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Improving therapy of NPM1-mutated AML

Total Cost €


EC-Contrib. €






 TREAT-NPM1-AML project word cloud

Explore the words cloud of the TREAT-NPM1-AML project. It provides you a very rough idea of what is the project "TREAT-NPM1-AML" about.

clinical    mutations    calls    action    model    promyelocytic    monitoring    leukemic    strategies    contributions    conduct    medical    minimal    seminal    fundamental    translation    models    disease    murine    drug    alone    unfit    vaccination    allogeneic    15    trial    003490    portion    forms    18    cell    chemotherapy    risk    feasibility    10    patients    similarly    itd    mice    2014    older    actinomycin    usually    adults    frequently    untreated    conventional    tools    mutated    mouse    protein    urgent    situ    compound    generate    npm1    residual    terminal    interacting    agents    gave    clinic    nucleophosmin    genetic    chemical    relapsed    understand    leukemia    acute    functions    flt3    therapy    age    mechanisms    anti    aml    discovered    dnmt3a    transplantation    year    pg02    previously    myeloid    therapeutic    50    demonstration    therapies    hematopoietic    cooperate    41    first    stratification    discovery    vitro    cured    molecular    classification    stem    lesion    eudract    interfering    approximately    accounting    combination    neoplasms   

Project "TREAT-NPM1-AML" data sheet

The following table provides information about the project.


Organization address
postcode: 6123

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country Italy [IT]
 Total cost 2˙895˙836 €
 EC max contribution 2˙895˙836 € (100%)
 Programme 1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
 Code Call ERC-2016-ADG
 Funding Scheme ERC-ADG
 Starting year 2017
 Duration (year-month-day) from 2017-11-01   to  2022-10-31


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    UNIVERSITA DEGLI STUDI DI PERUGIA IT (PERUGIA) coordinator 2˙895˙836.00


 Project objective

Acute myeloid leukemia (AML) is the most common acute leukemia in adults accounting for approximately 15,000 new cases/year in Europe and 20,000 new cases/year in US. Currently, 40-50% of AML patients (age 18-60 years) and only 5-10% of older patients (who are usually more frequently affected by the disease) can be cured using conventional chemotherapy /- allogeneic hematopoietic stem cell transplantation. Thus, AML still remains an urgent medical need which calls for new forms of molecular targeted therapies (similarly to those available for acute promyelocytic leukemia). The P.I. previously discovered the nucleophosmin (NPM1) mutations, the most common genetic lesion in AML (about one-third of cases) and gave fundamental contributions in the translation of this seminal discovery into the clinic (improved classification of myeloid neoplasms according to WHO, genetic-based risk- stratification of AML patients, monitoring of minimal residual disease and first demonstration of the anti-leukemic activity of actinomycin D). The present research proposal is focused on improving therapy of NPM1-mutated AML. Specifically, it is aimed to: i) identify novel chemical tools interfering with NPM1 functions by interacting with the N-terminal portion of the protein (objective 1); ii) conduct a clinical trial (AML-PG02; Eudract 2014-003490-41) with actinomycin D in older untreated and/or unfit patients with NPM1-mutated AML and to better understand in vitro and in mice models the mechanisms of action of this drug, used alone or in combination with other agents (objective 2); iii) develop compound mouse models aimed to investigate how NPM1 mutations cooperate with FLT3-ITD or DNMT3A mutations in promoting AML with the goal to better understand the characteristics of relapsed cases and to design new therapeutic strategies (objectives 3 and 4): and iv) generate a murine model for testing the feasibility of “in situ” vaccination in AML, especially in NPM1-mutated AML (objective 5).


year authors and title journal last update
List of publications.
2019 Paolo Sportoletti, Letizia Celani, Emanuela Varasano, Roberta Rossi, Daniele Sorcini, Chiara Rompietti, Francesca Strozzini, Beatrice Del Papa, Valerio Guarente, Giulio Spinozzi, Debora Cecchini, Oxana Bereshchenko, Torsten Haferlach, Maria Paola Martelli, Franca Falzetti, Brunangelo Falini
GATA1 epigenetic deregulation contributes to the development of AML with NPM1 and FLT3-ITD cooperating mutations
published pages: , ISSN: 0887-6924, DOI: 10.1038/s41375-019-0399-7
Leukemia 2019-11-27
2019 Brunangelo Falini, Orietta Spinelli, Manja Meggendorfer, Maria Paola Martelli, Barbara Bigerna, Stefano Ascani, Harald Stein, Alessandro Rambaldi, Torsten Haferlach
IDH1-R132 changes vary according to NPM1 and other mutations status in AML
published pages: 1043-1047, ISSN: 0887-6924, DOI: 10.1038/s41375-018-0299-2
Leukemia 33/4 2019-11-27
2018 Lorenzo Brunetti, Michael C. Gundry, Daniele Sorcini, Anna G. Guzman, Yung-Hsin Huang, Raghav Ramabadran, Ilaria Gionfriddo, Federica Mezzasoma, Francesca Milano, Behnam Nabet, Dennis L. Buckley, Steven M. Kornblau, Charles Y. Lin, Paolo Sportoletti, Maria Paola Martelli, Brunangelo Falini, Margaret A. Goodell
Mutant NPM1 Maintains the Leukemic State through HOX Expression
published pages: 499-512.e9, ISSN: 1535-6108, DOI: 10.1016/j.ccell.2018.08.005
Cancer Cell 34/3 2019-11-27
2018 Enrico Tiacci, Alessandra Venanzi, Stefano Ascani, Andrea Marra, Valeria Cardinali, Giovanni Martino, Veronica Codoni, Gianluca Schiavoni, Maria Paola Martelli, Brunangelo Falini
High-Risk Clonal Hematopoiesis as the Origin of AITL and NPM1 -Mutated AML
published pages: 981-984, ISSN: 0028-4793, DOI: 10.1056/nejmc1806413
New England Journal of Medicine 379/10 2019-11-27
2018 Xiaorong Gu, Quteba Ebrahem, Reda Z. Mahfouz, Metis Hasipek, Francis Enane, Tomas Radivoyevitch, Nicolas Rapin, Bartlomiej Przychodzen, Zhenbo Hu, Ramesh Balusu, Claudiu V. Cotta, David Wald, Christian Argueta, Yosef Landesman, Maria Paola Martelli, Brunangelo Falini, Hetty Carraway, Bo T. Porse, Jaroslaw Maciejewski, Babal K. Jha, Yogen Saunthararajah
Leukemogenic nucleophosmin mutation disrupts the transcription factor hub that regulates granulomonocytic fates
published pages: 4260-4279, ISSN: 0021-9738, DOI: 10.1172/jci97117
Journal of Clinical Investigation 128/10 2019-11-27

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