Re-envisioning risk biomarkers in acute myeloid leukemia (AML) at single-cell level
The reMARK-AML project aims to refine prognostic risk parameters in NPM1-mutated acute myeloid leukemia by developing novel single-cell diagnostic assays for improved patient outcomes.
Projectdetails
Introduction
Acute myeloid leukemia (AML) is one of the most aggressive, clinically and biologically heterogeneous hematological malignancies. The different genetics classify disease entities and define the prognostic risk category to which AML patients are assigned and the treatment choices.
Disease Entities and Prognostic Factors
There is one entity, the AML with nucleophosmin (NPM1) gene mutations, that accounts for one-third of all adult AML (the most frequent). Another gene mutation - the Fms-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) - acts as the tipping point for patient risk definition. This mutation drastically worsens the prognosis and affects treatment in those 40% of patients where it is detected.
Research Focus
This has been a main field of research for Prof. Maria Paola Martelli and her team for years, specifically through her ERC consolidator grant ContraNPM1AML. Here, she will exploit concepts stemming from her ERC-funded studies on the key role of kinases in the NPM1-mutated AML development, response to therapy, and initiating relapse.
Patient Prognosis
The focus will be on the 60% of patients regarded as having a favorable prognosis, who nevertheless in 40% of cases will relapse with an extremely dismal outcome. Currently, successful strategies to identify novel frontline key markers to refine risk are not available.
Project Overview: reMARK-AML
reMARK-AML takes on the challenge of changing prognostic risk parameters, aiming to re-envision the marker at the single-cell level. A first strategy has been designed and will be tested for proof-of-concept in patients in a prospective non-interventional study.
Collaboration and Development
We have established contacts with industrial partners for the development of fit-for-purpose diagnostic assay prototypes to test in the study.
Future Challenges
The success of reMARK-AML will open the next challenge of developing marketable diagnostic kits and defining a strong business plan in order to measure the impact of our idea and proof-of-concept in the real-world clinical setting.
Financiële details & Tijdlijn
Financiële details
Subsidiebedrag | € 150.000 |
Totale projectbegroting | € 150.000 |
Tijdlijn
Startdatum | 1-8-2023 |
Einddatum | 31-1-2025 |
Subsidiejaar | 2023 |
Partners & Locaties
Projectpartners
- UNIVERSITA DEGLI STUDI DI PERUGIApenvoerder
Land(en)
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