Trial Information

Cytokines as Biomarkers and Therapeutic Targets in Paraneoplastic Opsoclonus-Myoclonus Syndrome (OMS)

In this translational research, immunological mechanisms that underlie the assault of the
immune system on the brain in paraneoplastic opsoclonus-myoclonus syndrome (OMS) are under
evaluation. To test our principal hypothesis that there is an imbalance of pro-inflammatory
(Th1) and anti-inflammatory (Th2) cytokines in OMS, a comprehensive cytokine panel will be
measured by enzyme-linked immunoadsorbent assay (ELISA) and multiplexed fluorescent
bead-based immunoassay detection (LUMINEX 100 Lab MAP system)in blood and CSF of 400
children. To test the second hypothesis that cytokines could serve as biomarkers of disease
activity in OMS, cytokine concentrations will be correlated with clinical variables, such as
disease severity, OMS duration, prior relapses, and remissions, as well as immunological
variables, such as lymphocyte subset analysis. The cytokine 'biomarker profile' could aid
decision making for early intervention by identifying children at high risk for relapse and
poor outcome and allow targeting of the most implicated inflammatory cytokines by cytokine
therapies. To test our third hypothesis that lack of response to immunotherapy is due in
part to failure to increase the expression of anti-inflammatory Th2 cytokines, we will
determine the longitudinal effects of standard immunotherapy, such as steroids, ACTH, IVIg,
and chemotherapy, given in the course of clinical care on the cytokine profile. This
research could lead to the application of commercially-available cytokines and cytokine
blockers or to the development of new ones for OMS.