CAR T-cell therapy

Author:  Julia Dobke, Editor:  Maria Yiallouros, Reviewer:  Prof. Dr. med. Annette Künkele-Langer, Prof. Dr. med. Ursula Creutzig, English Translation:  Dr. med. Gesche Riabowol (geb. Tallen), Last modification: 2025/12/04 https://kinderkrebsinfo.de/doi/e217363

Chimeric Antigen Receptor T cell therapy (acronym: CAR T-cell therapy) is a new form of immunotherapy‎ for the treatment of cancer patients. It is based on the genetic‎ modification of a patient’s certain immune cells‎, the T lymphocytes‎. These are obtained from the patient, genetically engineered in the laboratory and then reinfused to the patient in their modified form as CAR T cells [see genetic engineering‎].

The T lymphocytes are provided with a new antigen receptor consisting of different building blocks that normally do not occur in this constellation. Due to this change, the new antigen receptor is called chimeric. This is where the term CAR stems from as an acronym for C=chimeric, A=antigen, R=receptor.

In 2018, the first two CAR T-cell therapies were approved in Europe for children and young adults with refractory‎ or relapsed ALL [see acute lymphoblastic leukaemia‎] and for adult patients with diffuse large B-cell lymphoma‎ (DLBCL) that wasn’t responding to conventional treatment. Additional CAR T-cell products for children and young adults are currently being tested in early clinical trials.

This form of therapy is expected to play an important role in the future, especially for children and adolescents with relapses (recurrence‎s) of acute lymphoblastic leukaemia. However, the use of CAR T cells is also being investigated in phase I/II clini-cal trials for other types of leukaemia (T-cell ALL, AML), B-cell and T-cell lymphomas, and solid tumors (e.g., medulloblastoma‎, neuroblastoma‎) [BUF2023‎] [CIC2024‎] [LOC2025‎] [RIC2018‎].

The following information on CAR T-cell therapy provides information on how this form of treatment works (particularly with regard to their use in acute lymphoblastic B-cell leukaemia), which patients may benefit from it, the treatment design, its risks and potential adverse events, as well as on aftercare and prognosis‎.

The information is not a substitute for the necessary educational discussions with the treatment team, but it can be used to prepare and better understand these discussions.