Neuromyelitis optica spectrum disorder future or investigational therapies
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Associate Editor(s)-in-Chief: Julinka Auta Fernandes
Overview
Research on future and investigational therapies for neuromyelitis optica spectrum disorder (NMOSD) seeks to improve attack recovery, prevent relapses, and achieve more durable disease control. Important research directions include targeting antibody-producing cells, modifying antibody recycling, and immune reconstitution.[1]
For established attack treatment and preventive immunotherapy, see medical therapy.
Future or Investigational Therapies
Many neuromyelitis optica spectrum disorder studies focus on patients with immunoglobulin G (IgG) antibodies against aquaporin-4 (AQP4), abbreviated AQP4-IgG. Findings in this population should not automatically be generalized to antibody-negative disease.[1]
Chimeric Antigen Receptor T-Cell Therapy
Neuromyelitis optica spectrum disorder research includes chimeric antigen receptor T-cell (CAR-T) therapy directed against antibody-producing cells. CT103A targets B-cell maturation antigen (BCMA).[2]
- Published evidence: A 2023 open-label, single-arm phase 1 study treated 12 patients with relapsed or refractory AQP4-IgG-positive NMOSD. Eleven remained relapse-free during a median follow-up of 5.5 months.[2]
- Safety: Grade 1–2 cytokine release syndrome occurred in all participants. Infections and substantial hematologic toxicity were important adverse effects.[2]
- Limitations: The small sample, short follow-up, absence of a control group, and possible effects of lymphodepleting conditioning limit conclusions about efficacy and durability. The findings do not establish a cure.[2]
Antibody Recycling Inhibition
Neuromyelitis optica spectrum disorder acute-attack research includes efgartigimod, which targets the neonatal Fc receptor (FcRn). Here, Fc denotes the crystallizable fragment of an antibody. Receptor blockade reduces IgG recycling and circulating IgG concentrations.[3]
- NMOSD-specific evidence: A 2025 retrospective case-control study compared 13 patients receiving efgartigimod with 20 receiving intravenous immunoglobulin during acute attacks of AQP4-IgG-positive NMOSD. Clinical improvement and reduced IgG levels were reported. The nonrandomized design does not establish treatment equivalence.[4]
- Additional preliminary evidence: A 2026 study included 27 patients with NMOSD or myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD). Thirteen received efgartigimod plus intravenous methylprednisolone, and 14 received methylprednisolone alone. Greater short-term improvement was reported with add-on treatment, but the small, mixed population limits NMOSD-specific conclusions.[3]
Larger controlled studies are needed to define patient selection, comparative effectiveness, and longer-term outcomes.
B-Cell Survival Factor Inhibition
Neuromyelitis optica spectrum disorder research includes telitacicept, a fusion protein that neutralizes B-cell activating factor (BAFF) and a proliferation-inducing ligand (APRIL), two factors involved in B-cell and antibody-producing cell survival.[5]
A 2025 retrospective cohort compared 15 patients receiving telitacicept with 26 receiving mycophenolate mofetil. The authors reported favorable relapse and disability outcomes with telitacicept, but median follow-up was less than one year. The small, nonrandomized study does not establish superiority or long-term safety.[5]
Autologous Hematopoietic Stem Cell Transplantation
Neuromyelitis optica spectrum disorder has been investigated as an indication for autologous hematopoietic stem cell transplantation (AHSCT), which aims to reconstitute the immune system after conditioning treatment.[6]
The 2025 joint recommendations from the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) and the European Society for Blood and Marrow Transplantation (EBMT) state that evidence is insufficient to support transplantation for NMOSD outside clinical trials. Exceptional rescue use may be considered for treatment-refractory or aggressive disease after expert multidisciplinary assessment. Conditioning-related toxicity and infectious risks must be weighed against the availability of effective immunotherapies.[6]
