CAR T-cell therapy
CAR T-cell therapy is a form of T-cell transfer therapy: the patient's own T cells are genetically modified in the laboratory to carry a chimeric antigen receptor (CAR) that recognizes a specific protein on cancer cells, then multiplied and returned by intravenous infusion. Several CAR-T products have FDA approval — all of them in blood cancers such as leukaemias, lymphomas and multiple myeloma. It is a one-time but demanding treatment: preparing the cells takes weeks, and serious side effects — above all cytokine release syndrome — can occur.
How it works
T cells are collected from the patient's blood, and in the laboratory a gene is added so that they produce a chimeric antigen receptor (CAR) on their surface. This receptor lets the T cells attach to a chosen protein on cancer cells — for example CD19 on leukaemia and lymphoma cells or BCMA on myeloma cells — and attack them. Growing enough cells takes about 2 to 8 weeks; in the meantime the patient usually receives chemotherapy that clears part of the existing immune cells, which helps the transferred CAR-T cells work more effectively. The modified cells are then given back through a vein.
Because millions of activated T cells can act at once, the therapy may trigger cytokine release syndrome — fever, low blood pressure and breathing problems caused by a surge of inflammatory signalling molecules; it is usually mild, but can be life-threatening, so treatment takes place in experienced centres. CAR-T cells can also occasionally recognize normal cells that carry the target protein. A related approach, TIL therapy, multiplies lymphocytes taken directly from the tumour instead of engineering them; the first TIL product, lifileucel, is approved for advanced melanoma.
Used in
Acute Myeloid Leukaemia (AML)
C92-C94
approved option in refractory or relapsed B-cell ALL in children and adults