A multi-institutional team of scientists, led by researchers at Weill Cornell Medicine’s Gale and Ira Drukier Institute for Children's Health, have received a five-year $8.297 grant to continue funding a Center for Lupus Research. The grant, awarded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases of the National Institutes of Health, will allow researchers to explore the underlying mechanisms of systemic lupus erythematosus (SLE) in children with the goal of better tailoring treatment.
“Pediatric lupus is often underrecognized, but up to a quarter of people with the illness have disease that starts in childhood,” said Dr. Virginia Pascual, program director of the Center for Lupus Research and the Drukier Director of the Drukier Institute for Children's Health at Weill Cornell Medicine.
SLE, a chronic immune disease, tends to be more aggressive in children than in adults. Symptoms can include joint pain, rash, fatigue, fever and sensitivity to light. “There is a tremendous need to understand the complexity of pediatric lupus,” said Dr. Pascual, who is also the Ronay Menschel Professor of Pediatrics at Weill Cornell Medicine.
Dr. Pascual and her colleagues received funding based on their prior research. They discovered that children with lupus have red blood cells that are a rich and unusual source of nucleic acids that activate other cells to cause inflammation. These cells, called macrophages, produce cytokines, or molecules that activate the immune system, creating an inflammatory response. The research team wants to better understand the underlying mechanisms of this inflammatory process.
They also want to determine why up to a third of children with lupus do not respond to standard of care, which consists of high doses of steroids and immunosuppressive medications.
“We want to apply all of the molecular techniques we have developed for studying this disease to understanding what’s going on in these patients at the time of diagnosis and through flares and remissions,” said Dr. Pascual.
The researchers will compare this data to information collected from children who do respond to treatment. The goal is to identify biomarkers of drug resistance and to develop new approaches to care.
Dr. Pascual’s research colleagues include Dr. Patrick Wilson, who was recruited as a professor of pediatrics and member of the Drukier Institute at Weill Cornell Medicine and a principal investigator at the Center for Lupus Research; Dr. Simone Caielli, assistant professor of immunology research in pediatrics and a junior investigator at the center. Other members include Dr. Duygu Ucar, associate professor at the Jackson Laboratory and the center’s administrative core associate director and co-investigator; Dr. Tracey Wright, chief of the Division of Pediatric Rheumatology at UT Southwestern Medical Center and principal investigator for the center’s clinical sample core; and several pediatric rheumatologists from Nationwide Children’s Hospital, who will contribute patients for the study.
“I am very fortunate to have received this award and to work with this amazing group of colleagues,” Dr. Pascual said. “Together, we have already generated some interesting data, and we hope to continue advancing the field.”