Research shows success of stem cell transplant in remission of rare autoimmune disease for 15 years
Two individuals diagnosed with neuromyelitis optica spectrum disorder (NMOSD), a serious and life-threatening autoimmune condition, achieved remission over a period of 15 years. The advance is attributed to an innovative treatment that involved stem cell transplantation. A recent study, published on June 15 in the scientific journal Med, points out that, after a single intervention, a man and a woman showed considerable improvements in their health and, currently, no longer need continuous medication to control the symptoms of the disease.
Nature published a statement detailing the pathology, in which the patients’ bodies produced antibodies that attacked the spinal cord and optic nerve, the connection between the eye and the brain. The clinical manifestations of neuromyelitis optica spectrum disorder (NMOSD) include eye pain, loss of vision, episodes of vomiting and weakness or paralysis that can affect arms and legs, highlighting the severity of the condition. This type of prolonged remission without the use of ongoing immunosuppressants represents a significant milestone, as management of autoimmune diseases often requires lifelong treatment.
The authors of the study underline the uniqueness of this result: “To date, no approved therapy allows patients to remain untreated, maintaining sustained control of the disease and the complete disappearance of pathogenic antibodies. Over more than 15 years of follow-up, both patients remained free of relapses without continuous immunosuppression, with an improvement in quality of life and permanent disappearance of the disease-causing antibodies”, the scientists state in their article.
Remarkable results after a single stem cell infusion
After the ineffectiveness of continuous drug treatments was confirmed, the two individuals underwent stem cell transplants. The objective of these cells was to restore the immune system’s defenses, completely replacing the patients’ immune system. Each received a single application of stem cells from their respective donors.
The researchers state that [the patients’] “new immune systems remained stable and displayed characteristics consistent with enhanced immune regulation.” They add that “these findings suggest that, in selected cases, immune system replacement may achieve long-term disease control and possibly cure,” as indicated in the study.
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Evaluating the positive points and adverse effects
Although promising, the methodology is still based on a small study sample, which indicates the need for more research involving patients with NMOSD to confirm both the safety and the identification of ideal candidates for this type of therapy. Study participants also experienced some side effects, including lymph node swelling, deficiency of certain antibodies, and one case of bladder cancer.
The occurrence of secondary cancers is not rare after stem cell transplants. However, the research authors reinforce that the application of treatment must take into account the risks involved and be considered only after the proven ineffectiveness of primary methods.
