How a ‘living drug’ could treat autoimmune diseases

In lupus, a Type of autoimmune disease, the body’s natural defense system cannot distinguish between its own and foreign cells, so it mistakenly attacks its own tissues and organs. The attackers are molecules called autoantibodies that target the body instead of protecting it from invaders like normal antibodies do. They trigger a cascade of inflammation throughout the body, leading to joint and skin problems, pain, fatigue and even organ damage.

Now German researchers report that they have used the own cells of lupus patients to treat this disease. The sample size was small, but the results were striking: Five people who received an infusion of boosted immune cells are now in remission from severe lupus after receiving the experimental treatment. The results appeared in the Journal on September 15 naturopathy. “This is so close to a cure in my opinion,” says Hoang Nguyen, senior scientific program manager at the Lupus Research Alliance, who was not involved with the study. “They corrected the cells that produce antibodies against the body’s own tissues.”

The approach is known as CAR-T therapy and has been used successfully against some notoriously difficult-to-treat cancers. However, researchers have speculated about its potential to treat autoimmune diseases for several years. The therapy involves modifying a patient’s T cells, a key component of the immune system, and turning them into assassins to efficiently find a specific target in the body. In this case, the target is B cells — the immune cells that make antibodies in healthy people and self-attacking autoantibodies in people with lupus.

Last year, the German team showed that a woman went into remission from severe lupus after receiving CAR-T therapy. The new paper followed five other people who got the therapy.

To deliver the tailored treatment, doctors took T cells from patients and then genetically engineered them in the lab to recognize a protein called CD19. This protein appears on the surface of autoantibody-producing B cells. Scientists grew more of the modified T cells in the lab until they had enough for a therapeutic dose — around 50 to 100 million, depending on the patient’s weight. The modified T cells were then reinfused into the patients to seek out and destroy their defective B cells.

After about 100 days, the patients began to form new B cells – but these did not produce any harmful autoantibodies. In fact, the autoantibodies had completely disappeared. One of the people treated has been symptom-free for 17 months – the longest follow-up period to date. The others have been in remission for five to 12 months. All patients were able to stop the medications they were taking to treat their disease, including immunosuppressants.

Lupus is a lifelong disease that cannot be cured. According to the Lupus Foundation of America, it affects an estimated 1.5 million people in the United States and 5 million people worldwide, many of them young women. Most patients are treated with steroids to tame the inflammation. Immunosuppressive drugs are also used, but they make the body more susceptible to infections and often have unpleasant side effects. New antibody drugs designed to protect the body from attacking itself may help some patients, but not all.

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