Dr Farid on the Rationale for Evaluating MDC-CAR-BCMA001 in R/R Myeloma and AL Amyloidosis

"BCMA has emerged as a very attractive target for immunotherapies. CAR T-cell therapies are not always an option, especially for patients with AL amyloidosis, where [these agents] are not currently approved. To address this gap, we pursued the compassionate use of our in-house manufactured CAR T-cell construct."

Kiavasch Mohammad Nejad Farid, MD, Heidelberg University Hospital, discusses the rationale for evaluating the novel second-generation BCMA-directed CAR T-cell construct MDC-CAR-BCMA001 for patients with relapsed/refractory multiple myeloma and systemic light chain (AL) amyloidosis in the compassionate-use setting.

Although BCMA is a well-established immunotherapeutic target in multiple myeloma, CAR T-cell therapy remains unavailable for AL amyloidosis, where treatment options are particularly limited and disease biology poses unique challenges, Farid began. Furthermore, BCMA-directed CAR T-cell therapy is not feasible for certain patients, particularly those with relapsed/refractory multiple myeloma who previously progressed on a BCMA-targeted agent, he explained. To potentially address these challenges and unmet needs, investigators implemented compassionate use of the in-house–manufactured BCMA-targeted CAR T-cell construct MDC-CAR-BCMA001 in patients with BCMA-pretreated triple-class refractory multiple myeloma and AL amyloidosis, Farid detailed.

Findings from this study were presented at the 51st Annual EBMT Meeting and showed clinical activity with MDC-CAR-BCMA001 among heavily pretreated patients in the overall study population. Of the 6 patients who received MDC-CAR-BCMA001, 5 achieved a hematologic response. Four of these responders achieved a complete response (CR), and 1 patient experienced a very good partial response (VGPR). Moreover, all patients with CR achieved minimal residual disease negativity. The median decrease in free light chains was 98.4%, underscoring the depth of hematologic response. The median time to initial response was 14 days, and the median time to best response was 41 days. Organ responses were also observed in this cohort. One patient each achieved a cardiac VGPR, a renal VGPR, and improvement in polyneuropathy.

Disclosures: Farid reported receiving travel grants from Pierre Fabre and Kite/Gilead.