drug_type
RELEVANT_DRUG
intervention_type
Cellular therapy (autologous CAR T cells)
drug_description
Autologous gene-modified T cells engineered to express a dual-target chimeric antigen receptor against CD19 and BCMA, intended to deplete CD19+ B cells and BCMA+ plasmablasts/plasma cells to suppress pathogenic autoantibody production in refractory immune thrombocytopenia.
nci_thesaurus_concept_id
C174412
nci_thesaurus_definition
A preparation of T-lymphocytes transduced with a lentiviral vector expressing a compound chimeric antigen receptor (cCAR) containing two distinct units of CARs, one specific for the tumor-associated antigen (TAA) B-cell maturation antigen (BCMA; tumor necrosis factor receptor superfamily member 17; TNFRSF17) and one specific for the TAA CD19, with potential immunomodulating and antineoplastic activities. Upon administration, the BCMA-CD19 cCAR T cells specifically and simultaneously target and bind to tumor cells expressing BCMA and/or CD19. This induces selective toxicity in tumor cells that express BCMA and/or CD19. BCMA, a tumor-specific antigen and a receptor for both a proliferation-inducing ligand (APRIL) and B-cell activating factor (BAFF), is a member of the tumor necrosis factor receptor superfamily (TNFRSF) and plays a key role in the survival of B-lymphocytes and plasma cells. BCMA is found on the surfaces of B-cells and is overexpressed on malignant plasma cells. CD19 is a B-cell-specific cell surface antigen overexpressed in B-cell lineage malignancies. Targeting two different antigens may improve coverage and protect against antigen escape and relapse as it is less likely for tumor cells to lose both antigens.
drug_mesh_term
Chimeric Antigen Receptor T-Cell Therapy
drug_category
CAR T
drug_class
Cellular Therapy
drug_delivery_route
Intravenous
drug_mechanism_of_action
Autologous T cells gene-modified to express dual chimeric antigen receptors targeting CD19 and BCMA. Upon binding CD19+ B cells and BCMA+ plasmablasts/plasma cells, CAR signaling activates T‑cell cytotoxicity and cytokine release, depleting B‑lineage cells and suppressing pathogenic autoantibody production; dual targeting reduces antigen escape.
drug_name
BCMA-CD19 cCAR T cells
nct_id_drug_ref
NCT06787989