Another breakthrough in CAR-T cell therapy: The second autoimmune disease was successfully cured
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Another breakthrough in CAR-T cell therapy: The second autoimmune disease was successfully cured.
Last year, a 41-year-old man noticed health problems he initially thought were a viral infection.
But his health took a sharp turn for the worse, and almost suddenly he had trouble walking and standing. Further tests revealed that he had a severe autoimmune disease called Anti-synthetase Syndrome .
This is a serious autoimmune inflammatory muscle disease that can be fatal if diagnosed too late, or if the patient does not respond adequately to drugs that suppress the immune system.
The disease is caused by the erroneous attack of aminoacyl-tRNA synthetase , which is involved in the synthesis of amino acids in the body, by the immune system, thereby affecting the functions of various cells, involving muscles, joints, skin and lungs.
Unfortunately, all of the conventional treatments used to suppress his immune system failed to work for him. Fortunately, the CAR-T cell therapy originally used for cancer treatment saved him, and he fully recovered from the autoimmune disease 6 months after the treatment.
Recently, Professor Georg Schett , head of the Department of Rheumatology and Immunology at the University of Erlangen-Nuremberg in Germany, and others published a paper titled: CD19 -targeted CAR-T cells in refractory antisynthetase syndrome in The Lancet [1] ] , describing the treatment of this patient.
The research team successfully treated a male patient with a severe autoimmune disease , Anti- synthetase Syndrome , using CAR-T cell therapy . Six months after CAR-T cell therapy, the The patient fully recovered from the autoimmune disease without the need for immunosuppressive drugs.
This is the world’s first patient with anti-synthetase antibody syndrome successfully treated with CAR-T cell therapy , and also the second autoimmune disease cured by CAR-T after systemic lupus erythematosus .
In this patient, all conventional treatments to suppress his immune system failed.
So Professor Georg Schett decided to try CAR-T cell therapy . They have previously used CAR-T cell therapy to successfully treat multiple autoimmune disease patients with systemic lupus erythematosus .
CAR-T cell therapy , the full name of Chimeric Antigen Receptor T-Cell Immunotherapy (Chimeric Antigen Receptor T-Cell Immunotherapy) , first isolates and purifies T cells from the patient’s blood, and then undergoes genetic engineering to allow T cells to carry and recognize tumor cells The tumor chimeric antigen receptor (CAR) forms CAR-T cells, and these CAR-T cells are expanded and then infused back into the patient, thereby efficiently killing tumor cells.
At present, the FDA has approved 6 CAR-T cell therapies for marketing, which are used to treat blood cancers such as leukemia, lymphoma and multiple myeloma.
In this study, the research team developed anti-CD19 CAR-T cells. CD19 is a leukocyte differentiation antigen expressed by B cells. CD19 is involved in the proliferation, differentiation, activation and antibody production of B cells. The anti-CD19 CAR-T cells can eliminate antibody-producing B cells by targeting the CD19 protein, thereby inhibiting the anti-synthetase antibody in the patient.
After receiving CAR-T cell therapy, the patient’s health improved significantly, with inflammation in the muscles, lungs and joints almost completely disappearing, and muscle strength and endurance restored. It was particularly surprising that the disease did not recur even after the complete cessation of all immunosuppressive drugs. Six months after CAR-T cell therapy, the patient had fully recovered from the autoimmune disease.
This is also the second autoimmune disease successfully treated by Professor Georg Schett ‘s team using CAR-T cell therapy. Previously, they had successfully treated several patients with systemic lupus erythematosus using anti-CD19 CAR-T cell therapy.
On August 5, 2021 , the team of Professor Georg Schett published a paper titled: CD19-Targeted CAR T Cells in Refractory Systemic Lupus Erythematosus in the New England Journal of Medicine (NEJM) [ 2] .
The research team performed CAR-T cell therapy on a 20-year-old female patient with refractory systemic lupus erythematosus , and the therapy made her condition go into remission quickly without obvious side effects.
This patient is the first systemic lupus erythematosus patient to receive CAR-T cell therapy in the world. Although it was only one patient, the results of this trial showed that the CAR-T therapy responded quickly and the patient’s symptoms quickly subsided.
On September 15, 2022, Professor Georg Schett’s team published a research paper entitled: Anti-CD19 CAR T cell therapy for refractory systemic lupus erythematosus in the journal Nature Medicine .
The study reported that 5 patients with refractory systemic lupus erythematosus ( 4 females and 1 male, with an average age of 22 years ) were treated with CAR-T cells, and all patients improved. No relapse occurred, and all achieved drug-free remission.
Taken together, these successful cases demonstrate the feasibility of treating autoimmune diseases with CAR-T cell therapy.
Paper link :
1. https://doi.org/10.1016/S0140-6736(23)00023-5
2. https://www.nejm.org/doi/full/10.1056/NEJMc2107725
3. https://www.nature.com/articles/s41591-022-02017-5
Another breakthrough in CAR-T cell therapy: The second autoimmune disease was successfully cured
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