According to a new study published in the prestigious scientific journal Med, neuroscientists and transplantologists have documented a complete, long-term cure in patients with Neuromyelitis Optica Spectrum Disorder (NMOSD). Neuromyelitis optica is a rare and aggressive autoimmune pathology in which the body’s immune system mistakenly attacks the optic nerves and the spinal cord, frequently resulting in blindness, loss of bladder control, and paralysis. In 60% to 98% of cases, the disease is characterized by permanent relapses, and until now, medicine lacked a therapy that would allow patients to discontinue medication and achieve the complete eradication of autoimmune antibodies.
The researchers summarized the outcomes of 15- and 16-year follow-ups of two patients who, following the failure of conventional treatments, underwent allogeneic hematopoietic stem cell transplantation (alloHCT) from healthy donors in 2009 and 2010. Prior to the procedure, the patients underwent aggressive chemotherapy conditioning (with fludarabine and treosulfan) and monoclonal antibody therapy (with rituximab) to completely destroy their old, defective immune cells that were producing the pathogenic AQP4-IgG antibodies.
Long-term monitoring, which included regular neurological evaluations and brain and spinal cord MRI scans, demonstrated that both patients have been completely free of disease symptoms and relapses for over 15 years, without any immunosuppressive treatment. Blood analyses confirmed that the disease-causing antibodies had entirely disappeared from the body, as the patients’ immune systems were completely replaced by the healthy cells of the donors.
Both patients report a significant improvement in their quality of life. One of them achieved a full recovery of physical capabilities and has since become a father of two. According to the scientists, this case provides solid scientific evidence (proof of concept) that stem cell transplantation can radically alter the course of autoimmune diseases, although large-scale studies are required to refine the method’s safety profile and selection criteria.
Note: Terminolgically, this pathology is known by several synonyms in medical circles. In Georgian clinical practice, it is often called opticoneuromyelitis or by its historical name — Devic’s disease. In the modern international classification, it is referred to as Neuromyelitis Optica Spectrum Disorder (NMOSD), as it has been proven that the autoimmune process is not confined to the optic nerve and spinal cord alone but encompasses a broader neurological spectrum.
