In-body CAR-T therapy eases multiple sclerosis in 16-patient trial

September 3, 2026 at 06:17 PM2 min

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Sixteen people with multiple sclerosis or other autoimmune conditions improved after receiving an in-body CAR-T therapy, according to a small clinical trial published in The New England Journal of Medicine. The treatment used a modified lentivirus to engineer T cells inside the body, reducing autoantibody-producing B cells. Researchers called the results a proof-of-concept but said larger trials are needed.

Key Facts

The trial involved 16 people with multiple sclerosis or other autoimmune conditions, including those causing muscle weakness or inflammation of the brain, spinal cord, and eyes.
Participants received a single injection of a lentivirus designed by Shenzhen Genocury Biotech and were monitored for about six months.
Following treatment, participants generated more CAR T cells over time, which depleted B cells and autoantibody levels.
Replacement B cells did not produce autoantibodies, suggesting the immune system had been reset.
David Simon of Charité — University Medicine Berlin called the study a very exciting proof-of-concept for in vivo CAR-T-cell therapy.

Trial Design

The trial, published this week in The New England Journal of Medicine, used a modified lentivirus to deliver genetic instructions for chimeric antigen receptors (CARs) into participants' T cells. The CAR T cells targeted autoantibodies expressed by B cells, which attack healthy tissue in autoimmune diseases. Participants received a single injection of the viral cells into their bloodstream and were monitored for about six months. The lentivirus was designed by biotechnology company Shenzhen Genocury Biotech in China.

Clinical Results

Following treatment, participants generated more CAR T cells over time, which helped deplete B cells and autoantibody levels. Replacement B cells did not produce such autoantibodies, suggesting the immune system had been reset. Dai-Shi Tian, a neurologist at Tongji Medical College at Huazhong University of Science and Technology in Wuhan, China, said the responses are promising signals but not yet definitive evidence of efficacy or permanent immune tolerance.

Expert Reaction

David Simon, a clinician-researcher at Charité — University Medicine Berlin, called the study a very exciting proof-of-concept for in vivo CAR-T-cell therapy. Simon noted that in vivo therapy is cheaper and faster to produce than conventional CAR-T-cell therapies made in a laboratory. Bing Du, an immunologist at East China Normal University in Shanghai, said developing an in vivo CAR-T treatment for any disease is a big achievement. Du added that the results are good and comparable with those for ex vivo CAR-T-cell therapy.