CART cell therapy has been a major breakthrough in treating B cell cancer, and now biotechnology companies are applying it to a variety of diseases, including systemic lupus erythematosus, diabetes and organ rejection. This paper will introduce the principles, applications and the future prospects of this innovative therapy.
What is a CART cell therapy? CAR T cell therapy is an immune cell-based therapy that is genetically engineered to recognize and attack specific cancer cells or abnormal B cells. At the heart of this therapy is to introduce a specific receptor called the chimeric antigen receptor (CAR) into the T cells of patients, allowing these T cells to target and eliminate cells carrying a specific antigen.
A revolutionary treatment case: a 20-year-old German female patient was diagnosed with systemic lupus erythematosus (SLE), a disease caused by an autoimmune response. Although she received a variety of treatments, including hydroxychloroquine, high-dose glucocorticoids, cyclophosphamide, and therapies for B cells (such as belimumab and rituximab), her condition remained uncontrolled. Her doctor, Georg Schett, a rheumatologist at Friedrich Alexandria University, decided to try a new approach: CART cell therapy. Surprisingly, the therapy worked very well for her. On day nine after treatment, CART cells accounted for more than one-fourth of her circulating T cells. Her B cells were completely cleared, and, after 5 weeks, double-stranded DNA autoantibody levels decreased to near zero. This dose of CART cells effectively reset her immune system back to an undiseased state. A few years later, she still had no recurrence and did not need to continue using the medication to control her condition.
Expanding application prospects: The success of CART cell therapy in the treatment of systemic lupus erythematosus has attracted wide attention in the scientific research community. Scientists have begun to explore the application of this therapy to other autoimmune diseases, such as anti-synthase syndrome, pemphigus, scleroderma, myasthenia gravis and rheumatoid arthritis. Results from preliminary trials show that many of these diseases can also benefit from CART cell therapy. Currently, biotechnology companies are also actively involved in the development of CART cell therapy. Philadelphia-based Cabaletta Bio introduced CABA-201, a CART cell therapy for CD19 with humanized CAR that reduces the risk of an immune response to treatment, thus potentially allowing the use of repeated doses. Kyverna Therapeutics Company has developed a humanized CD19 CAR T cell therapy for clinical trials of multiple autoimmune diseases.
Challenges and Future of CART cell therapy: Although CART cell therapy demonstrates great potential, it also faces some challenges. The side effects of treatment, such as cytokine release syndrome and neurological symptoms, are major problems to be overcome. In addition, the high cost of treatment is also a major obstacle to its widespread application. However, these issues are expected to be resolved gradually as technology advances and more clinical trials are conducted. In the future, CART cell therapy is expected to play a key role not only in cancer treatment, but also to revolutionize in the treatment of autoimmune diseases. We expect this therapy to bring hope and cure to more patients.
CAR receptors targeting the B cell antigen CD19 are engineered and expressed in T cells; these CAR T cells target and kill CD19-positive B cells.
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