Recent Development with Regional Approval
Obinutuzumab Beta
Neuromyelitis optica spectrum disorder treatment development includes obinutuzumab β (MIL62), a glycoengineered type II antibody targeting cluster of differentiation 20 (CD20).[7]
In the 2026 BeInmost randomized, double-blind phase 3 trial, relapse by week 52 occurred in 2 of 45 participants receiving obinutuzumab β (4.4%) and 21 of 46 receiving placebo (45.7%). The study supports efficacy against placebo; it does not establish superiority over other active therapies.[7]
Regulatory update: China's National Medical Products Administration (NMPA) records approval of obinutuzumab β on February 10, 2026. The manufacturer announced its approval for NMOSD.[8][9]
Research Priorities
Neuromyelitis optica spectrum disorder research priorities include determining the durability of remission, improving treatment sequencing and safety, understanding antibody-negative disease, and developing approaches that promote recovery and tissue repair. Controlled comparisons and longer follow-up are needed before early treatment signals can guide routine practice.[1]
References
- ↑ 1.0 1.1 1.2 Kümpfel T, Giglhuber K, Aktas O, et al. (2024). "Update on the diagnosis and treatment of neuromyelitis optica spectrum disorders (NMOSD) – revised recommendations of the Neuromyelitis Optica Study Group (NEMOS). Part II: Attack therapy and long-term management". Journal of Neurology. 271 (1): 141–176. doi:10.1007/s00415-023-11910-z. PMC 10770020 Check
|pmc=value (help). PMID 37676297 Check|pmid=value (help). - ↑ 2.0 2.1 2.2 2.3 Qin C, Tian DS, Zhou LQ, et al. (2023). "Anti-BCMA CAR T-cell therapy CT103A in relapsed or refractory AQP4-IgG seropositive neuromyelitis optica spectrum disorders: phase 1 trial interim results". Signal Transduction and Targeted Therapy. 8: 5. doi:10.1038/s41392-022-01278-3. PMC 9810610 Check
|pmc=value (help). - ↑ 3.0 3.1 Yin W, Wang S, Lu W, Duan T (2026). "Efficacy and safety of efgartigimod as an add-on therapy in patients with NMOSD and MOGAD at the acute attack phase". Frontiers in Immunology. 17: 1793153. doi:10.3389/fimmu.2026.1793153. PMC 13201218 Check
|pmc=value (help). PMID 42206038 Check|pmid=value (help). - ↑ Liu J, Li M, Wu H, et al. (2025). "Comparing the efficacy of efgartigimod and intravenous immunoglobulin on AQP4-IgG-positive neuromyelitis optica spectrum disorder during acute attacks". British Journal of Clinical Pharmacology. 91 (12): 3521–3528. doi:10.1002/bcp.70190. PMID 40841127 Check
|pmid=value (help). - ↑ 5.0 5.1 Zuo S, Yang W, Wang X, et al. (2025). "Efficacy and Safety of Telitacicept and Mycophenolate Mofetil in the Treatment of Neuromyelitis Optica Spectrum Disorders". Annals of Indian Academy of Neurology. 28 (5): 671–677. doi:10.4103/aian.aian_190_25. PMC 12610963 Check
|pmc=value (help). PMID 41081809 Check|pmid=value (help). - ↑ 6.0 6.1 Muraro PA, Mariottini A, Greco R, et al. (2025). "Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis and neuromyelitis optica spectrum disorder — recommendations from ECTRIMS and the EBMT". Nature Reviews Neurology. 21: 140–158. doi:10.1038/s41582-024-01050-x.
- ↑ 7.0 7.1 Wu L, Gu L, Fan W, et al. (2026). "Obinutuzumab β for aquaporin-4-positive neuromyelitis optica spectrum disorder: a phase 3 randomized controlled trial". Nature Medicine. 32: 3672–3681. doi:10.1038/s41591-026-04583-4. PMID 42575985 Check
|pmid=value (help). - ↑ National Medical Products Administration (2026-02-14). "2026年02月14日药品批准证明文件送达信息" [Drug approval certificate delivery information, February 14, 2026] (in 中文). Retrieved 2026-10-30.
- ↑ Beijing Mabworks Biotech Co., Ltd. (2026-02-14). "天广实MIL62（倍捷欣®）获批，改写视神经脊髓炎谱系疾病治疗格局" [Mabworks announces approval of MIL62 (Bejescin) for neuromyelitis optica spectrum disorder] (in 中文). Retrieved 2026-10-30.