Hospital Clínic-IDIBAPS' CAR T-Cell Therapy for Multiple Myeloma Demonstrates Efficacy in Resistant Cases

The research, unveiled in The Lancet Oncology, indicates that every patient who received this specific CAR T-cell treatment, formulated at the institution, exhibited a positive reaction. Nearly 70% of these reactions were full after a period of 18 months, while the remainder were partial. This study received backing from the Carlos III Health Institute and the “la Caixa” Foundation.

Dr. Carlos Fernández de Larrea, a hematologist at the Hospital Clínic and leader of the IDIBAPS Myeloma and related conditions research team, spearheaded the study.

The inception of CAR T-cell therapy for multiple myeloma at the Hospital Clínic greatly benefited from the unwavering support of the "la Caixa" Foundation. This was due to a strategic partnership to bolster groundbreaking cancer research with significant impact and to enhance the quality of care for cancer patients. The Carlos III Health Institute (ISCIII) and the Bosch Aymerich Foundation also provided specific funds for this clinical trial.

The study was a collaborative effort with the Clínica Universidad de Navarra and three other Spanish medical institutions.

EUROPE'S PIONEERING CAR T-CELL TREATMENT FOR MULTIPLE MYELOMA

CAR T-Cell therapy is emerging as a potent solution for treating stubborn multiple myeloma. This cell-based gene therapy enables patients to be their own donors by modifying their T cells to target cancer cells.

The ARI0002h CAR T-cell therapy specifically targets the BCMA antigen present on myeloma cancer cells. Preliminary studies revealed its high specificity and potency against malignant car t cells multiple myeloma cells.

This marks the second CAR T-cell treatment formulated at the Hospital Clínic-IDIBAPS. Dr. Manel Juan, who heads the Immunology Service at the Hospital Clínic, highlighted the humanized nature of this therapy, which differentiates it from previous therapies.

ADJUSTING THE TREATMENT DOSE TO MINIMIZE ADVERSE EFFECTS

Multiple myeloma constitutes about 10% of all bone marrow cancers. In this condition, plasma cells proliferate abnormally, leading to tumor formation in bones.

The Hospital Clínic-IDIBAPS team crafted the new CAR T-cell therapy product, ARI0002h, to cater to these patients.

To gauge the effectiveness of the ARI0002h CAR T-cell therapy, a trial was initiated involving five hospitals to treat 30 myeloma patients who had experienced relapses post two or three prior treatments.

Carlos Fernández de Larrea emphasized the benefits of a fractional initial dose to mitigate severe side effects without compromising the treatment's efficacy.

The findings from The Lancet Oncology revealed that post an 18-month monitoring period, every patient responded positively to the treatment. Moreover, the majority of the responses were complete, with a few being very good partial responses and some being partial responses.

Despite the encouraging outcomes, patients undergoing the ARI0002h treatment still face relapses. Dr. Fernández de Larrea stressed the need for strategies to address this challenge and extend the longevity of the CAR T-cell therapy.

An additional 30 patients were included in this study, and 12 more received the treatment on compassionate grounds.

Currently, the Spanish Agency of Medicines and Medical Devices (AEMPS) is reviewing the documentation based on this study's results for approval.

Study reference:
Oliver-Caldés A, González-Calle V, Cabañas V, et al. Fractionated initial infusion and booster dose of ARI0002h, a humanised, BCMA-directed CAR T-cell therapy, for patients with relapsed or refractory multiple myeloma (CARTBCMAHCB-01): a single-arm, multicentre, academic pilot study. Lancet Oncol 2023.
https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(23)00222-X/fulltext