Alicia Gerke, MD, MBA, assistant professor of Pulmonary, Critical Care, and Occupational Medicine, has been awarded a two-year, $300,000 grant from the Food and Drug Administration. Gerke will continue her research into sarcoidosis, a multi-system inflammatory disease with significant morbidity, including organ dysfunction and even death. Though it is relatively rare–affecting fewer than 185,000 people nationally each year–its natural history is unclear and has no known curative treatment. Randomized clinical trials of drugs have not resulted in proven efficacy most likely in part because half of patients with sarcoidosis resolve naturally, and because the currently used primary outcome measures may not be the best indicator of drug efficacy.
Under this grant, Gerke intends to establish a large, inclusive, retrospective cohort of sarcoidosis patients and define the natural history of sarcoidosis in this population, including their health care use and a longitudinal analysis of their clinical outcomes. Being able to separate the cohort further—into worsening, chronic, or improving categories—should provide important markers for predicting earlier which patients will worsen and thus possess the capacity to show the largest effect of an intervention. Identifying the most appropriate candidates and sample sizes for clinical drug trials should produce more reliable results in future studies. This information should also modify the current treatment guidelines, and ultimately, lesson disease severity and protect organ function.