Findings Advance Development of New Treatments and Tools to Manage Lupus
(Washington, DC) Research findings from studies supported by the Lupus Foundation of America’s (LFA) National Research Program are providing new information to better understand the causes and progression of lupus, and contributing to development of new tools to treat and manage lupus, a misunderstood and unpredictable autoimmune disease. The findings were presented this week in Boston during the American College of Rheumatology (ACR) Annual Meeting.
The Foundation’s national research program continues to tackle the biggest challenges in lupus and address issues that have been neglected or received inadequate funding. The goals of the program are to improve quality of life, secure better outcomes, speed development of new treatments and get them into the hands of people who need them.
“Our national research is aimed at solving problems that have impeded lupus research for decades,” said Gary Gilkeson, Professor of Medicine/Microbiology and Immunology at the Medical University of South Carolina and Chair of the Lupus Foundation of America Medical-Scientific Advisory Council. “The studies we fund seek to answer some of the most difficult questions in lupus and it’s exciting to have their findings presented at this prestigious meeting of rheumatologists. It demonstrates the high quality of the work we are supporting through our national research program.”
LFA-REAL™ System is First-of-its-Kind Tool to Monitor Disease Activity
R. Paola Daly, MHS, Lupus Foundation of America Outcomes and Health Senior Manager, and Sarah Gilman, MPH, Publications and Education Content Manager, presented data associated with the development of a new patient reported outcome instrument that forms half of the Lupus Foundation of America’s Rapid Evaluation of Activity in Lupus System (LFA-REAL™), the first global and organ-specific disease assessment tool developed from both a physician and patient perspective to be used in clinical practice and clinical trials. As part of the patient instrument’s development process, the Foundation conducted in depth interviews and focus groups with lupus patients from around the nation. An analysis of these interviews confirmed that mental and emotional indicators of well-being are just as important to people with lupus as physical indicators. Watch a video about the LFA-REAL System.
Adult Stem Cell Research Advances toward Human Trials in Lupus
Research on stem cells holds promise to better understand how diseases occur, and discover new ways to treat lupus. Studies, primarily done in Asia, suggest that mesenchymal stem cells may be effective in treating humans with lupus. Findings from a Foundation-supported study of adult stem cells were presented by Dr. Erin Collins of the Medical University of South Carolina (MUSC). Investigators tested in the laboratory the abilities of mesenchymal stem cells (stem cells that can generate bone, cartilage, and fat cells and support the formation of blood and fibrous connective tissue) ,retrieved from umbilical cords as well as the bone marrow of people with lupus and healthy individuals, to determine which type of stem cells were most effective at modulating B cell functions. The results showed that adult stem cells obtained from umbilical cords suppressed lupus B cell proliferation, while the stem cells from bone marrow of people with lupus and healthy individuals did not suppress lupus B cell proliferation (the rapid and repeated production of new B cells). With this data, the researchers at MUSC were able to secure a planning grant from the National Institutes of Health to prepare for future human clinical trials.
Studies on Lupus in Children and Adolescents Provide Insight on Organ Involvement
Lupus in children and adolescents often requires more aggressive drug treatments than in adults, which comes with important ramifications for biological, psychological, and social development that have not been adequately studied. The Foundation’s Michael Jon Barlin Pediatric Lupus Research Program facilitates research on lupus in children and adolescents in areas where there is the greatest need, including lupus nephritis, neuropsychiatric lupus and health-related quality of life.
Dr. Laura Lewandowski, a pediatric rheumatology fellow at Duke University Medical Center, and the 2014 recipient of the Foundation’s Career Development Award, presented data from the largest study of a cohort (group) of children with lupus from South Africa. The investigators found that these children had higher levels of disease activity and progress to end stage organ damage at rates greater than members of pediatric lupus cohorts in developed nations.
Dr. Stacy P. Ardoin of the Ohio State University College of Medicine, who received a 2012 pediatric lupus research grant from the Foundation, was one of the presenters during the prestigious Edmond L. Dubois, MD Memorial Lecture. Findings from her research suggest that subclinical myocardial inflammation (undetected inflammation of the heart) may be common in lupus flares. This information may aid doctors in better managing cardiovascular disease complications, which are higher among people with lupus than in the general population.
Epigenetic Research Helps to Explain Gender Difference in Lupus
Lupus is more common in females than males, thus research on lupus in males has been limited. However, evolving evidence suggests that males with lupus may have a more sudden and severe onset of lupus with more serious complications. The Foundation’s national research program has supported the work of Dr. Bruce Richardson at the University of Michigan who is conducting epigenetic research (the study of changes to how genes function) to gain insight into the causes of lupus, including lupus in males.
Dr. Gabriela Gorelik presented the findings from Dr. Richardson’s lab, which provided additional insight into why lupus may be more common in females than males. Their findings confirmed that a specific enzyme, O-linked N-acetylglucosamine transferase (OGT), is found in higher amounts (over expressed) in females with lupus, altering T cell signaling and modifying gene expression, predisposing people to develop the disease. The findings may help identify new approaches that could be used to control the disease. The Lupus Foundation of America recently joined with other lupus groups to present Dr. Richardson with the Lupus Insight Prize, recognizing his lab’s important contributions to understanding the causes and progression of lupus.