Phase 1 Study of CART-ddBCMA, a CAR-T therapy utilizing a novel synthetic binding domain, for the treatment of subjects with relapsed and refractory multiple myeloma (https://www.myeloma.org/videos/phase-1-car-t-ddbcma-novel-synthetic-binding-domain-relapsed-refractory-multiple-myeloma)
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 (http://clinicaltrials.gov/show/NCT04155749)