Neuromyelitis optica (NMO) is an autoimmune disease that affects the central nervous system.
Patients have relapses (also known as attacks) which are often quite severe and leave them
with significant disability. Without treatment, within 5 years 50% of NMO patients are blind
in one or both eyes or require walking assistance (cane, walker or wheelchair).
NMO has only been relatively recently described and is fairly rare. Most NMO patients' immune
systems produce abnormal antibodies against aquaporin-4 (AQP4), which is found in certain
cells in the central nervous system. When these AQP4 antibodies bind to AQP4, they trigger a
cascade of events involving the immune system which eventually leads to damage to the nervous
system. This ultimately leads to disability, some of which is permanent.
Until now, treatments for NMO have been mostly focused on decreasing production of this AQP4
antibody. However, recent experiments in animal models of NMO have shown the importance of
what happens inside the central nervous system after the antibody binds to the nervous system
cell. Specifically, researchers have noted the importance of a specific cell type,
eosinophils, in causing damage in NMO lesions. In a recent study, researchers showed they
could prevent damage from NMO by blocking eosinophils using cetirizine, which is a popular
over-the-counter allergy medicine.
Cetirizine is already known to be safe and well-tolerated in the general population. In this
study, the researchers plan to add cetirizine on to patients' current NMO treatment. The
researchers aim to show that it is safe, well-tolerated, and that with cetirizine, NMO
patients have less relapses and therefore less disability over the course of the year
following initiation of treatment. The researchers also plan to study how cetirizine changes
the immunological profile in NMO patients by examining blood and cerebrospinal fluid.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Icahn School of Medicine at Mount Sinai
Collaborators:
Guthy Jackson Charitable Foundation Guthy Jackson Foundation