Grants Awarded

Date of Award
December 2016

Amount of Award
$50,000

Layperson Summary
The purpose of this research study is to understand more about how histiocytosis affects the brain. There are severe and advanced forms of histiocytic disease (Langerhans and others) in the brain that are life threatening and very difficult to treat. What causes these is not understood, and this is part of the reason that effective treatments are lacking. Also, our experience is that many patients, young and old, with histiocytosis have problems with thinking, memory, or walking, but scans may be unrevealing and no explanation can be found. We are trying to find a way to identify the mild and early forms of brain involvement of histiocytosis so that we can better understand how the disease works and how it may be treated. In a small number of adults with histiocytosis, we used advanced MRI technology to analyze the brains of patients with neurologic symptoms (trouble with thinking and memory) but whose scans were thought to be normal. What we found was that with these advanced methods, there is detectable brain shrinkage in these patients that cannot be seen with the human eye. This was a very important finding because it suggests that histiocytosis is affecting the brain in ways that we always suspected but until now had no way to detect or prove. For this study, we are going to perform this kind of MRI scanning (what is called “structural MRI”) and also “functional MRI” to investigate how the brain functions in patients with histiocytosis. We are also going to perform detailed testing of thinking and memory in order to characterize very carefully what kind of challenges patients face. With all of this information together, we can start to figure out what areas and functions of the brain are affected by histiocytosis and what kinds of specific deficits it causes. We believe that this study will create the roadmap that is needed for future research about how we can prevent and treat brain problems from histiocytosis.