An "off-the-shelf" CD2 universal CAR-T therapy for T-cell malignancies

Leukemia. 2023 Dec;37(12):2448-2456. doi: 10.1038/s41375-023-02039-z. Epub 2023 Oct 5.

Abstract

T-cell malignancies are associated with frequent relapse and high morbidity, which is partly due to the lack of effective or targeted treatment options. To broaden the use of CAR-T cells in pan T-cell malignancies, we developed an allogeneic "universal" CD2-targeting CAR-T cell (UCART2), in which the CD2 antigen is deleted to prevent fratricide, and the T-cell receptor is removed to prevent GvHD. UCART2 demonstrated efficacy against T-ALL and CTCL and prolonged the survival of tumor-engrafted NSG mice in vivo. To evaluate the impact of CD2 on CAR-T function, we generated CD19 CAR-T cells (UCART19) with or without CD2 deletion, single-cell secretome analysis revealed that CD2 deletion in UCART19 reduced frequencies of the effector cytokines (Granzyme-B and IFN-γ). We also observed that UCART19ΔCD2 had reduced anti-tumor efficacy compared to UCART19 in a CD19+NALM6 xenograft model. Of note is that the reduced efficacy resulting from CD2 deletion was reversed when combined with rhIL-7-hyFc, a long-acting recombinant human interleukin-7. Treatment with rhIL-7-hyFc prolonged UCART2 persistence and increased survival in both the tumor re-challenge model and primary patient T-ALL model in vivo. Together, these data suggest that allogeneic fratricide-resistant UCART2, in combination with rhIL-7-hyFc, could be a suitable approach for treating T-cell malignancies.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Antigens, CD19
  • Humans
  • Immunotherapy, Adoptive / methods
  • Mice
  • Neoplasm Recurrence, Local
  • Precursor T-Cell Lymphoblastic Leukemia-Lymphoma*
  • Receptors, Antigen, T-Cell
  • Receptors, Chimeric Antigen* / genetics
  • T-Lymphocytes

Substances

  • Receptors, Chimeric Antigen
  • Receptors, Antigen, T-Cell
  • Antigens, CD19