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Dissecting to hit the therapeutic targets in nucleophosmin (NPM1)-mutated acute myeloid leukemia

Total Cost €


EC-Contrib. €






 ContraNPM1AML project word cloud

Explore the words cloud of the ContraNPM1AML project. It provides you a very rough idea of what is the project "ContraNPM1AML" about.

neoplasms    uncovered    nucleophosmin    throughput    drugs    classification    screening    soon    therapeutic    discovered    30    genetic    hypothesis    avail    relies    synthetic    succumb    chemical    landscape    hematologic    generation    intracellular    despite    disease    antracyclinecytarabine    leukemogenesis    aml    vulnerabilities    place    frequent    leukemia    analyzing    young    pharmacological    concurrent    cured    scheme    molecular    interference    difficult    myeloid    lethal    insights    context    whilst    discoveries    either    genome    genes    issue    revisited    screenings    mechanisms    therapy    recurrent    genomics    strategies    improvements    patients    heterogeneity    crispr    subtype    entity    mutations    classify    libraries    trials    adult    alteration    laboratories    npm1    gained    elderly    interactions    mutated    treat    complementary    group    models    advantage    missing    sequencing    drug    clinical    somatic    deciphering    50    malignancies    unravel    acute    network    hereto   

Project "ContraNPM1AML" 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 1˙883˙749 €
 EC max contribution 1˙883˙749 € (100%)
 Programme 1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
 Code Call ERC-2016-COG
 Funding Scheme ERC-COG
 Starting year 2017
 Duration (year-month-day) from 2017-04-01   to  2022-03-31


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    UNIVERSITA DEGLI STUDI DI PERUGIA IT (PERUGIA) coordinator 1˙883˙749.00


 Project objective

Acute myeloid leukemia (AML) is a group of hematologic malignancies which, due to their molecular and clinical heterogeneity, have been traditionally difficult to classify and treat. Recently, next-generation, whole-genome sequencing has uncovered several recurrent somatic mutations that better define the landscape of AML genomics. Despite these advances in deciphering AML molecular subsets, there have been no concurrent improvements in AML therapy which still relies on the ‘antracyclinecytarabine’ scheme. Hereto, only about 40-50% of adult young patients are cured whilst most of the elderly succumb to their disease. Therefore, new therapeutic approaches which would take advantage of the new discoveries are clearly needed. In the past years, we discovered and characterized nucleophosmin (NPM1) mutations as the most frequent genetic alteration (about 30%) in AML, and today NPM1-mutated AML is a new entity in the WHO classification of myeloid neoplasms. However, mechanisms of leukemogenesis and a specific therapy for this leukemia are missing. Here, I aim to unravel the complex network of molecular interactions that take place in this distinct genetic subtype, and find their vulnerabilities to identify new targets for therapy. To address this issue, I will avail of relevant pre-clinical models developed in our laboratories and propose two complementary strategies: 1) a screening-based approach, focused either on the target, by analyzing synthetic lethal interactions through CRISPR-based genome-wide interference, or on the drug, by high-throughput chemical libraries screenings; 2) a hypothesis-driven approach, based on our recent gained novel insights on the role of specific intracellular pathways/genes in NPM1-mutated AML and on pharmacological studies with ‘old’ drugs, which we have revisited in the specific AML genetic context. I expect our discoveries will lead to find novel therapeutic approaches and make clinical trials available to patients as soon as possible.


year authors and title journal last update
List of publications.
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 2019-11-26
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: 1827-1832, ISSN: 0887-6924, DOI: 10.1038/s41375-019-0399-7
Leukemia 33/7 2019-11-26

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