Tumors, Car-T cell therapy: first 3 children successfully treated

The first three children undergoing therapy with freshly obtained Car-T cells in Italy were successfully treated, thanks to an innovative automated production. The three young patients had a particular type of leukemia – acute lymphoid leukemia with B-cell precursors (Lla-Bcp) – which proved refractory to all conventional therapies. This was announced by the Ministry of Health in a note. As early as two weeks after the infusion, complete remission of the disease was ascertained in the three children. For them, conventional treatments had not produced the expected results. One of the three children had even failed an allogeneic hematopoietic stem cell transplant procedure, the ministry recalls. The three children were treated with genetically modified cells produced by the pharmaceutical workshop of the Bambino Gesù pediatric hospital in Rome through an automated system, developed as part of the Car-T Italia project. The project was promoted by the Parliament, which allocated 10 million euros to the Ministry of Health, to make this new therapeutic approach available throughout the country. For all three children, time has now matured to assess response to treatment and complete remission of the disease has been documented. The three small patients were included in the new experimental treatment protocol and treated with cells that were genetically modified to express a receptor called Car (Chimeric Antigen Receptor) directed towards the tumor target Cd19. “The automated system allows to produce several batches of Car-T cells simultaneously within the same environment in just two weeks, to reduce the staff required for manufacturing, to contain the risk of product contamination and to generate highly standardized procedures and, therefore, reproducible, significantly limiting production costs. Thanks to the characteristics of this approach, the therapy is made more accessible even for patients suffering from the most aggressive forms of the disease “, underline the experts. In the three cases it was possible to obtain a number of Car-T cells much higher than the dose necessary for the first phase of the treatment, allowing to freeze part of the cells produced, for possible future consolidation treatments. A spare capacity, which demonstrates the feasibility and efficiency of the production process – continues the Ministry of Health – In the three patients the infusion of the fresh product was well tolerated, with only fever, due to the release of inflammatory molecules (cytokines) from Car-T cells. The success on the three children “is an extremely promising preliminary result – it is emphasized – confirming how the new Car-T cell platform demonstrates a remarkable efficacy against refractory forms of Lla-Bcp, despite having been tested only at the dose level. lower, as foreseen by the trial phase I. The study will continue with the evaluation of two other dose levels and with the activation of phase II, in which the recommended dose will be used, identified in phase I “. on Car-T cells for malignant haematological diseases and solid tumors is conducted by the Irccs of the Alliance Against Cancer Network (Acc) on the basis of the agenda of the Chamber of Deputies of 30 December 2019, which outlined the implementation path for the development of these new therapies. The project is aimed at better understanding the mechanisms that regulate the efficacy or possible toxicity associated with the use of this innovative form of immunotherapy, to develop this therapeutic approach also for patients with solid neoplasms and to identify the productive processes capable of to make these therapies available to as many patients as possible. “Research on advanced therapies is of strategic value, in order to offer citizens the best possible treatment options. The results of this important project are a significant step forward in this direction and confirm that it is essential to continue investing in research “underlines the Minister of Health, Roberto Speranza, commenting on the results obtained from the Car-T Italia project.” As project coordinator and head of the Bambino Gesù hospital group, I am particularly happy for this result – comments Franco Locatelli, director of the Department of Oncohematology of the Child Jesus and President of the Higher Health Council – The decision of the Italian Parliament, to which all of us researchers belonging to the Project are extraordinarily grateful, to invest in such an innovative field thus finds a concrete therapeutic realization that will certainly be followed by others applications in patients suffering from different oncological pathologies “.