Site Editors

Shaji K. Kumar, MD

Prashant Kapoor, MD, FACP


Novel CAR T-Cell Therapy for Resistant Myeloma in Early-Phase Study

By: Lauren Harrison, MD, MS
Posted: Thursday, July 21, 2022

A novel version of a chimeric antigen receptor (CAR) T-cell therapy has demonstrated clinical activity among patients with relapsed or refractory multiple myeloma. This CAR T-cell therapy uses a synthetic binding domain—named a D-domain (dd)—that targets B-cell maturation antigen (BCMA). Matthew J. Frigault, MD, of Massachusetts General Hospital, Boston, presented the early results of a phase I trial testing this investigational immunotherapy (CART-ddBCMA) at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting (Abstract 8003).

This study enrolled 25 patients with multiple myeloma who had undergone a median of five prior lines of therapy. Patients received 30 mg/m2 of fludarabine and 300 mg/m2 of cyclophosphamide for 3 days as lymphodepletion. CART-ddBCMA was administered at a dose of either 100 x 106 CAR-positive T cells or 300 x 106 CAR-positive T cells. Patients were monitored for adverse effects as well as response to therapy.

After a median follow-up of 9.8 months, all 25 patients were evaluable for safety, and 24 were evaluable for efficacy. The overall response rate was 100%, since 67% of patients achieved a complete response and 88% achieved a very good partial response or better. Conversion to a complete response did occur with longer follow-up, and some patients achieved it as late as 9 months after CAR T-cell infusion. A total of 20 patients were evaluable for measurable residual disease, and 85% have achieved a response of at least 10-5. Among the first six patients who were treated, four continue to be in a complete response 18 months after infusion. The progression-free survival and overall survival were not yet evaluable, because 79% of patients were in ongoing response at the time of data cutoff.

All enrolled patients experienced cytokine-release syndrome, but only one patient in the higher-dosage group had grade 3 cytokine-release syndrome. Four patients developed immune effector cell–associated neurotoxicity syndrome. Standard management led to resolution of these toxicities within 30 days.

Disclosure: For a full list of authors’ disclosures, visit

By continuing to browse this site you permit us and our partners to place identification cookies on your browser and agree to our use of cookies to identify you for marketing. Read our Privacy Policy to learn more.