Efficacy and Safety of Bortezomib as add-on Treatment in Relapsing Neuromyelitis Optica Spectrum Disorder
Status:
Completed
Trial end date:
2017-01-31
Target enrollment:
Participant gender:
Summary
Neuromyelitis Optica Spectrum Disorders (NMOSD) is characterized by the pathogenic anti-AQP4
antibody, which can be produced by specific plasma cells. The patients who are not responsive
to rituximab treatment may be due to the presence of short-lived and long-lived plasma cells.
Previous studies confirmed that the proteasome inhibitor bortezomib (Velcade®, approved for
therapy of multiple myeloma) eliminated both plasmablasts and plasma cells by activation of
the terminal unfolded protein response. Treatment with bortezomib may help deplete plasma
cells producing auto-antibodies. Therefore, the rationale for using bortezomib in NMOSD is in
that bortezomib may help eliminate autoreactive plasma cells and reduce anti-AQP4 antibodies
titers. It is noted that bortezomib may protect astrocytes from NFκB-dependent inflammatory
damage in early events in NMOSD pathogenesis.
The purpose of this study is to determine if the drug bortezomib contributes to reduce the
average relapsing rates (ARRs) and alleviate neurological disability in NMOSD patients.