for Therapeutic Immunology and Infectious Disease, University of Cambridge, Cambridge, UK.
Ryan A FlynnStem Cell Program and Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA. ryan.flynn@childrens.harvard.edu.ORCID http://orcid.org/0000-0001-5013-0442
Konstantinos TzelepisCambridge Stem Cell Institute, University of Cambridge, Cambridge, UK. kt404@cam.ac.uk.ORCID http://orcid.org/0000-0002-4865-7648

Funding

Molecular regulation of native hematopoiesisR01HL128850 · NHLBI · BOSTON CHILDREN'S HOSPITAL · PI CAMARGO, FERNANDO · 2016 to 2024
$4.9M
Damon Runyon Cancer Research Foundation (Cancer Research Fund of the Damon Runyon-Walter Winchell Foundation) #74-23Medical Research Council MR/X008371/1NHLBI NIH HHS R01 HL128850Wellcome Trust 203151Wellcome Trust 218481Wellcome Trust 306752Wellcome Trust (Wellcome) RG106133Wellcome Trust (Wellcome) RG83195Wellcome Trust (Wellcome) RG94424
6 · The paper itself

Abstract

Immunotherapies for acute myeloid leukemia (AML) and other cancers are limited by a lack of tumor-specific targets. Here we discover that RNA-binding proteins and glycosylated RNAs (glycoRNAs) form precisely organized nanodomains on cancer cell surfaces. We characterize nucleophosmin (NPM1) as an abundant cell surface protein (csNPM1) on a variety of tumor types. With a focus on AML, we observe csNPM1 on blasts and leukemic stem cells but not on normal hematopoietic stem cells. We develop a monoclonal antibody to target csNPM1, which exhibits robust anti-tumor activity in multiple syngeneic and xenograft models of AML, including patient-derived xenografts, without observable toxicity. We find that csNPM1 is expressed in a mutation-agnostic manner on primary AML cells and may therefore offer a general strategy for detecting and treating AML. Surface profiling and in vivo work also demonstrate csNPM1 as a target on solid tumors. Our data suggest that csNPM1 and its neighboring glycoRNA-cell surface RNA-binding protein (csRBP) clusters may serve as an alternative antigen class for therapeutic targeting or cell identification.

Indexed as

Leukemia, Myeloid, AcuteNuclear ProteinsRNA-Binding ProteinsAnimalsAntibodies, MonoclonalCell Line, TumorHumansMiceNucleophosminXenograft Model Antitumor AssaysAntibodies, MonoclonalNPM1 protein, humanNpm1 protein, mouseNuclear ProteinsNucleophosminRNA-Binding Proteins

Identifiers

PMID40269321
PMCPMC7618518

What OpenQuestion holds

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Registered trials

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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.