Car-T: the first adult patient with rheumatological disease treated in Italy
An interesting article titled “Car-T: the first adult patient with rheumatological disease treated in Italy” has been recently published by About Pharma on 7th Jan , 2025.
The article describes how one of the most relevant discoveries of 2024 is the use of Car-T cells for the treatment of the most serious forms of some autoimmune diseases, which has made important progress towards the clinic.
The author continues, reporting how at the beginning of the year Bambino Gesù Pediatric Hospital in Rome had published the data on the administration of gene therapy to three children suffering from particularly severe forms of systemic lupus erythematosus and dermatomyositis (now 5 children), which months after treatment, they were in disease remission and were no longer taking immunosuppressive drugs.
The article continues, mentioning while a few months later a study published in the New England Journal of medicine demonstrated the safety and effectiveness of infusion with Car-T cells in 15 people with three different autoimmune diseases.
Finally, last December 24, the first adult patient suffering from rheumatological disease (systemic sclerosis or scleroderma) refractory to treatments was treated in Italy, as part of the Catarsis study promoted by Fondazione Policlinico Gemelli Irccs in collaboration with the Bambino Gesù Children's Hospital of Rome.
The article continues describing the Italian Catarsis clinical study and underlines how it opens a new chapter in the treatment of diseases that depend on dysregulation of the immune system. Systemic autoimmune rheumatological diseases such as SLE, systemic sclerosis, dermatomyositis/polymyositis, ANCA-associated vasculitis refractory to usual treatments, but which are also looked at with interest for severe asthma. The study will enroll a total of eight adult patients and will last two years.
Principal investigator is Maria Antonietta D'Agostino, Professor of Rheumatology at the Catholic University of Sacro Cuore and director of the Rheumatology Unit of the Agostino Gemelli Irccs University Polyclinic Foundation.
Indeed, Catarsis is the first academic phase I study in Italy, very important for patients suffering from severe pathologies who can finally aspire to defeat their disease, thanks to the 'reset' of the immune system, but also an example of a cooperative and coordinated project, in which different departments (research, production, clinical, administrative and trial management), operating at different structures, works together to achieve such an important goal.
It is important to underline, from the author, how only about fifty patients with rheumatological diseases have been treated with Car-T so far in the world, most of them in Germany, by the group of Professor Georg Schett of the University of Erlangen (Frederich Alexander) and visiting professor at the Catholic University of Sacro Cuore which was the first in the world to treat these pathologies with Car-T (the results were published in prestigious journals such as Lancet, Nature Medicine and New England Journal of Medicine).
Reading the article we understand how the transition from open systems to closed systems, mobile and/or prefabricated units is the right direction, for structures who would like to sustain this kind of research, and it implies the need to open discussion tables with ATMP cell factory manufacturing and quality managers, creating a consistent level of awareness on the added values which an isolator can offer in terms of scalability, flexibility, and sustainability .
See our website for more information on closed system and isolators https://bioairatmpsolutions.com
If you would like to have more info on the article clinck on this link