Novel Prognostic Biomarkers of Systemic Sclerosis to Aid Future Drug Development (PrognoSScis)

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Novel Prognostic Biomarkers of Systemic Sclerosis to Aid Future Drug Development (PrognoSScis)

Overview

Scleroderma is a chronic connective tissue disease generally classified as an autoimmune disease. This disease causes inflammation in the skin and other areas of the body. The inflammation associated with Scleroderma triggers the body’s own immune system to make too much collagen, leading to the hardening and tightening of the skin and connective tissues, including scar tissue (fibrosis). Scleroderma can also extend beyond the skin, and when this occurs it is called Systemic Sclerosis (SSc). For SSc patients the complications can result in damage to the heart, lungs, kidneys and the digestive system. As the tissues of these affected organs become hard and fibrous, they function less efficiently having a substantial impact on morbidity, mortality and the quality of life for patients.

This project aims to discover novel prognostic molecular biomarkers to provide information on the likely natural course of Systemic Sclerosis (SSc) in a patient on standard of care treatments and better inform on the rate of progression of organ-specific disease such as interstitial lung disease and skin fibrosis. By further discerning the heterogeneity among this patient population it will enable better patient stratification for clinical trials and the likelihood of response to targeted therapeutics, ultimately improving patient outcomes and opportunities for more tailored and effective treatment options.

Goals

Identify blood-based and tissue biomarkers in patients with early SSc involvement, with and without ILD, to predict outcomes and a reliable clinical course (i.e., skin/lung progression, PROs, and composite endpoints, etc.).
1. Establish a unique dataset of retrospective patient cohorts and data from industry and academia to support the appropriately powered studies in Aim 1 and Aim 2.
2. Perform multi-dimensional, molecular characterization of disease spectrum to better define and understand disease heterogeneity in diverse SSc and ILD clinical subtypes.
Validate prognostic biomarkers identified in Goal 1 that correlate with clinical subtypes, predict poor outcomes and trial endpoints, while accounting for background immunosuppressive therapy.

Partner With Us

The Foundation for the National Institutes of Health (FNIH) is actively seeking private-sector participation in this Project. A total of $3,713,184 in private funds over two and a half years is required to fully support the project. The below chart outlines the contributions necessary for participation as a full-funding partner. Financial support at other levels and in-kind contributions are also welcome.

Funding partners may have multiple participants on the Project Team, and full-funding partners may cast one vote on project decisions. Throughout the life of the project, the FNIH will work to ensure that all partners have ample opportunity to provide input and share valuable expertise and receive broad acknowledgment for being a scientific and funding partner. The FNIH aims to secure funding commitments in Q3/Q4 2023. This will enable private part¬ners’ active involvement in the immediate next steps of the process and ensure that project activities can begin in Q4 2023/Q1 2024.