A girl with autoimmune disease was treated with an experimental therapy

Affected by systemic lupus erythematosus as an adolescent, the young woman was treated with a next-generation therapeutic strategy, called CAR-T, a type of immunotherapy based on genetically modified T cells.

A state-of-the-art therapeutic strategy, called T-CAR and used up to now to fight some cancers, is gaining more and more space in the treatment of other pathologies. Researchers from the Erlangen University Hospital in Germany used it to treat a twenty year old girl with systemic lupus erythematosus, a potentially fatal autoimmune disease characterized by an alteration of the immune system that causes the formation of antibodies that target the genetic material contained in cells. This attack causes severe inflammation in various anatomical areas, which can manifest itself with rashes on the body, severe joint pain, various blood diseases, problems with the heart, lungs, kidneys and even the nervous system.

In the case of the young woman treated in Germany, Thu-Thao V, who had been struggling with the disease for four years, all drug treatments to control the condition, such as hydroxychloroquine, steroids and B-cell immunotherapies currently available, had proved ineffective. to convince doctors to try the CAR-T experimental route. “CAR-T stands for T cell therapy expressing a chimeric receptor for the antigen“Explained Professor Andreas Mackensen, Director of the fifth Department of Medicine, Hematology and Oncology of the Erlangen University Hospital who, together with his colleague, Professor Georg Schett, Director of the Third Department of Medicine, used CAR for the first time in the world. T against systemic lupus erythematosus.

The therapy, described in detail in a publication on New England Journal of Medicine, which falls within the so-called gene therapies, because it works by reprogramming the patient’s immune cells, it is based on specific immune cells (the T cells) that have been extracted from a sample of the girl’s blood and genetically modified, and then re-infused. The genetic modification involved the insertion of CAR genetic material, the chimeric antigen receptor which is an artificial receptor. “CAR – Mackensen added – recognizes special antigens on the surface of target cells and destroys them“. In the case of the young patient, the cells were reprogrammed to “render harmless” the B cells that produce the autoantibodies.

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The treatment was administered in March 2021. “We were very surprised at how quickly the patient’s condition improved immediately after the cell infusion – reported Professor Dimitrios Mougiakakos, senior physician at the 5th Department of Medicine -. CAR-T cells did their job excellently and quickly rendered disease-causing B cells harmless. All symptoms of systemic lupus erythematosus have disappeared along with the antibodies that were attacking the body”.

Almost six months after treatment, the young woman has completely stopped taking immunosuppressive drugs, including cortisone, and is currently symptom-free, with no signs of relapse. “I can finally breathe properly and sleep all night, I no longer have water retention due to kidney failure, the rash on my face has disappeared and my hair is growing much denser “ says the twenty-year-old. His heart function has also returned to normal, with his heart rate dropping from an average of 115 to 130 beats per minute to 80 beats per minute.

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