Rare autoimmune disease halted for 15 years after stem-cell transplant

The findings, published in the journal Med, involve two people diagnosed with neuromyelitis optica spectrum disorder (NMOSD), a debilitating disease in which the immune system mistakenly attacks the spinal cord and the optic nerve connecting the eye to the brain.

NMOSD can cause recurring episodes of vision loss, eye pain, vomiting, weakness, and even paralysis. While existing therapies can reduce the risk of relapses, they often require lifelong treatment and were ineffective in the two patients involved in the study.

Researchers used an allogeneic hematopoietic stem-cell transplant, a procedure in which blood-forming stem cells are taken from a donor and transplanted into a patient. According to the study authors, this is the first time the method has been used to treat NMOSD.

The first patient, a man with severe disease, received donor stem cells from his sister in 2009. A woman with the same condition underwent the procedure the following year using stem cells from an unrelated donor. Both received a single infusion.

Before the transplant, doctors used chemotherapy drugs and antibody-based therapies to eliminate the malfunctioning immune cells responsible for the disease. Additional medication was given to reduce the risk of graft-versus-host disease, a potentially serious complication in which donor cells attack the recipient's tissues.

More than 15 years later, neither patient has experienced a return of disease-related antibodies or symptoms. The man's neurological condition improved enough for him to resume a normal life and start a family, while the woman gained better use of her arms and no longer needs medication to manage symptoms.

Scientists say the treatment effectively replaced the patients' immune systems with healthy donor-derived immune cells, potentially removing the source of the autoimmune attack altogether.

Despite the promising results, researchers caution that the procedure carries significant risks. Stem-cell transplants can lead to serious infections and other complications. The two patients also experienced some adverse effects after treatment, including swollen lymph nodes, antibody deficiency requiring medical care, and bladder cancer.

Because the study involved only two patients, experts say larger clinical trials will be needed to determine whether the approach can safely benefit more people with NMOSD. Researchers suggest the treatment should be considered primarily for younger patients whose disease does not respond to standard therapies or who have multiple autoimmune disorders.

Earlier, Qazinform News Agency reported that scientists launched the first cell rejuvenation trial.