NCIt definition : A preparation consisting of autologous T-cells that are enriched to be primarily stem
memory T-cells (Tscm) and are transfected by electroporation with a proprietary transposon-based
DNA plasmid vector (PiggyBac) containing an undisclosed selection gene and encoding
both an unidentified human-derived safety switch and a chimeric antigen receptor (CAR)
based on a proprietary non-immunoglobulin scaffold molecule Centyrin (CARTyrin), which
specifically recognizes human B-cell maturation antigen (BCMA; tumor necrosis factor
receptor superfamily member 17; TNFRSF17), with potential immunostimulating and antineoplastic
activities. Upon administration, autologous anti-BCMA-CAR-expressing Tscm P-BCMA-101
specifically recognize and induce selective toxicity in BCMA-expressing tumor cells.
Use of CARTyrin may elicit less immunotoxicity than agents based on antibody-derived
single chain variable fragments (scFv), and this agent may exhibit increased persistence
and decreased exhaustion for the administered T-cells. If significant side effects
occur, the safety switch mechanism can induce the rapid attenuation or elimination
of P-BCMA-101. BCMA, a tumor-specific antigen and a member of the tumor necrosis factor
receptor superfamily (TNFRSF) that binds to both a proliferation-inducing ligand (APRIL;
TNFSF13) and B-cell activating factor (BAFF; TNFSF13B), plays a key role in plasma
cell survival. BCMA is found on the surfaces of plasma cells and is overexpressed
on malignant plasma cells.;