Descrizione del progetto
Il ruolo dello screening e del trattamento precoce nei tumori maligni del midollo osseo
Il mieloma multiplo (MM), una rara forma di cancro che costituisce l’1 % di tutti i tumori e determina il 2 % di tutti i decessi per cancro, è difficile da diagnosticare e impossibile da trattare. La stragrande maggioranza dei casi di MM viene diagnosticata quando è già allo stadio di malignità completa e quindi non beneficia dell’impatto di un trattamento precoce. Il progetto Edit-SMM, finanziato dall’UE, studierà i pazienti affetti da MM per realizzare nuove scoperte sulla biologia e sull’evoluzione di questa neoplasia del midollo osseo. Si baserà sul primo studio in assoluto che ha esaminato i campioni di sangue di 80 000 persone di età superiore ai 40 anni, sottoposte a screening per individuare i precursori della malattia. I risultati faranno luce sul ruolo dello screening e del trattamento precoce del MM, con la possibilità di trovare una cura.
Obiettivo
Multiple myeloma (MM) is a bone marrow malignancy that is always preceded by asymptomatic precursor conditions, monoclonal gammopathy of undetermined significance (MGUS) or smoldering MM (SMM). SMM is a more advanced precursor and is associated with a high risk of progression to MM. More than 94% of MM cases are diagnosed when already at the stage of full-blown malignancy and therefore do not benefit from the impact of early treatment.
Recent studies indicate a benefit of early treatment but all prior SMM studies are based on patients diagnosed due to other medical issues and are therefore biased. There are important and urgent questions on the role of screening and early treatment in SMM. There is also an unmet need to develop a risk score for progression that considers the biology and genetics of the disease.
In Edit-SMM we plan to build on the success from the Iceland Screens, Treats, or Prevents MM (iStopMM) study, where remarkably over 54% of the population (N=80,759) consented to a nationwide screening for MM precursors. It includes clinical evaluation and follow-up, biobanking, cross linking to central health data registries, repeated questionnaires on patient reported outcomes, groundbreaking methods of detection of SMM, and in-depth genetics.
Edit-SMM will identify all patients with SMM diagnosed in iStopMM. These will be followed and treated with the global objective of inducing a paradigm shift by improving diagnostics, disease classification, and population implementation of early intervention in MM by detecting and treating MM at a precursor state, hopefully leading to improved quality of life and potentially a cure. The main objectives are to gather knowledge on the epidemiological, clinical, and patient reported outcomes of SMM, perform a unique nationwide early treatment trial, and leverage this unparalleled collection of samples to make major new discoveries on the biology and evolution of SMM.
Campo scientifico
Parole chiave
Programma(i)
- HORIZON.1.1 - European Research Council (ERC) Main Programme
Argomento(i)
Meccanismo di finanziamento
HORIZON-ERC - HORIZON ERC GrantsIstituzione ospitante
101 Reykjavik
Islanda