Descrizione del progetto
Un solido esame clinico per la diagnosi di malattie autoimmuni
Il sistema immunitario è strettamente regolato attraverso una complessa interazione tra le cellule e altre proteine di segnalazione, come le chemochine. Queste ultime fungono da chemioattrattori, guidando le cellule immunitarie verso i siti di infezione o infiammazione. La chemochina CXCL4 è stata riconosciuta come un attore centrale nella patogenesi della sclerosi sistemica (SSc), nota anche come sclerodermia, una complessa malattia autoimmune caratterizzata da un’eccessiva cicatrizzazione dei tessuti in vari organi. Il progetto ASSYSt, finanziato dal Consiglio europeo della ricerca, mira a sviluppare un esame affidabile per misurare i livelli di CXCL4 per uso clinico. Il test consentirà di utilizzare la CXCL4 come biomarcatore per la diagnosi precoce della SSc, per prevedere la malattia e come bersaglio terapeutico per il trattamento o la prevenzione.
Obiettivo
Systemic Sclerosis (SSc, scleroderma) is a complex autoimmune disorder of unclear aetiology, culminating in excessive extracellular matrix deposition (fibrosis/tissue scarring) in skin and internal organs (e.g. joints, heart, lung, gastrointestinal tract and kidneys). More than half of the patients will ultimately die as a result of organ complications. The medical need is exacerbated by the lack of curative treatments and the lack of specific and sensitive molecular markers to facilitate reliable diagnosis and prognosis.
My team has recently demonstrated, through research funded by the ERC Starting Grant CIRCUMVENT, that the chemokine CXCL4 is a central player in the pathogenesis of SSc. Our results suggest that CXCL4 has great potential as 1) a biomarker to assist early diagnosis, 2) a biomarker to predict SSc disease course, and 3) a therapeutic target for the treatment or prevention of fibrosis in patients with SSc. Currently, a robust, accessible, reliable, cheap and fast assay to determine CXCL4 levels for clinical use is lacking. This prohibits the use of CXCL4 to predict clinical and treatment outcome and complicates the large-scale replication experiments across multiple labs and cohorts that are necessary to validate the diagnostic value of CXCL4.
Therefore, we argue that a reliable in vitro assay measuring circulating CXCL4 levels is highly needed and can be deployed as a research support tool to accelerate the implementation of clinical strategies based on CXCL4 and a clinical decision support tool in SSc to complement current diagnostic protocols. ASSYSt will focus on 1) the development of a reliable assay to adequately determine circulating CXCL4 levels irrespective of sample collection and storage conditions, 2) assessing the market potential of each of the applications listed above, and 3) defining a strong business strategy to guide route to market.
Parole chiave
Programma(i)
Argomento(i)
Meccanismo di finanziamento
ERC-POC - Proof of Concept GrantIstituzione ospitante
3584 CX Utrecht
Paesi Bassi