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Base edited CD7 CAR T-cell therapy for childhood R/R T-ALL: preliminary results from a phase I trial

May 13, 2024
Learning objective: After reading this article, learners will be able to cite a new clinical development in ALL.

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The survival outcomes for patients with relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) are poor; therefore, there is a need for novel targeted therapies in this patient population.

Here, we summarize preliminary results of a phase I trial (EudraCT:2021-004312-25) evaluating the efficacy and safety of CD7-directed chimeric antigen receptor (CAR) T-cell therapy in children with R/R T-cell ALL, presented by Chiesa at the 50th Annual Meeting of the EBMT.

Study design1

This was an open-label, single-center phase I study including patients aged 6 months to 16 years with R/R CD7+ (>99%) T-cell malignancies and bone marrow involvement who were eligible for allogeneic hematopoietic stem cell transplantation (allo-HSCT).

  • The treatment schema is outlined in Figure 1
  • The primary endpoint was safety
  • Secondary endpoints included
    • disease remission by Day 28;
    • 1-year overall survival and disease-free survival;
    • time and duration of remission;
    • expansion, persistence, and elimination of base-edited CAR T-cells; and
    • side effects.

Figure 1. Treatment schema* 

Allo-HSCT, allogeneic hematopoietic stem cell transplantation; BM, bone marrow; CAR, chimeric antigen receptor; MRD, minimal residual disease.
*Data from Chiesa.1
Dose: 0.2−2.0 × 106 per kilogram; max: 5 × 104 per kilogram TCRαβ T cells

Key findings1,2

  • A total of 10 patients were included in the study
  • Patient 1 with refractory T-ALL underwent a bone marrow transplantation from a 10/10 matched-unrelated donor after total body irradiation/etoposide conditioning
    • experienced early relapse at 6 weeks post-HSCT
    • had 9% disease prior to lymphodepletion
  • Patient 2 had refractory T-ALL and 80% blasts prior to lymphodepletion
  • Patient 3 had bone marrow and central nervous system relapsed T-ALL following two allo-HSCTs
  • The outcomes for the three patients who subsequently received base-edited CD7-directed CAR T-cell therapies are summarized in Figure 2

Figure 2. Outcomes of CD7-directed CAR T-cells in children with R/R T-ALL*

CAR, chimeric antigen receptor; CMV, cytomegalovirus; CNS, central nervous system; CR, complete remission; HSCT, hematopoietic stem cell transplantation; ICANS, immune effector cell-associated neurotoxicity syndrome; IR, immune reconstitution; MRD, minimal residual disease; PCR, polymerase chain reaction; T-ALL, T-cell acute lymphoblastic leukemia.

*Data from Chiesa.1


Key learnings
  • CD7-directed CAR T-cell therapy demonstrated good preliminary anti-leukemic responses and a good safety profile in high-risk patients with R/R T-ALL.  
  • A high-risk of infections was observed, thus screening and monitoring  of infections is essential in this patient population.
  • The use of subsequent allo-HSCT following CAR T-cell infusion allowed for normal donor-derived T-cell immune reconstitution.

  1. Chiesa R. Base edited CD7 CAR-T cell therapy in relapsed T-ALL. Oral abstract #Paed1-3. 50th Annual Meeting of the European Society for Blood and Marrow Transplantation. Apr 16, 2024. Glasgow, UK
  2. Chiesa R, Georgiadis C, Syed F, et al. Base-edited CAR7 T Cells for relapsed T-cell acute lymphoblastic leukemia. N Engl J Med. 2023;389(10):899-910. DOI: 1056/NEJMoa2300709


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