Project Term
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Ospedale Pediatrico Bambino Gesù
Rome,
Italy
Project Summary
CD7-CAR T cells with CD7-PEBL represents an innovative technology that enables the production of CAR T cells able to escape the fratricide for the treatment of relapsed/refractory T-ALL. At Bambino Gesù Children’s Hospital, in collaboration with Prof. Campana (Singapore), we have tested the safety of these CAR T cells in children/young adults with relapsed/refractory T-ALL obtaining promising results, in terms of both safety and efficacy. We now aim to activate a Phase 2 multicenter study to further explore the efficacy of this approach.
Lay Abstract
CD7-CAR T cells with CD7-PEBL represents an innovative technology that was developed by the group of Prof. Campana to enable the production of CAR T cells directed against the blasts of T-ALL and able to escape the fratricide. Thanks to the collaboration with his group, at Bambino Gesù Children’s Hospital, we have activated a clinical trial to test the safety and efficacy of these CAR T cells in children/young adults with relapsed/refractory T-ALL. The study enrolls children and young adults (from 6 months up to 25 years) with CD7-positive (> 98% CD7 expression on blast cells) T-ALL or lymphoblastic lymphoma (LL) and one of the following: first or subsequent relapse; relapse after allogeneic hematopoietic stem cell transplantation (if at least 100 days have elapsed and there is no evidence of active GvHD); CNS disease; extramedullary disease; refractory T-ALL/LL. The initial phase of the study (phase 1), aimed at evaluating the toxicity of the approach and identifying the recommended cell dose, has been completed and we observed a very favorable safety profile of the treatment, associated with a very promising efficacy.
We now aim to activate a multicenter study to further explore the efficacy of this approach (phase 2). The phase 2 will enroll the same patient population of the phase 1 portion of the study. The primary objective of the study will be to define the ability of the approach to induce a complete elimination of the disease (so called complete remission – CR). In order to evaluate this objective, we will assess the proportion of patients that achieve the CR within 28 days after infusion of the CAR T cells.
If the results obtained so far in terms of safety and efficacy will be confirmed also in the phase 2 study, this approach will induce a remission of the disease in a substantial proportion of children/young adults with relapsed/refractory T-ALL, enabling them to receive a consolidation with allogeneic hematopoietic stem cell transplantation in the best conditions to benefit from the procedure.
Program