Public Car-Ts, between access and sustainability: the Spanish model and the Italian challenge
Spain has already approved two born in the academy and Italy is also trying. Is it really possible to democratise advanced therapies?
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Key points
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Idibaps-Hospital Clínic in Barcelona is the only European hospital to have fully developed two academic Car-T therapies approved by a regulatory authority (the Spanish regulator Aemps). These Car-Ts are safe, more accessible and cheaper than those currently available from the pharmaceutical industry. The first, Ari-0001, is indicated for refractory or relapsing B-cell acute lymphoblastic leukaemia, and has already obtained Prime designation from Ema for an accelerated approval pathway; a pathway that for the time being only concerns the adult population, but work is under way to obtain funding for the paediatric population as well. The second, Ari-0002, was created to treat multiple myeloma. Already 500 patients have been treated, and now preparations are under way for several public cell factories in Spain to produce these potentially life-saving therapies. Is this also a viable route in our country? And what do Italian colleagues think of the choices made in Spain?
Monza, Bergamo and Rome in the front line
.Andrea Biondi, a paediatrician and one of Italy's leading leukaemia experts, recently returned from a meeting with Aifa for potential approval of the Car-T therapy, produced in the pharmaceutical workshop of the Tettamanti Foundation in Monza, and in that of the Papa Giovanni XXIII Hospital in Bergamo with haematologist Alessandro Rambaldi. "We presented the preclinical and clinical, phase I and phase II results of our Car-Cik', a therapy that differs from conventional Car-Ts because it uses a non-viral vector and is based on allogeneic cells from a healthy donor. In the future, it could also be derived from cord blood. Two therapeutic indications have been proposed to Aifa: the treatment of relapsed post-transplant acute lymphoblastic leukaemia for indications not covered by authorised products, in cases where, for example, autologous collection is not feasible or in certain categories of patients (e.g. with HIV); and early post-transplant use, in patients at high risk of relapse with early molecular signals, to intervene before overt progression.
The production and sustainability node
.According to Biondi, it is a scalable and affordable strategy that can cover unmet clinical needs and represent an area of innovation for public research: 'the real role of academia is to explore what industry is not addressing, to test Car-Ts in uncovered indications and in alternative use strategies. The academy has a duty to do this'.
The Spanish model, according to the paediatrician, is replicable, 'but we must keep the role of academic research distinct from the development of a commercial product'.
The other big Car-T workshop in Italy is at the Bambin Gesù Hospital, where Franco Locatelli's team is working to have its innovative Car-T for neuroblastoma, the most frequent extracranial solid tumour in paediatric age, approved by the Ema (positive study results with the allogeneic version of Car-T were recently published). The therapy received Prime designation from the regulator, the first time for a Car-T product against solid tumours developed in an academic setting. But at the Bambino Gesù, as in Monza, Car-T has also been tested since 2018 against B-cell acute lymphoblastic leukaemia, the most frequent form of leukaemia in children.
