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The academic trial at Hospital Clínic and Sant Joan de Déu in Barcelona that tested a CAR-T cell therapy made in the hospital itself, and led to Spain authorising it in 2021.
CART19-BE-01 tested ARI-0001, a CD19-directed CAR-T cell product with 4-1BB and CD3-zeta signalling designed and manufactured at Hospital Clínic de Barcelona, in adults and children with relapsed or refractory CD19-positive leukaemia or lymphoma. Patients received cyclophosphamide and fludarabine and then ARI-0001 cells, at first as a single dose and, after a protocol amendment, split into three fractions (10%, 30% and 60%) with the full dose given only in the absence of cytokine release syndrome.
In the 2021 report in Molecular Therapy, 58 patients were included and 47 treated: 38 with acute lymphoblastic leukaemia, 8 with non-Hodgkin lymphoma and 1 with chronic lymphocytic leukaemia. In acute lymphoblastic leukaemia, grade 3 or higher cytokine release syndrome occurred in 13.2% (26.7% before the amendment against 4.3% after), and the measurable residual disease-negative complete response rate at day 100 was 71.1%. The registry lists 50 participants and completion on 13 September 2022. On these results the AEMPS authorised ARI-0001 under the hospital exemption in February 2021 for adults over 25 with relapsed or refractory acute lymphoblastic leukaemia.
Spain's use of the hospital exemption is a working model for public hospitals making cell therapies at lower cost; the paper is the account of how it was done by the people who did it.
This is the evidence behind the AEMPS's February 2021 authorisation of ARI-0001 under the hospital exemption, which let a public hospital make and give its own CAR-T to adults with relapsed acute lymphoblastic leukaemia. The fractionated dosing it introduced is the approach later used for the hospital's BCMA CAR-T.
Shares Julio Delgado, Instituto de Salud Carlos III (ISCIII), Manel Juan, Point-of-care and decentralised cell manufacturing.
Shares Julio Delgado, Instituto de Salud Carlos III (ISCIII), Manel Juan, Point-of-care and decentralised cell manufacturing.
Shares Varnimcabtagene autoleucel, Point-of-care and decentralised cell manufacturing, Cytokine release syndrome (CRS), CD19.
Shares The hospital exemption pathway for advanced therapies and the case of the CAR-T ARI-0001, Varnimcabtagene autoleucel, Hospital Clínic de Barcelona / IDIBAPS, CAR-T cell therapy.
Shares Instituto de Salud Carlos III (ISCIII), CART19-BE-01: A Multicenter Trial of ARI-0001 Cell Therapy in Patients with CD19(+) Relapsed/Refractory Malignancies, Hospital Clínic de Barcelona / IDIBAPS.
Shares Varnimcabtagene autoleucel, Point-of-care and decentralised cell manufacturing, CD19, CAR-T cell therapy.
Shares Varnimcabtagene autoleucel, Point-of-care and decentralised cell manufacturing, ICANS (neurotoxicity), Cytokine release syndrome (CRS).
Shares ICANS (neurotoxicity), Cytokine release syndrome (CRS), CD19, CAR-T cell therapy.
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