Summary by Edoardo Campodonico and Giorgio Orofino, IRCCS Ospedale San Raffaele, Milan, Italy.
The 1st Joint Educational Meeting of the EBMT ADWP and CTIWP took place in Milan from October 10th to October 12th, 2024 and focused on “CAR T-cells, cell therapies and immunology in autoimmune diseases”. This event is the ultimate product of prolonged efforts from both working parties aimed at creating a fertile and inter-disciplinary discussion between multiple involved specialists. The application of HCT and engineered cell therapies to target difficult-to-treat autoimmune disorders is a “hot topic” at the forefront of medical advancement: the application of such complex treatment modalities to address non-hematologic issues mandates a constant and constructive cross-talk between hematologists and disease specialists. In this respect, the meeting outstandingly achieved this eclecticism and provided food for thought for every attendee, regardless of their field of involvement.
Thursday, 10 October 2024
After a profitable morning devoted to the ADWP and CTIWP business meetings concluded by a unique combined session to discuss joint projects, the meeting officially kicked off with a couple of sessions dedicated to the foundations of cell therapy and autoimmune disorders. Session I served as an appropriate “first course” for non-transplant physicians. Annalisa Ruggeri provided a clear and concise summary of the current state-of-the-art in CAR T-cell therapy as of 2024, most importantly in the field of hematological malignancies. In addition, John Snowden and Raffaella Greco recapitulated in great detail the recent EBMT recommendations for the application of HCT and cellular therapies in both systemic and organ-specific autoimmune diseases. The following session, covered by Hans Ulrich Scherer, Paolo Muraro and Florent Malard introduced us into the mesmerizing labyrinth of rheumatic and neurological autoimmune disorders by tackling the pathophysiologic basis of a selection of disorders potentially treatable by HCT and CAR T, including systemic lupus erythematosus (SLE), vasculitis, multiple sclerosis (MS) and neuromyelitis optica spectrum disorders (NMOSD), including novel therapeutic targets like microbiome. This proved extremely valuable to maximize cross-talk beween the different areas and provide a basis for future discussions.
The first day ended with a keynote lecture held by Chiara Bonini, a world-renowned specialist in the field, concerning the most recent efforts in harnessing T-cell immunity to fight both solid cancers (ie ovarian carcinoma and GastroIntestina tumors), acute myeloid leukemia and-quite interestingly-autoimmune disorders.
Friday, 11 October 2024
The following day opened with a refreshing session focused on new achievements in pathogen-directed immune-reconstitution and in CAR T-cell kinetics’ monitoring in peripheral blood, held by Elena Tassi, Simona Pagliuca and Jurgen Kuball.
Two big sessions on ‘controversies in cellular therapies’ covered both rheumatological than neurological autoimmune diseases. The first panel addressed the role of standard treatments, HCT and CAR-T cells in SLE, SSc and Myositis, thanks to Luca Moroni, Tobias Alexander, Marco Matucci, Nicoletta del Papa, Christina Bergmann and Jorg Henes. During the subsequent session, neurological disorderds such as MS, NMO and Myasthenia Gravis were discussed. Sara Gil-Perotin and Lucia Moiola gave a detailed lecture on the state of the art on conventional treatments, while Matilde Inglese and Basil Sharrack covered the role of HCT for this diseases, finally, Johanna Richter, Wei Wang and Aiden Haghikia introduced the role of CAR-T cells, with incouraging preliminary results already available. CAR-T cells are emerging as promising strategy for refractory patients and could indeed become a breakthrough in the near future for the treatment of autoimmune diseases.
During the last session of the second day, Jurgen Kuball presented the challenges and efforts for the efficient engineering of T cell products, Vanderson Rocha gave a comprehensive overview on the access on CAR-T cells in Brazil, and some interesting data on “Off-the-Shelf” CAR-T and CAR-T regs were presented by Eolia Brissot and Matteo Doglio.
Saturday, 12 October 2024
The last day started with an intense and touching lecture in memory of Riccardo Saccardi and his outstanding contribution to the field given by Paolo Muraro, who brillantly presented the biological and clinical aspects of HCT in MS across the years. The second session of the day was based on CAR-T cell toxicities, with Ana Alarcon updating on CRS and neurological toxicities management, Johanna Richter discussed toxicities on the first patients with MS treated with CAR-T cells and Fabian Müller updated on the excellent safety profile in patients with autoimune disordes treated in their center, with a longer follow up now available.
Subsequently, during the penultimate session, Arsene Mekinian explored the frequent, but often misdiagnosed, autoimmune complications post HCT (other than GvHD), and Fabio Ciceri gave an outstanding lecture on secondary malignancies after cellular therapies, from the decade-long awareness on potential insertional mutagenesis in pivotal gene therapy trials to the most recent concers on potential second malignancies after CAR-T cell therapies.
Finally, during the last session of the meeting, Angela Genchi presented data on a phase I trial taking advantage of neural stem cells for progressive MS, Dominique Farge gave an excellent overview of mesenchymal stromal cell therapy in different disease settings and Richard Burt presented some preliminary data on an innovative approach based on induced pluripotent stem cells for different diseases.
During the meeting many case presentations, selected from the best abstracts submitted at this meeting, have been reported as oral and poster presentations.