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Autoimmune Diseases of Rheumatoid Arthritis and Lupus
National Institutes of Health (NIH), 10 biopharmaceutical companies, and multiple non-profit organizations launched an unprecedented new public-private partnership in February 2014. Managed through the Foundation for the NIH (FNIH), the Accelerating Medicines Partnership® (AMP®) program brings high-level government, industry, and non-profit foundation partners together to identify and validate the most promising biological targets for therapeutics. The partners designed a bold milestone-driven research plan to tackle the challenge for the autoimmune diseases of rheumatoid arthritis and systemic lupus erythematosus (lupus), as well as for Alzheimer’s disease and type 2 diabetes. One of the features that makes this public-private partnership unique is that AMP data is publicly accessible to the broad biomedical community for further research. The Phase This fact sheet addresses the AMP research plan for rheumatoid arthritis and lupus.
Rheumatoid arthritis (RA) and lupus are relatively common, severe disorders. About 1.5 million people or about 0.6 percent of the U.S. adult population have rheumatoid arthritis. Estimating how many people in the U.S. have lupus is difficult because symptoms vary widely and onset is often hard to pinpoint. Both conditions are autoimmune diseases that occur when the immune system mistakenly attacks parts of the body that it is designed to protect. They represent just two of a larger number of autoimmune disorders, including multiple sclerosis, Crohn’s disease, ulcerative colitis, type 1 diabetes, and psoriasis. These diseases share common flaws in immune function and regulation, leading to inflammation that destroys tissues. They can last a lifetime, cause severe disability, greatly affect quality of life, and are associated with increased risk of death.
Treatments for these diseases typically have been aimed at decreasing inflammation and pain by slowing down tissue damage and reducing complications. Most people with RA have only a partial response to available drugs, and many only respond to drugs for a limited period of time. In the case of lupus, no effective targeted therapies exist for the most severe forms of the disease. Research is needed to better understand the underlying disease process and identify parts of the immune system that aren't functioning correctly. This insight will help reveal the most promising new biological targets for drug development and match existing drugs to patients with specific molecular profiles who are most likely to benefit.
This project is analyzing, at the single cell level, tissue and blood samples from people with RA and lupus to pinpoint genes, proteins, biological pathways, and signaling networks. This type of analysis will allow insights into key aspects of diseases processes and allow comparisons between diseases. The project will explore differences between those RA patients who respond to therapies and those who do not, and will provide a better systems level understanding of disease mechanisms in both RA and lupus. This knowledge is essential for the development of targeted therapies and for the application of existing and future therapies to appropriate patient populations.
The partnership is using several new or developing technologies to analyze information from single cells and groups of cells involved in autoimmunity; collecting tissue samples, including synovium (the tissue that lines joints) from people with RA and kidney from people with lupus for molecular analysis; developing computational tools to integrate different data types to characterize molecular pathways; and making the data available to the broad research community for further analysis through dbGaP (rheumatoid arthritis; lupus) and ImmPort (accession: SDY997, SDY998). The first datasets were released in 2018.
Researchers mining the data can seek to identify treatment targets to develop medicines for diseases of interest. The data also has potential implications for precision medicine, as AMP RA/SLE researchers identify differences in the pathways active in the tissue of different patients.
The steering committee (SC) for RA and lupus is comprised of representatives from NIH, FNIH, and participating companies and non-profit organizations. The SC meets on a regular basis and is responsible for monitoring progress towards milestones. The SC operates under the direction of the overall AMP Executive Committee comprised of representatives from NIH, FNIH, participating companies, the Food and Drug Administration, and non-profit organizations.
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|Rheumatoid Arthritis and Lupus
ACCELERATING MEDICINES PARTNERSHIP and AMP are registered service marks of the U.S. Department of Health and Human Services.
This page last reviewed on July 29, 2021