B -Cell Maturation Antigen-CD19 Dual-Targeted Chimeric Antigen Receptor- T -Cell Therapy for Relapsed or Refractory...

Area of Science:

• Hematology
• Immunotherapy
• Oncology

Background:

• Systemic light chain (AL) amyloidosis is a rare plasma cell disorder.
• Treatment options for relapsed/refractory AL amyloidosis are limited.
• Chimeric antigen receptor (CAR)-T cell therapy is a potential novel treatment strategy.

Purpose of the Study:

• To investigate the efficacy and safety of BCMA-CD19 dual-targeted CAR-T cell therapy.
• To evaluate the treatment response in patients with refractory/relapsed AL amyloidosis.
• To assess the feasibility and tolerability of this novel therapeutic approach.

Main Methods:

• An exploratory, single-center trial was conducted.
• Six patients with refractory/relapsed AL amyloidosis received BCMA-CD19 dual-targeted CAR-T cells.
• Patients underwent preconditioning with fludarabine and cyclophosphamide.

Main Results:

• All 6 patients achieved hematologic complete response (100%) and renal response (100%).
• Median time to hematologic response was 9 days and renal response was 75 days.
• Grade 1 cytokine release syndrome occurred in 2 patients; no neurotoxicity was observed.

Conclusions:

• BCMA-CD19 dual-targeted CAR-T cell therapy is feasible and tolerable.
• The therapy demonstrated promising activity in patients with relapsed/refractory AL amyloidosis.
• Further investigation is warranted to confirm these findings.