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Five people are now in remission thanks to genetically modified immune cells

Five people have no symptoms of Lupus after receiving an experimental treatment that stopped the body attacking itself.

That treatment, called CAR-T cell therapy, seems to have reset the patients’ immune systems, Transmitting their autoimmune disease into remissionResearchers report September 15, 2005 Nature Medicine. It’s not yet clear how long the relief will last or whether the therapy will work for all patients.

Even so, the results could be “revolutionary,” says immunologist Linrong Lu of the Shanghai Immune Therapy Institute at the Shanghai Jiao Tong University School of Medicine, who was not involved in the study. CAR-T cell therapy has been used for various types of cancer, but it’s still in testing for autoimmune diseases (SN: 2/2/22).

All five participants experienced remission in the new study without the need for additional drugs other than the genetically engineered CAR–T cells. The target of those engineered cells — immune cells key for fighting off infections — returned a few months after being wiped out. Some of those cells are primed to attack viruses and bacteria but not the study participants’ healthy cells.

It’s unknown how many people worldwide have lupus, a painful disease in which some Antibodies, also known as antibodies, attack healthy tissue and organs. (SN: 4/25/19). A total of 161,000 to 322,000 Americans have systemic Lupus Erythematosus. While there are effective therapies, those treatments don’t work for everyone.

Five people were included in the study who had this common type of lupus with symptoms that are resistant to several commonly used lupus medications, including hydroxychloroquine. However, laboratory tests in mice suggested that CAR-T cell therapy might be able to help. Georg Schett, an immunologist, and his colleagues took T cells from each patient’s body and genetically modified them to kill all antibodies-producing cells. All five participants — four female and one male ages 18 to 24 — were in remission three months after being treated with the altered cells.

The CAR-T cell killed off the antibody-producing cells called B cells. However, B cells are important in fighting infectious diseases like measles. Luckily, the immune cells weren’t gone permanently, says Schett, of Friedrich-Alexander-Universität Erlangen-Nürnberg in Germany. A few months later, the patients’ bone marrow had made more. The patients’ bone marrow had made more, the B cells were back. But, the lupus wasn’t. 

“Which means, in a way, that we have a reset of the immune system in these young individuals,” Schett says.

The immune system typically has checkpoints which eliminate cells attacking the body rather than foreign invaders. Autoimmune diseases such as lupus occur when these cells that recognize and attack “self” escape scrutiny. Schett suggests that the same mistake might need to be made twice in order to have lupus back. “So far we think the disease is gone.”

It takes more time for the team to monitor the participants in order to know the truth. The researchers reported on August 2021 in the New England Journal of MedicineThe first participant to be treated, a 20 year-old woman, was In remission within three months of receiving the drug. Schett states that the patient is now in good health after being healthy for over a year. The remaining four patients were healthy for six months to one year. It will take time to see how long these people are lupus-free.

It is still not clear which people would benefit the most from CAR–T cell therapy. Individuals with Lupus may have different symptoms and severity. Lu states that patients in the early stages of the disease could be more likely to benefit from this treatment than those who have already become too severe. CAR-T cells therapy may still be an option for patients suffering from the disease, provided that future clinical trials are successful.

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