ASSYSt SIGNED

Project "ASSYSt" data sheet

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 Project objective

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.