Results of ARI‐0001 CART19 cell therapy in patients with relapsed/refractory CD19‐positive acute lymphoblastic leukemia with isolated extramedullary disease

Abstract

We evaluated outcomes of 18 patients with isolated extramedullary disease (iEMD) relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL) treated with the CD19-directed CAR T cells ARI-0001 in two centers (adult and pediatric), including patients treated in the CART19-BE-01 trial and the consecutive compassionate use program.

A data management system for precision medicine

“…Figure 4). The data for the AIDPATH use case 5 was derived from multicentre clinical trial ARI-0001 (54,55) and ARI-0002h (56).…”

The academic point‐of‐care anti‐CD19 chimeric antigen receptor T‐cell product varnimcabtagene autoleucel (ARI‐0001 cells) shows efficacy and safety in the treatment of relapsed/refractory B‐cell non‐Hodgkin lymphoma

“…In Spain, the results from the CART19-BE-01 clinical trial evaluating the safety and efficacy of the academic anti-CD19 CART-cell product varnimcabtagene autoleucel (var-cel, ARI-0001 cells) manufactured by Hospital Clínic de Barcelona (HCB), [9][10][11] led to its approval, as Hospital Exemption, by the Spanish Agency of Medicines (AEMPS) for patients older than 25 years of age with R/R acute lymphoblastic leukaemia (ALL). 12 The product's price was subsequently agreed upon with the Spanish Ministry of Health at €89 270.…”

Chimeric Antigen Receptor (CAR) T-Cell Products for Pediatric Cancers: Why Alternative Development Paths Are Needed

“…The first academic CD19-specific CAR T-cell product was granted eligibility to the PRIority MEdicines (PRIME) scheme of the EMA, ARI0001. 41 This regulatory support of single-academic centers to act as license holders is a positive step forward. However, the expense, skills, effort of licensing, and the necessary (legal) postmarketing responsibilities for a complex agent to obtain a license for a rare disease exceeds the existing infrastructure of most academic hospitals.…”

“…Acknowledgment of the potential of cell and immune therapies to advance medicine has led an increasing number of academic institutions in Europe and beyond to acquire the necessary infrastructure and expertise to act as manufacturing sites for ATMPs. 41,[46][47][48] a proof-of-concept study for ALL and then adopting this for solid tumors in children and adolescents. Overall, this will enable productive cooperation between industry, academia, and regulators to overcome current roadblocks in the development of CAR T-cell therapeutics for children and adolescents and ultimately save lives.…”

“…Acknowledgment of the potential of cell and immune therapies to advance medicine has led an increasing number of academic institutions in Europe and beyond to acquire the necessary infrastructure and expertise to act as manufacturing sites for ATMPs. 41,46-48 A European network of academic ATMP manufacturing centers could overcome limitations of cross-border trafficking of products over long distances and close the current gap between needs and availability (Fig 1). In close cooperation between biomedical/pharmaceutical industry and academia, we are currently establishing the infrastructure of this network, starting with a proof-of-concept study for ALL and then adopting this for solid tumors in children and adolescents.…”