Preclinical evaluation of CD8+ anti-BCMA mRNA CAR T cells for treatment of multiple myeloma

Leukemia. 2021 Mar;35(3):752-763. doi: 10.1038/s41375-020-0951-5. Epub 2020 Jul 6.

Abstract

Chimeric antigen receptor (CAR) T-cell therapy remains limited to select centers that can carefully monitor adverse events. To broaden use of CAR T cells in community clinics and in a frontline setting, we developed a novel CD8+ CAR T-cell product, Descartes-08, with predictable pharmacokinetics for treatment of multiple myeloma. Descartes-08 is engineered by mRNA transfection to express anti-BCMA CAR for a defined length of time. Descartes-08 expresses anti-BCMA CAR for 1 week, limiting risk of uncontrolled proliferation; produce inflammatory cytokines in response to myeloma target cells; and are highly cytolytic against myeloma cells regardless of the presence of myeloma-protecting bone marrow stromal cells, exogenous a proliferation-inducing ligand, or drug resistance including IMiDs. The magnitude of cytolysis correlates with anti-BCMA CAR expression duration, indicating a temporal limit in activity. In the mouse model of aggressive disseminated human myeloma, Descartes-08 induces BCMA CAR-specific myeloma growth inhibition and significantly prolongs host survival (p < 0.0001). These preclinical data, coupled with an ongoing clinical trial of Descartes-08 in relapsed/refractory myeloma (NCT03448978) showing preliminary durable responses and a favorable therapeutic index, have provided the framework for a recently initiated trial of an optimized/humanized version of Descartes-08 (i.e., Descartes-11) in newly diagnosed myeloma patients with residual disease after induction therapy.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Apoptosis
  • B-Cell Maturation Antigen / genetics
  • B-Cell Maturation Antigen / immunology*
  • CD8-Positive T-Lymphocytes / immunology*
  • Cell Proliferation
  • Drug Evaluation, Preclinical
  • Humans
  • Immunotherapy, Adoptive / methods*
  • Male
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Multiple Myeloma / immunology
  • Multiple Myeloma / metabolism
  • Multiple Myeloma / pathology
  • Multiple Myeloma / therapy*
  • RNA, Messenger / genetics*
  • Receptors, Chimeric Antigen / immunology*
  • Tumor Cells, Cultured
  • Xenograft Model Antitumor Assays

Substances

  • B-Cell Maturation Antigen
  • RNA, Messenger
  • Receptors, Chimeric Antigen
  • TNFRSF17 protein, human