Alicia Gerke, MD, UI assistant professor of internal medicine, has been awarded a two-year, $300,000 grant from the Food and Drug Administration (FDA) for a retrospective study in sarcoidosis, which is a multi-system inflammatory disease that causes significant illness, including organ dysfunction, and even death. The grant is one of six awarded by the FDA for natural history studies in rare diseases.
The aim of these studies is to better understand how specific rare diseases develop and progress over time, with the hope of eventually improving diagnostics and therapies for these conditions. This is the first time the FDA has provided funding through its Orphan Products Grants Program to conduct rare disease natural history studies.
Sarcoidosis affects fewer than 185,000 people nationally each year. Its natural history is unclear and there is no known curative treatment.
Under this grant, Gerke will establish a large, inclusive, retrospective cohort of sarcoidosis patients. By tracking these patients over time, she hopes to define the natural history of sarcoidosis, including health care usage and longitudinal analysis of their clinical outcomes. Separating the cohort of patient into worsening, chronic, or improving categories, should also provide important markers for predicting which patients will worsen and would, therefore, be most likely 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, lessen disease severity, and protect organ function.