A British woman who had suffered from an autoimmune disease for more than 30 years was cured with a single gene therapy. Genetically modified T cells eliminated the immune cells that had been attacking her own body. Immune gene therapy long used for cancer is now expected to overcome autoimmune diseases as well.
University College London (UCL) Hospital said on the 12th (local time) that "in a phase 1 clinical trial of infusing CAR-T cells into patients with severe lupus, five patients aged 19 to 50 reached remission." Remission is a state in which symptoms or signs of a disease decrease or disappear completely so that daily life is not impaired, and it can be seen as meaning a cure.
Lupus is a chronic autoimmune disease in which the immune system attacks the body's own tissues. Lupus means wolf in Latin. The disease was so named because its hallmark skin rash looks as if it were a bite mark from a wolf. An estimated 5 million people worldwide have lupus, and it mainly develops in young women.
◇52-year-old lupus patient skis again after 30 years
The researchers administered Obcell (brand name Obe-cel), a CAR-T immune cancer drug from Autolus Therapeutics, to nine adults with severe lupus. All had received other treatments without effect, and most had lupus nephritis, a severe form of the disease that affects the kidneys. Five patients received a low-dose CAR-T therapy, and three received a high dose.
Lupus occurs when there is a problem with B cells, a type of immune cell. B cells produce antibodies and trigger immune responses that neutralize invaders, but when they attack normal cells instead of enemies, they cause autoimmune disease. Autolus's CAR-T therapy seeks out and destroys the problematic B cells.
After an average follow-up of 11 months, five patients in the low-dose group reached a cured state. The researchers noted that follow-up for the three high-dose patients has been only three months so far, but they believe these patients could also be cured.
Karl Peggs, director of the Biomedical Research Centre at UCL Hospital, said, "Although larger studies are needed, CAR-T cell therapy has shown the potential to reset the immune system and free patients from the vicious cycle of chronic autoimmune disease."
A representative case is Katie Tinkler, a 52-year-old mother of three. Tinkler had to quit her job as a fitness instructor due to pain and fatigue from severe lupus she had suffered since age 20. She could not walk properly and at times could not even lift a mug. The researchers said that after receiving CAR-T therapy, she skied again for the first time in 10 years and was able to dance at her daughter's wedding.
◇Immune reset raises hopes of conquering autoimmune diseases after cancer
CAR-T cells are T cells with a chimeric antigen receptor (CAR). Like the chimera in Greek mythology, a creature with features of multiple animals, this means genes that find antigen proteins on the surface of cancer cells are combined with T cells. The U.S. Food and Drug Administration (FDA) has approved seven CAR-T therapies, from Kymriah by Novartis in 2017 to Obe-cel by Autolus last year. All are approved to treat blood cancers in which immune cells that should protect the body have become cancerous.
Autolus designed Obe-cel to find and attack B cells that cause autoimmune disease instead of cancer cells. First, T cells were extracted from the patient's blood and engineered to detect the CD19 protein on the surface of B cells. These genetically modified T cells were then expanded in large numbers and reinfused into the patient.
After CAR-T treatment, B cells that cause disease nearly disappeared in patients. A few months later, B cells reappeared, but they were immature cells, not mature B cells that attack normal cells as before. The problematic B cells had vanished and normal B cells had begun to grow. The researchers explained that this meant CAR-T therapy had reset the immune system itself.
The medical community expects CAR-T therapy to revolutionize lupus treatment. A single infusion can be effective, meaning patients may no longer need to take drugs for life as they do now. CAR-T has been delivering results one after another in treating autoimmune diseases.
Researchers at University Hospital Erlangen of Friedrich-Alexander University in Germany said in April that a woman with three autoimmune diseases has been living a healthy, drug-free daily life for 14 months after a single CAR-T treatment. The team said this is the world's first record of resetting a patient's immune system in its entirety to achieve a state approaching cure, beyond improvement.
Curocell, a Korean biotech company, won approval in April from the Ministery of Food and Drug Safety for Rimcato, Korea's first CAR-T therapy, as a treatment for blood cancer. Since last year, it has also been conducting clinical trials in patients with lupus. Recently, the trial population was expanded from adults to children and adolescents. In April, the Ministry of Health and Welfare approved a clinical trial to administer CD19 CAR-T cells to pediatric and adolescent patients with refractory systemic lupus erythematosus (SLE).
References
Med (2026), DOI: https://doi.org/10.1016/j.medj.2026.101075