Autologous Mesenchymal Stem Cells for the Treatment of Neuromyelitis Optica Spectrum Disorders

Autologous Mesenchymal Stem Cells for the Treatment of Progressive and Refractory Neuromyelitis Optica Spectrum Disorders: an Open-label Phase 2a Proof-of-concept Study

Neuromyelitis optica (NMO) is a demyelinating and degenerative disorder of the central nervous system affecting vision and brain and spinal cord function which leads to accumulating disability with a 5 year-mortality of approximately 30%. Survivors are typically left with severe morbidity secondary to blindness, quadriparesis and respiratory failure. No agent has been found to be highly effective in halting disease activity.Based on recent outcomes of Multipotent mesenchymal stromal cells in autoimmune diseases including multiple sclerosis, and based on the mechanisms of neuromyelitis optica, the investigators anticipate that mesenchymal stem cells transplantation may provide lasting disease stability for neuromyelitis optica patients.

Primary objective was to assess feasibility and safety; the investigators compared adverse events from up to 3months before treatment until up to 12 months after the infusion.

As a secondary objective, the investigators chose efficacy outcomes to assess the Expanded Disability Status (EDSS)、annual relapse rate (ARR) and time to next relapse after transplant.

Third objective anterior visual pathway and pyramidal tract as a model of wider disease. Masked endpoint analyses was used for electrophysiological and selected imaging outcomes.

Inclusion Criteria:

• Clinically definite neuromyelitis optica or neuromyelitis optica spectrum disorder

• Age > 18 year

• EDSS > 3

• Progression continued relapses or worsening MRI after at least a year of attempted therapy as evidenced by one or more of the following:

  • Increase of 1 EDSS point (if baseline EDSS<5.0) or 0.5 EDSS points (if baseline EDSS >5.5)
  • Moderate-severe relapses in past 18 months
  • Gadolinium enhancing lesions (double or triple dose Gd)
  • 1 new T2 lesion

• Evidence of recent inflammatory disease, as evidenced by any one of the following:

  • 1 moderate-severe relapses in past 18 months
  • 1 Gd-enhancing lesions (single, double or triple dose Gd)
  • 1 new T2 lesion

Exclusion Criteria:

• Received Immune inhibitors immunomodulator during the three months before the trial
• Significant cardiac, renal, or hepatic failure or any other disease that may affect the results of the study
• Allergies
• Pregnant or possibly pregnant
• Cognitive decline to understand or sign the informed consent
• Brain tumor, HIV (+) tumor marker (+), blood pressure (BP): 200 /110 mmHg
• Judged not suitable by doctors