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Two people were the first to receive therapy for a disease that damages the spinal cord and optic nerve.
A man and woman with a rare and devastating autoimmune disease are in remission for more than 15 years after they have received a stem cell transplant.
The positive results, last week in the magazine Withsuggest that the experimental treatment justifies a larger clinical trial, the scientists say.
Both people suffered from a serious illness and potentially fatal in which immune cells produce antibodies that trigger an attack to the spinal cord and the nerve that connects the eye to the brain, causing a disease known as neuromyelitis optica spectrum disease (NMOSD).
Symptoms tend to appear in attacks that last days or months and include eye pain, loss of vision, vomiting, and weakness or paralysis on the arms and legs. Current treatments can prevent these crises with continued medication, but they did not work in these two patients,
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After stem cell transplantation, the man’s neurological function improved, allowing her to resume a normal life and have two children.
The woman began to be able to use his arms more effectively than before treatment and no longer need medication to reduce symptoms.
“I don’t think we can say it’s a curebut, on the other hand, it solved the problem that the disease caused over a very long period”, explains the biomedical engineer Jiao Jiao Lifrom the University of Technology Sydney, Australia, who was not involved in the study.
In the treatment, called allogeneic hematopoietic stem cell transplantstem cells are collected from another person’s blood. The procedure has been used to treat some cancerssickle cell disease and other blood diseases.
Massimo Filippi, study co-author and neurologist at IRCCS San Raffaele Hospital in Milan, Italy, and his colleagues say this is the first use of this therapy in the treatment of NMOSD.
The man was the first to receive the allogeneic transplant, with his sister’s stem cells, in 2009. The following year, the woman received cells from an unrelated donor. The two participants received a single infusion of stem cells from the respective donors.
Keeping these people symptom-free for such a long period of time is exciting, says Bruce Milthorpecientista da University of Technology Sydney.
Reset the immune system
Before the transplant, participants received chemotherapy drugs called fludarabina e treossulfanoas well as a monoclonal antibody drug to eliminate the B cells of the immune system, responsible for producing antibodies that attack the spinal cord and optic nerve.
Before receiving stem cell transplants, patients also received a short course of antibodies and immunosuppressive drugs to stop the donor’s cells from attacking the recipient’s healthy cells — a complication known as dgraft-versus-host diseasecommon after stem cell transplants.
This complication can be fatalsays Li. Neither patient developed antibodies associated with NMOSD, and both developed healthy immune systems, the study authors report.
Li explains that the procedure completely replaces the immune system of the person. Other versions of the treatment, which use the patient’s own stem cells, restart the immune system.
However, adds the researcher, these versions may not work as well in people with autoimmune diseases if the B cells that produce the antibodies responsible for the attack are not completely eradicateds.
Milthorpe says it’s not clear whether a stem cell transplant would benefit all people with NMOSDdue to the small sample size of the study. Finding compatible donors can also be difficult. Still, he adds, the study can serve as a basis for starting a clinical trial.
