Mount Sinai scientists have become the first to report a potentially serious side effect related to a new form of immunotherapy known as CAR-T cell therapy, which was recently approved for the treatment of multiple myeloma. Their findings were published as a case study in Nature Medicine in December.
CAR-T cell therapy uses genetically engineered immune system cells known as chimeric antigen receptor (CAR) T cells. In the specific version at issue, the CAR-T cells were used to target a protein known as B cell maturation antigen (BCMA). BCMA is commonly found in multiple myeloma, and this therapy has shown impressive response rates in people with particularly complex, treatment-resistant multiple myeloma.