Scientists studying lupus or rheumatoid arthritis can obtain information on the individual cells of patients taking part in Phase 1 clinical trials, the National Institutes for Health has announced.
A key reason the NIH started the Accelerating Medicines Partnership for Rheumatoid Arthritis and Systemic Lupus Erythematosus initiative was to provide researchers with such information. The hope was that the data will lead to treatments for the two autoimmune diseases. Scientists can obtain the information at the Immunology Database and Analysis Portal.
Researchers used state-of-the-art technology to look at the individual kidney cells of lupus patients.
The rationale was that by focusing on single cells, researchers might be able to identify specific cell pathways’ contributions to the disease. The overarching goal was to give them more understanding of autoimmune disorders. An autoimmune disease is one in which the immune system attacks healthy tissue in addition to invaders.
“This pioneering program seeks to speed the development of new ways to combat a range of devastating diseases that affect millions of people,” Francis S. Collins, the director of the NIH, said in a news release. The initiative “is entering an exciting phase as experts around the world will begin to mine this invaluable biomedical resource in search of tomorrow’s cures.”
The Phase 1 trials began in March 2016 after the completion of Phase 0 studies. The objective of the Phase 0 studies was to look at different ways of obtaining and prepping tissue and to obtain a small number of samples from different sources.
There were three Phase 1 goals: comparing healthy and diseased cells, collecting a small number of similar cell samples, and determining that the results were good enough for the studies to enter the Phase 2 stage.
The individual-cell information may give scientists clues to future targets for lupus and rheumatoid arthritis treatments. The information could help them find genes, proteins, biological pathways and other factors that influence the two conditions.
Another possibility is that the information could be useful in precision medicine. This could evolve out of researchers identifying differences in pathways active in the tissue of different patients.
Researchers are planning Phase 2 trials that include more lupus and rheumatoid arthritis patients.
A long-term Phase 2 multi-center lupus study will involve patients with active kidney disease. Researchers will examine genes’ production of protein and cell properties over time, with a particular focus on disease subgroups. This will help them understand the forms of the disease.
“No single organization has the resources to take on the challenges facing the rheumatoid arthritis and lupus communities,” said Maria C. Freire, the president and executive director of the Foundation for the National Institutes of Health. The initiative “brings together government, pharmaceutical and not-for-profit expertise to collaboratively move therapies forward for these autoimmune diseases.”
The initiative is one of three projects the NIH started in 2014 to identify promising biological targets for therapies and to try to reduce the time and cost of treatment development.
The initiative is managed by the NIH’s National Institute of Arthritis and Musculoskeletal and Skin Diseases and National Institute of Allergy and Infectious Diseases (NIAID). The Foundation for the National Institutes of Health is in charge of working with partners in the effort, including the Lupus Foundation of America and Lupus Research Alliance.
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