The Global Team Science Award ($3 million over three years) supports interdisciplinary, collaborative, and highly synergistic projects that push the boundaries of innovation and bridge research and clinical efforts in lupus.
2021 Award
Virginia Pascual, MD
Director of the Drukier Institute for Children’s Health
Ronay Menschel Professor of Pediatrics
Weill Cornell Medicine
https://drukierinstitute.weill.cornell.edu/faculty
The Global Team Science Award project led by Dr. Virginia Pascual aims to study childhood-onset systemic lupus erythematosus (cSLE) to understand not only lupus in children but the onset at any age for the following reasons: cSLE is very severe in the symptoms children present with as well as the consequences of lupus and its treatment; children with lupus often have a strong connection between their genetic makeup (DNA) and their disease; children also lack other illnesses that could complicate the understanding of research findings.
Dr. Pascual’s team of Lupus of Early Onset (LEO) investigators will perform a two-part project. The first project will track 75 pediatric SLE patients at multiple timepoints from when they first develop the disease and at various timepoints to record the type, features and activities of immune cells and antibodies present in their blood. Investigators will compare these results to data they already have on hand from other studies investigating lupus in humans and from preclinical models mimicking lupus disease in humans. Investigators will do this comparison to pinpoint biomarkers which may be used to separate patients into major disease groups within pediatric SLE. The second project will study the genetic makeup of more than 700 pediatric SLE patients and their family members to determine which alterations in the genetic makeup of children with SLE (relative to their family members) can serve as a predictor of major pediatric SLE disease groups identified in project 1. This will allow Dr. Pascual’s team to characterize, as never before, the full extent to which patterns in their DNA contribute to disease risk. Ultimately, this can help form the basis for personalized therapy for people with lupus of any age.
What this study means for people with lupus:
“Together, these studies represent the largest and most comprehensive investigation of childhood lupus to date.,” said Dr. Pascual. “Our findings will begin to penetrate the veil of complexity surrounding lupus, ultimately allowing us to understand which patients – children or adults – are likely [to] respond best to specific treatments.”
Ignacio Sanz, MD
Emory University
LRA Global Team Science Award recipient Dr. Ignacio Sanz and his team hope to define the changes in immune cells called B cells in lupus patients that correspond to patients’ clinical symptoms and disease outcomes. Lupus is most common in African American females. In SLE, these B cells make antibodies that attack the patient’s own organs and it hasn’t been clear why patients can have a wide range of symptoms with many different treatment outcomes. One reason seems to be because the B cells in each lupus patient can be different. Dr. Ignacio Sanz’s research has already identified many distinct B cell populations found in African American females with severe lupus. But more research is needed to understand the differences in B cells and how these differences contribute to the progression of lupus in patients. Dr. Sanz and his team hope to do this by pinpointing the contribution of specific B cell populations to the disease manifestation and progression in African American patients with lupus. Researchers will create “B cell profiles” which will group together distinct B cell types that can be associated with specific types of lupus, and thus help guide individualized patient treatment. This work will help us better understand the origin of the disease in specific patients, development of certain symptoms, and variability in responses to treatment. Ultimately, this research will help to develop more personalized therapies for lupus patients.
What this study means for people with lupus:
“This research will enable clinical investigators to more precisely design and evaluate clinical studies and standard of care treatments with the ultimate goal of implementing safer and more effective personalized treatments,” said Dr. Sanz.
