Breakthrough in MS treatment: Cells produced inside the body help drive disease into remission

A single injection that prompts the body to produce its own therapeutic cells has shown early promise against multiple sclerosis and other severe autoimmune diseases, according to a study published in The New England Journal of Medicine.

The approach uses in vivo CAR-T treatment, unlike conventional ex vivo therapy, which removes blood cells from a patient, genetically modifies them in a laboratory and returns them to the body over a process that can take weeks. In the new method, a genetically modified, harmless lentivirus delivers instructions for producing chimeric antigen receptors in patients’ T cells.

The study was conducted by Huazhong University of Science and Technology and medical centers in China. It included 16 patients with multiple sclerosis or autoimmune conditions that cause muscle damage. The therapeutic cells multiplied inside the body and destroyed faulty B cells and autoantibodies that attack healthy tissue.

Researchers found that newly produced B cells no longer attacked healthy tissue after treatment, suggesting that the immune system had returned to what the article described as its “factory settings.” MS patients showed improvements in motor and cognitive functions, along with reduced chronic fatigue. Patients with muscle inflammation recorded stronger muscle scores and less inflammation.

Most patients experienced mild inflammatory reactions, which experts said could be controlled within two weeks. German and Chinese scientists nevertheless warned that the method needs careful assessment before wider clinical use. Genetic material delivered by the virus could mutate over the long term and potentially turn cells cancerous. Participants will therefore undergo close monitoring for at least 10 years for cancer, infections and possible disease relapse. Larger Phase II and III trials are planned before the treatment could become a routine therapy worldwide.