Funds Major Study of Lupus Biomarkers to Predict Flares
The 2017 Lupus Insight Prize was awarded on June 15th to Virginia Pascual, MD for a project that shows great promise in understanding the events leading to lupus flares. Dr. Pascual was recently named Founding Director of the Gale and Ira Drukier Institute for Children’s Health at Weill Cornell Medicine in New York City.
The award was presented by George Tsokos, MD, Chief, Division of Rheumatology, Beth Israel Deaconess Medical Center at FOCIS 2017, the 17th Annual Meeting of the Federation of Clinical Immunology Societies (FOCIS). The $150,000 award recognizes a major, novel insight and/or discovery with the promise of changing thinking about lupus as well as a high likelihood of generating further advances in the diagnosis and treatment of the disease.
Dr. Pascual’s study will look at blood samples from children diagnosed with lupus over two years. By taking frequent samples before symptoms of a flare appear and using novel technologies that can dissect how genes are expressed in one cell at a time, Dr. Pascual aims to identify biomarkers of the earliest stages of disease activity with unprecedented depth. This research may uncover targets for the development of new drugs that can potentially block lupus flares before the disease damages vital organs. Starting with children whose lupus is not active when they enter the study will improve on earlier studies by eliminating the effects of treatments on biomarkers in the blood.
Kenneth M. Farber, Co-President and Co-CEO, Lupus Research Alliance noted, “Dr. Pascual’s 2017 Lupus Insight Prize builds on her transformative biomarker discoveries to identify the changes in blood cells that occur as lupus progresses from remission when symptoms are lessened to a disease flare when symptoms are worse.”
“Dr. Pascual was selected for the Insight Prize because of her scientific vision and dedication to improving the lives and health of all those living with or at risk of lupus,” commented Margaret Dowd, Co-President and Co-CEO. Her work is creating a paradigm shift in the lupus field by opening new avenues for personalized drug development and pointing the way to better, more effective clinical trial design.”
For more than 25 years, Dr. Pascual has focused on understanding the root causes of autoimmune diseases, including systemic lupus erythematosus, in children. Her groundbreaking studies of lupus biomarkers are generating new roadmaps that are transforming lupus research and paving the way for earlier diagnosis and personalized approaches to treatment of this complex disease.
Among her most important discoveries, in 2016, Dr. Pascual and her colleagues reported a landmark, four-year study of 158 children with lupus. Over the course of the study, they collected blood samples from these children every three months and looked at how blood cells changed in tandem with the onset and remission of lupus symptoms. The team identified many changes that could serve as biomarkers of lupus disease activity. In what is widely considered to be a major advance for the field, Dr. Pascual found that the children could be divided into seven distinct groups—each with its own pattern or roadmap of these newly discovered biomarkers.
This exciting observation addressed one of the biggest roadblocks in lupus research and patient care: the disease is highly variable. Individuals with lupus exhibit diverse symptoms; the disease progresses through cycles of remission and flaring at different rates; and the severity of the disease also varies from one person to the next. This high degree of variation is one reason for the failure of clinical trials of potential new lupus treatments—if only one in seven participants in a trial has a disease process that can respond to the treatment, then the trial is unlikely to show a successful outcome. Knowing this, Dr. Pascual and other lupus researchers can now design more personalized treatments and test them in clinical trials that are specifically targeted to patients with a biomarker roadmap that matches a particular treatment. For targeted treatments that are shown to be effective in a clinical trial, physicians could use biomarkers to identify which patients are most likely to respond and which are not—thus, improving patient care.
