Dr. Matthew J. Frigault on a Phase 1 Study of CART-ddBCMA,

What is the background of this study? 

CART-ddBCMA is an autologous CAR-T cell therapy encoding a novel non-scFv synthetic binding domain targeting BCMA with a 4-1BB costimulatory motif and CD3-zeta T-cell activation domain. The novel binding domain is based on a computationally-derived triple-helix protein scaffold that is small (73 amino acids), stable, engineered to reduce immunogenicity, and can be modified to bind alternative target.  

In this video: 

Dr. Matthew J. Frigault (Massachusetts General Hospital, Boston, MA, USA) explains the methods and objective of this study. 

Conclusions:  

Early efficacy results are encouraging, with 9/9 (100%) overall response rate and manageable toxicities. Eight of nine responses are ongoing and responses continue to deepen. These data are encouraging in high-risk subjects with penta-refractory myeloma. Subjects continue to be enrolled and treated.  

Clinical trial information: NCT04155749