Effect: What are CAR T-cells and how do they work?

Author:  Julia Dobke, Editor:  Maria Yiallouros, English Translation:  Dr. med. Gesche Riabowol (geb. Tallen), Last modification: 2026/01/09 https://kinderkrebsinfo.de/doi/e217369

Chimeric antigen receptor T cells (CAR T cells) are genetically engineered cells of a patient [see genetic engineering‎]. They are produced from the patient's T lymphocytes‎, i.e. they stem from the body's own (autologous‎) cells. CAR T cells are used to recognise malignant cancer cells that would normally not be attacked by the original T lymphocytes.

T lymphocytes: tasks in the immune system

The immune system‎ consists of various components that collaborate to ensure an efficient immune response‎ to a pathogen. An important group of immune cells are the T lymphocytes‎ (also called T cells). T lymphocytes or T cells are formed in the bone marrow‎ and go through a maturation phase in a specific, relatively small organ in the chest area, the thymus gland‎. In this phase, the T lymphocytes learn to recognise pathogens or altered (mutated) endogenous cells in an individual’s body so that the entire immune system can get ready for an attack. This property of the T cells is used for the production of CAR T cells.

The surface of T lymphocytes presents with many identical structures (so-called receptor‎s). These receptors serve to recognise specific foreign proteins named antigen‎s, which can be found, for example, on the cell surface of a pathogen or on a body cell that is infected with a virus‎. Once the T lymphocyte has identified a cell carrying a cer-tain antigen, the matching T cell receptor will bind to it and the cell is subsequently destroyed.

Effect and production of CAR T cells

The novel cell therapy with CAR T cells makes use of the principle of action of T lymphocytes‎ described above: usually, leukaemia‎ (or other malignant) cells are not directly recognised as harmful by the patient's own immune defence‎s. The T lymphocytes must first be artificially enabled to recognise, for example, leukaemia cells as foreign in order to destroy them. Therefore, they are genetically modified in a special laboratory so that the receptor‎s on their cell surface are capable of identifying the proteins (antigens) on the cell surface of leukaemia cells as harmful in order to subsequently destroy them.

For genetic‎ modification, the T lymphocytes are filtered from the patient’s blood by a special procedure called leukapheresis‎. The T lymphocytes obtained in this way are sent to a special laboratory that has permission to produce CAR T cells. To do so, a new antigen receptor‎ with special properties is inserted into the patient’s T lymphocytes using a virus‎ for the transport (called viral vector). Since this antigen receptor consists of different building block-patterns that usually do not present this way, it is also called the chimeric antigen receptor (CAR).

For example, the artificial receptor can recognize the antigen‎ CD19 of B lymphocytes‎ (also B cells), which is found on the surface of many leukaemia‎ cells in acute lymphoblastic leukaemia‎ (as well as on certain lymphoma‎ cells). As a result, CAR T cells are able to destroy these B leukaemia (or, in the framework of studies, also B lymphoma) cells. (However, CAR-T cells have not yet been used in Europe to treat B-cell lymphomas in children and adolescents.)

Reinfusion of T lymphocytes as CAR T cells

The CAR T cells are reinfused into the patient by transfusion‎ via a central venous catheter‎. The artificial antigen receptor‎ of the T lymphocytes now recognises the patient’s leukaemia or lymphoma cells, which present the antigen CD19 on their surface, as harmful and destroys them.

The CAR T cells are also able to continue dividing in the patient's body and can remain active there for a longer period of time.