Single CAR-T Treatment Shows Promise for Relieving Multiple Sclerosis Symptoms, Trial Finds
A single injection that genetically reprograms immune cells inside the body may offer a simpler approach to treating difficult autoimmune diseases, according to an early clinical trial.
Published in the New England Journal of Medicine, the study tested in vivo CAR T-cell therapy in 16 people with neurological autoimmune disorders, including multiple sclerosis and related neurological autoimmune conditions.
CAR T therapy has already been used against blood cancers. Traditionally, doctors collect T cells, modify them in a laboratory and return them to the body. This can take weeks, cost hundreds of thousands of dollars and require chemotherapy, increasing treatment burden and infection risk for people with chronic autoimmune disease.
The new approach aims to avoid this manufacturing process. Researchers led by Dai-Shi Tian gave patients a virus carrying genetic instructions that transformed T cells into CAR T cells inside the body. These cells targeted B cells that can produce harmful autoantibodies.
Patients were followed for six months. None developed severe nerve inflammation. Eleven had temporary increases in inflammatory molecules that settled after about two weeks.
CAR T cells persisted for months and were followed by a major reduction in disease-causing B cells. New B cells no longer appeared to produce harmful autoantibodies, suggesting an immune reset.
Patients with multiple sclerosis reported less fatigue and better movement and cognitive function, while nerve injury markers decreased. Other participants also showed improved muscle strength, inflammation and quality of life.
However, the findings remain preliminary. The study involved only 16 patients and had no control group. Because the treatment uses a virus that inserts genetic material into cells, longer follow-up is needed to assess potential risks, including cancer and infections. Larger controlled trials are needed to determine how effective and durable this treatment is.
REFERENCE: Yu-Hang Cheng, et al.; Lentiviral In Vivo CD19 CAR T-Cell Therapy in Neurologic Autoimmune Disorders; New England Journal of Medicine; 2026; doi:10.1056/NEJMc2603114
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