CAR-T cells have been used to treat several autoimmune diseases, starting with lupus: in many cases patients have gone into remission.
Remission is the most awaited word by patients suffering from autoimmune diseases, chronic pathologies due to an error in the functioning of the immune system. It means disappearance of symptoms and is found more and more often in the conclusions of scientific studies that have tested the effectiveness of CAR-T cell therapies against this class of diseases.
An informative article just published on Nature takes stock of the results of a dozen studies that in the last three years have studied the effects of this form of advanced therapy against debilitating autoimmune conditions such as lupus, ulcerative colitis, rheumatoid arthritis. The very positive results of these trials suggest that the future of therapies against these pathologies can rely heavily on CAR-Ts.
CAR-T: what are they
CAR-Ts (the acronym stands for Chimeric Antigens Receptor Cells-T) are T lymphocytes (white blood cells that normally recognize threats posed by viruses or malignant cells, maintaining the memory of the attacks suffered) engineered to recognize and target a specific cellular antigen, in this case the one expressed by patients’ B lymphocytes. In those affected by autoimmune diseases, B lymphocytes, which normally produce antibodies directed against viruses and bacteria, do not recognize the healthy part of the body and begin to attack it. Once reinfused into the patient, the CAR-Ts target each tissue and eradicate all the “crazy” B lymphocytes.
CAR-T therapies against autoimmune diseases
CAR-T cell therapies are typically used against several types of cancer. Their use against autoimmune diseases exploded in 2021, after the case of a twenty-year-old German woman who, thanks to CAR-Ts, ended up in persistent remission from a severe form of systemic lupus erymatosus, a rare autoimmune disease that can affect various organs and tissues, such as blood, skin, kidneys, lungs, nervous system. The patient had tried many therapies without success, including those with monoclonal antibodies. A single dose of CAR-T extinguished the symptoms of his disease in just a few weeks.
Since then, driven by this first positive result, several trials have been started to test CAR-Ts against other autoimmune conditions, such as rheumatoid arthritis (which affects the joints), systemic sclerosis (which attacks connective tissue, the one that supports and connects other tissues) and myositis, which affects the muscular system. More lupus trials have reached phase III, the most advanced.
In early 2025, a team of immunologists from East China Normal University in Shanghai published promising results from a pilot study that used immune cells from an external donor to treat a drug-resistant form of lupus in multiple patients.
An important step because it opens up the possibility of creating a generic version of CAR-Ts, to be produced in mass and not in a personalized form as is done now, starting from the individual patient’s cells.
The study involved four women, one of whom went into remission and no longer takes drugs, while the other three, freed from symptoms such as arthritis, swelling of blood vessels and alopecia, now follow maintenance therapy with low doses of steroids.
In 2024, the same group exploited a CAR-T therapy obtained from donor cells modified with CRISPR to treat three patients, one with a severe form of myositis and two with systemic sclerosis. The patients went into remission.
Convincing studies on the use of CAR-Ts are also underway against myasthenia gravis, an autoimmune disease that causes muscle weakness in the muscles needed for breathing or swallowing and therefore leads to disabling symptoms, and against ulcerative colitis, a chronic inflammatory condition of the colon that generates ulcers, diarrhea with blood loss and intestinal malabsorption.
Although it is not clear whether CAR-T therapies represent a permanent cure against autoimmune diseases, many are betting that in the coming years they will become a much more widespread treatment, at least against lupus, the autoimmune disease for which they are currently most studied.
CAR-Ts still involve very high costs, but on the other side of the scale we must put, in addition to the improvement in the patients’ quality of life, economic benefits: many of the immune diseases mentioned generate expensive costs in terms of healthcare, pharmacological treatments and days of work lost, costs saved when patients go into remission.
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