Present and Future Therapies in Neuromyelitis Optica Spectrum Disorders

Abstract

Neuromyelitis optica spectrum disorders (NMOSD) are important evolving entities, which have reached much attention in the recent years. NMOSD are characterized by inflammatory lesions in the optic nerves, spinal cord, and central parts of the brain, as well as an autoimmune process directed against aquaporin-4. As disability in NMOSD accumulates by inflammatory damage from attacks, both the treatment and prevention of attacks are decisive for the long-term outcome. NMOSD attacks are treated with highdose intravenous corticosteroids and apheresis therapies, in particular therapeutic plasma exchange. In cases of incomplete remission, escalation of attack treatment is recommended. Preventive therapy is immunosuppressive and should by commenced as early as possible. Apart from classical immunosuppressants such as azathioprine and mycophenolate mofetil, repurposed biologicals are increasingly used. B-cell depletion with rituximab and other agents, inhibition of the interleukin-6 receptor with tocilizumab, and blockade of complement-mediated damage by eculizumab all are promising therapeutic strategies evaluated in randomized controlled trials. In this review, we will discuss present and future immunotherapies for NMOSD and also consider combination of treatments, plasma, cellular and other therapies. Current advances in immunopathological knowledge are translated into innovative concepts and begin a new era of NMOSD therapy.

Efficacy of glatiramer acetate in neuromyelitis optica spectrum disorder: a multicenter retrospective study

“…Although data in our nation-wide registry are collected in a highly standardized way within a study protocol and most other studies evaluating therapeutic efficacy in NMOSD were also done retrospectively [9,33], a prospective randomized controlled trial would be the optimal design to assess treatment efficacy. Second, our patients received GA therapy for a median of less than 1 year.…”

The clinical potential of Affibody-based inhibitors of C5 for therapeutic complement disruption

“…pharmacokinetics, administration, and distribution. For example in autoimmunity, neuromyelitis optica (NMO), Myasthenia gravis (MG), and ANCA associated vasculitis could benefit from either shorter periods of C5 (or alternatively C5a) blockade to induce remission or for chronic treatment [16][17][18]. Promising phase 2 data for these indications have been presented and registration studies for Soliris are ongoing in NMO and MG [18].…”

Neuromyelitis optica spectrum disorders: Emerging therapies

“…The oral corticosteroid, prednisolone, is chosen as a combination therapy with immunosuppressants or a regimen of tapering therapy after high‐dose corticosteroids after acute attacks. In Japan, oral corticosteroid monotherapy is preferentially used as an initial preventive treatment because of its faster onset compared with immunosuppressants . In cases of its inefficiency in preventing acute attacks or when severe adverse effects occur, immunosuppressants such as azathioprine, methotrexate, tacrolimus or cyclosporine A are recommended as add‐on or second‐line therapy.…”